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45 studies

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  • DepressionAnxiety

    Acceptance and Commitment Therapy for Adolescents and Young Adults With Sickle Cell Disease

    Testing whether therapy sessions can help teens and young adults with sickle cell cope with

    📍 Children's Hospital Los Angeles · Los Angeles, CA · Get directions →

    The current study seeks to build on previous research that demonstrates the efficacy of Acceptance and Commitment Therapy in combating stigma by investigating the feasibility and acceptability of a protocol to support Black adolescents and young adults in coping with race related stress. The study will consist of a small, purposeful, non-randomized sample (N = 30) of clients enrolled into a 10-session Acceptance and Commitment Therapy group.

    ✓ Study care freePhase 1Ages 14–21Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Anxiety

    Study to Evaluate the Efficacy and Safety of VQW-765 for the On-Demand Treatment of Social Anxiety Disorder

    Testing a single oral dose medicine versus placebo for social anxiety in adults

    📍 Vanda Investigational Site · Phoenix, AZ · +30 more US sites · Get directions →

    This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of a single oral dose of VQW-765 compared to placebo in adults with social anxiety disorder.

    ✓ Study care freePhase 3Ages 18–65Learn more →
  • DepressionAnxiety

    ACT for Individuals With MNCD and Their Care Partners

    Testing acceptance commitment therapy for depression and anxiety in dementia

    📍 Texas Tech University Plaza Building · Lubbock, TX · Get directions →

    This project aims to implement and evaluate a dyadic intervention (i.e., acceptance commitment therapy) for persons with AD/ADRD and their care partners. We hypothesize the intervention will be feasible, acceptable, and show preliminary efficacy of the dyadic interveniotns.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    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

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–55Learn more →
  • DepressionAnxiety

    Perinatal Depression & Anxiety (PDA) and Maternal Gut Microbiome

    Studying how gut bacteria in pregnant people relates to depression and anxiety

    📍 Sunnybrook Health Science Center · Toronto, Ontario · Get directions →

    Perinatal depression and anxiety (PDA) are the leading causes of maternal mortality in developed countries. Women with a history of depression have a 20 fold higher risk of PDA at subsequent pregnancies.

    ✓ Study care freePhase 2Ages 18–43WomenLearn more →
  • DepressionAnxiety

    Nebulized Ketamine for the Treatment of Major Depressive Disorder

    Testing a breathing mist of ketamine to reduce symptoms of major depression

    📍 Maimonides Medical Center · Brooklyn, