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83 studies
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- Eczema / atopic dermatitis
Molecular Signatures of Cutaneous Dupilumab Response
Studying how dupilumab affects immune cells in eczema skin
📍 University of California, San Francisco · San Francisco, CA · Get directions →
This study examines the effect of IL4RA blockade with dupilumab on the immune cells of atopic dermatitis skin lesions.
- Sickle cell disease
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Testing different doses of DISC-3405 to check safety in sickle cell disease
📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →
This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.
- Eczema / atopic dermatitis
A Study to Learn About Study Medicine Called PF-08049820 in People With Eczema
Testing whether an experimental medicine is safe and works for eczema
📍 Northridge Clinical Trials · Northridge, CA · +3 more US sites · Get directions →
The purpose of this study is to learn if the study medicine (PF-08049820) is safe and effective for the treatment of atopic dermatitis (AD), also known as eczema, or atopic eczema. People with this condition may have severe itching and rashes on the skin.
- Sickle cell disease💵 May compensate
Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Testing higher doses of VAS-101 in people with stable sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.
- Lupus (SLE)Eczema / atopic dermatitis
Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis
Testing whether baricitinib is safe in people with Job syndrome and lupus or eczema
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin and lung infections and problems with blood vessels, connective tissues, and bones.
- Eczema / atopic dermatitis
Cardamom and Topical Roseomonas in Atopic Dermatitis
Testing cardamom and a skin-applied germ to treat eczema
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Atopic dermatitis (AD), also called eczema, is a chronic skin condition. AD can make skin dry and itchy, and sometimes it can lead to serious health problems, such as asthma, food allergies, eye infections, and sleep problems.
- Sickle cell disease
A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)
Testing the safety and how an oral carbon monoxide liquid works in sickle cell disease
📍 Cevaxin - The Panama Clinic · Panama City, · Get directions →
This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.
- Sickle cell disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults
📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →
The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.
- Eczema / atopic dermatitis
A Long-term Safety and Efficacy Study Evaluating APG777 in Atopic Dermatitis
Testing the long-term safety and effects of a drug in people with eczema
📍 Investigational Site · Fountain Valley, CA · +21 more US sites · Get directions →
This is a multicenter, double-blind, Long-Term Extension (LTE) study to evaluate the long-term safety and efficacy of APG777 in patients with moderate-to-severe AD who have completed treatment in an APG777 Parent Study (NCT06395948). The LTE study will consist of 3 periods: 1) Screening Visit will coincide with the last visit of the Maintenance Period in the Parent Study 2) Extended Treatment Period 3) Post-treatment Follow-up Period.
- Eczema / atopic dermatitis
A Study of BBT001 in Healthy Volunteers (HVs) and in Adult Patients With Atopic Dermatitis (AD)
Testing BBT001 in healthy adults and adults with eczema
📍 First OC Dermatology · Irvine, CA · +2 more US sites · Get directions →
This is a Phase 1, randomized, blinded, placebo controlled, single-ascending dose (SAD) and multiple-ascending dose (MAD) study of BBT001 in healthy volunteers (HVs) and adult patients with moderate to severe Atopic Dermatitis (AD).
- Eczema / atopic dermatitis
Dupilumab De-escalation in Pediatric Atopic Dermatitis
Testing whether stopping or lowering eczema treatment works for kids
📍 Johns Hopkins Univerisity · Baltimore, MD · Get directions →
This is a pilot investigator-blinded, randomized clinical trial to assess the feasibility of dupilumab treatment discontinuation or dose-reduction in children aged 1-17 years who have achieved sustained atopic dermatitis (AD) control on dupilumab.
- Sickle cell disease
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
Testing whether extra arginine lowers pain in children with sickle cell
📍 Children's Healthcare fo Atlanta at Hughes Spalding · Atlanta, GA · +1 more US site · Get directions →
The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.
- Eczema / atopic dermatitis
A Study Evaluating BFB759 in Moderate to Severe Atopic Dermatitis
Testing a medicine for moderate to severe eczema, compared with a placebo
📍 Mountain West Derm - Blackhart PLLC dba Skin Cancer and Dermatology Institute · Phoenix, AZ · +39 more US sites · Get directions →
This is a double-blind, placebo-controlled study where subjects are participating for approximately 36 to 40 weeks. The study compares how well BFB759 works and how safe it is compared with a placebo.
- Sickle cell disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Testing a once-daily mouth medicine in teens with sickle cell disease
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.
- Eczema / atopic dermatitis
A Study to Evaluate the Efficacy and Safety of IMG-007 in Adult Participants With Moderate-to-Severe Atopic Dermatitis
Testing different doses of a medicine versus placebo for eczema
📍 Inmagene Site 1 · Lancaster, CA · +14 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy and safety of different dose regimens of IMG-007, compared to placebo.
- Eczema / atopic dermatitis
Study to Evaluate Soquelitinib in Participants With Moderate to Severe AD
Testing how different doses of soquelitinib work for moderate to severe atopic dermatitis
📍 Corvus Clinical Trials · South San Francisco, CA · Get directions →
The main purpose of this study is to see how well different doses of soquelitinib work in participants with atopic dermatitis (AD) as compared to a placebo (pill with no medicine in it). To check this, the study doctors will examine participants' skin regularly to track how their AD changes during the study.
- Eczema / atopic dermatitis
Ph 1a/1b Single Ascending Dose and Multiple Ascending Dose Study of ARQ-234
Testing a new drug dose in people with eczema and healthy volunteers
📍 Clinical Site 101 · Fair Lawn, NJ · Get directions →
This is a first-in-human, Phase 1, double-blind, randomized, placebo-controlled, dose-escalation study evaluating ARQ-234. The study is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ARQ-234 in two populations: healthy volunteers and participants with moderate to severe atopic dermatitis (AD).
- Sickle cell disease
RH Genotype Matched RBC Transfusions
Testing whether matching donor red blood cells by RH type helps people with sickle cell who need
📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →
To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.
- Eczema / atopic dermatitis
Treatment of Atopic Dermatitis and Alopecia Areata With Abrocitinib in Individuals With Down Syndrome
Testing a daily medicine for eczema and hair loss in people with Down syndrome
📍 Icahn School of Medicine at Mount Sinai · New York, NY · Get directions →
This is a single-center, open-label, basket phase 2b trial that will enroll Down Syndrome (DS) participants with at least one inflammatory skin condition (Atopic Dermatitis (AD) and/or Alopecia Areata (AA)). Patients will receive Abrocitinib 100 mg daily for 12 weeks.
- Eczema / atopic dermatitis
A Long Term Extension Study to Evaluate the Safety and Efficacy of Afimkibart (RO7790121) in Participants With Atopic Dermatitis
Checking the long-term safety and effects of an eczema medicine in people who previously studied it
📍 Hamilton Research, LLC · Alpharetta, GA · +2 more US sites · Get directions →
This study will assess long-term safety and efficacy of Afimkibart (also known as RO7790121) in participants with Atopic Dermatitis (AD) who participated in previous afimkibart clinical trials.
- Sickle cell disease
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Testing a one-time blood stem cell transplant using gene editing for severe sickle cell disease
📍 University of California, Los Angeles · Los Angeles, CA · +1 more US site · Get directions →
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).
- Eczema / atopic dermatitis
Lebrikizumab in Moderate-to-severe Atopic Dermatitis
Testing a medicine called lebrikizumab for long-term improvement in eczema
📍 Physioseq USA - CA · Folsom, CA · +1 more US site · Get directions →
This research is studying a drug already approved for the treatment of atopic dermatitis (AD). This research collects health-related information and blood and skin samples to understand if the study drug, lebrikizumab, leads to long-term improvement in AD skin.
- Sickle cell disease
Cannabidiol in Sickle Cell Disease
Testing twice-daily cannabidiol doses for people with sickle cell disease
📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →
Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.
- Sickle cell disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Testing early heart screening and iron overload treatment in people with sickle cell disease
📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Sickle cell disease
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
Testing whether routine red blood cell transfusions in pregnancy reduce sickle cell flare-ups or
📍 Grady Health System · Atlanta, GA · Get directions →
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD. RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.
- Eczema / atopic dermatitis
The Impact of Botox on Neuroimmune Interactions in Atopic Dermatitis
Testing how Botox changes skin immune signals in people with eczema
📍 University of Pittsburgh Medical Center · Pittsburgh, PA · +1 more US site · Get directions →
The purpose of this study is to understand cellular and molecular interactions in the skin of participants with mild-to-moderate AD, and how botulinum toxin alters these interactions.
- Eczema / atopic dermatitis
Ethnic Differences in Mechanisms of Action of Dupilumab
Studying how a common eczema treatment works differently by race
📍 University of Michigan · Ann Arbor, MI · Get directions →
Previous research has shown that Asian and African Americans are more likely to develop atopic dermatitis (AD) than their Caucasian counterparts. However, limited information is known about AD in Asian and African American populations because most molecular studies have focused on Caucasians with AD.
- Sickle cell disease
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related
📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?
- Sickle cell disease
Gene Editing For Sickle Cell Disease
Testing whether gene editing is safe for people with sickle cell disease
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
- Sickle cell disease
Allo HSCT for High Risk Hemoglobinopathies
Testing donor stem cell transplant for people with high-risk sickle cell
📍 Masonic Cancer Center · Minneapolis, MN · Get directions →
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
- Eczema / atopic dermatitis
Safety and Pharmacokinetics of LPX-TI641 in Atopic Dermatitis and Psoriasis
Testing the safety of LPX-TI641 and how the body handles it in eczema
📍 Red River Research Partners · Fargo, ND · +1 more US site · Get directions →
The goal of this clinical trial is to study the drug LPX-TI641 in patients with atopic dermatitis and psoriasis. We will compare the safety and tolerability of LPX-TI641 to placebo ( a look-alike solution) that contains no drug.
- Eczema / atopic dermatitis
FIH Study to Evaluate the Tolerability of PF-07832837 in Healthy Adults and Patients
Testing the safety of PF-07832837 in healthy people and eczema patients
📍 Anaheim Clinical Trials, LLC · Anaheim, CA · +2 more US sites · Get directions →
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetics of escalating single and repeat doses of PF-07832837 in healthy participants and in participants with moderate to severe atopic dermatitis. An additional goal is to assess the pharmacodynamics of PF-07832837 in participants with moderate to severe AD, including potential effects on clinical signs and symptoms
- Eczema / atopic dermatitis
A 3-part Study of SYX-5219 in Healthy Volunteers and Participants With Atopic Dermatitis
Testing how a new medicine works and is tolerated in people with eczema
📍 Sitryx Clinical Site · Arkansas City, AR · +5 more US sites · Get directions →
The purpose of this study is to evaluate the study drug, SYX-5219, in a multi-part First-in-Human (FiH) study to be conducted in healthy volunteers and participants with Atopic Dermatitis (AD). The objectives of this study are to determine the safety, tolerability and levels of SYX-5219 in the blood and urine when SYX-5219 is given in each part of the study (SAD, MAD, Food Effect and Participants with AD).
- Sickle cell disease
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease
📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.
- Sickle cell disease
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
Testing how BMS-986470 affects the body and safety in sickle cell disease
📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.
- Sickle cell disease
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Measuring how long red blood cells last in sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early.
- Eczema / atopic dermatitis
Targeted Investigation of Microbiome 2 Treat Atopic Dermatitis (TIME-2)
Testing an approach involving the skin microbiome to treat eczema
📍 University of California, San Diego: Dermatology Clinical Trials Unit · San Diego, CA · +7 more US sites · Get directions →
This is a Phase 1b, randomized, placebo/vehicle-controlled, double-blinded, multi-center trial. It is designed to assess the safety and efficacy of S.
- Sickle cell disease
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Testing a partial stem cell transplant from matched donors for severe sickle cell disease or
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens.
- Sickle cell disease
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Testing how safe escalating doses of fostamatinib are in people with stable sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.
- Sickle cell disease
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Testing gene editing in stem cells for severe sickle cell disease
📍 Boston Children's Hospital · Boston, MA · Get directions →
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Sickle cell disease
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Testing empagliflozin to treat kidney disease related to sickle cell
📍 University of Illinois Chicago, Sickle Cell Center · Chicago, IL · Get directions →
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA.
- Eczema / atopic dermatitis
A Phase 2b Study of the Effects of Camoteskimab in Adults With Moderate-to-Severe Atopic Dermatitis
Testing how camoteskimab works in adults with moderate to severe eczema
📍 AllerVie Clinical Research · Birmingham, AL · +32 more US sites · Get directions →
This is a phase 2b, multicenter, randomized, double-blind, placebo-controlled study.
- Eczema / atopic dermatitis
A Study to Assess Molecular Changes in Adult Participants With Moderate to Severe Hidradenitis Suppurativa or With Moderate to Severe Atopic Dermatitis Receiving Subcutaneous Injections of Lutikizumab
Testing how a skin injection changes molecules in adults with eczema
📍 Physioseq, LLC /ID# 267266 · Sacramento, CA · +1 more US site · Get directions →
Hidradenitis suppurativa (HS) and atopic dermatitis (AD) are chronic inflammatory skin diseases that lead to the development of skin lesions and symptoms such as pain and discomfort. The purpose of this study is to assess molecular changes in adult participants with moderate to severe HS or with moderate to severe AD.
- Sickle cell disease
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
Testing a transplant plan with daratumumab to reduce blood cell failure in sickle cell disease
📍 Children's National Hospital · Washington D.C., DC · Get directions →
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.
- Eczema / atopic dermatitis
Evaluate Efficacy and Safety of Repeat Subcutaneous Doses of FB825 in Adults With Moderate-to-Severe Atopic Dermatitis
Testing repeat under-the-skin doses of FB825 for adult eczema
📍 First OC Dermatology · Fountain Valley, CA · +5 more US sites · Get directions →
This is a randomized, placebo-controlled and double-blind study to evaluate the efficacy, pharmacokinetics, and Safety of repeat subcutaneous doses of FB825 in adults with moderate-to-severe atopic dermatitis.
- Eczema / atopic dermatitis
A Double-Blind, Randomized, Vehicle-Controlled Phase 2 Study to Assess the Efficacy and Safety of GX-03 in Adult Subjects With Moderate to Severe Atopic Dermatitis (Eczema)
Testing a topical ointment to see if it helps adult eczema and is safe
📍 ALS Global · Irving, TX · Get directions →
This is a Phase 2, randomized, double-blind, vehicle-controlled clinical study evaluating the efficacy and safety of GX-03 topical ointment in adult subjects with moderate to severe atopic dermatitis (eczema). The study plans to enroll up to 120 eligible patients with a target of at least 100 completing the study and an ability to expand enrollment up to 200 subjects based on a pre-specified interim assessment conducted by an Independent Data Monitoring Committee (IDMC).
- Sickle cell disease
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Testing whether motixafortide is safe and can raise stem cells in people with sickle cell disease
📍 University of Alabama at Birmingham · Birmingham, AL · +2 more US sites · Get directions →
This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.
- Sickle cell disease
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
Testing different ways to use hydroxyurea for treating sickle cell anemia
📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.
- Sickle cell disease
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Testing the safety and effects of panobinostat in adults with sickle cell disease
📍 Augusta University · Augusta, GA · Get directions →
The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.
- Sickle cell disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Testing how a new medicine works in healthy people and sickle cell patients
📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).
- Sickle cell disease
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and
📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Sickle cell disease
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
Testing a stem cell transplant plan for children with sickle cell disease using a mismatched blood
📍 Alberta Children's Hospital · Calgary, Alberta · Get directions →
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression.
- Sickle cell disease
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Testing stem cell transplant with specific immune cell removal for sickle cell
📍 New York Medical College · Valhalla, NY · Get directions →
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.
- Sickle cell disease
Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease
Testing whether an IV dose of citrulline can treat sudden pain in hospitalized sickle cell patients
📍 Children's National Hospital · Washington D.C., DC · Get directions →
The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline.
- Sickle cell disease
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Testing an injectable birth control shot to reduce pain in women with sickle cell disease
📍 Emory University · Atlanta, GA · +1 more US site · Get directions →
This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain.
- Sickle cell disease
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
Testing the safety of a medicine (given under the skin) in adults with sickle cell disease
📍 UAMS Medical Center · Little Rock, AR · +4 more US sites · Get directions →
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD.
- Sickle cell disease
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
Testing a vein drug (hemopexin) to treat pain crises from sickle cell
📍 Univ. of California, San Francisco Health Care · Oakland, CA · +15 more US sites · Get directions →
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.
- Sickle cell disease
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
Testing whether children with sickle cell disease can take daily ADHD medicine and monitoring its
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects. The study will assess any thinking or attention problems participants may have both before taking this drug and after.
- Sickle cell diseaseDementia / Alzheimer's
Biomarker-based Trial of NPC-1 for Alzheimer's Pathology
Testing an early treatment to change Alzheimer’s blood markers in adults with memory decline
📍 Massachusetts General Hospital · Boston, MA · Get directions →
This early phase, open label, single arm clinical trial will determine the intraindividual safety, tolerability and effects of NPC1 (parthenolide and ipriflavone) on blood-based biomarkers of Alzheimer's disease (AD) pathology among adults with subjective cognitive decline, mild cognitive impairment, or Alzheimer's disease and objective indicators of seeding AD pathology
- Sickle cell disease
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell
📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →
This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.
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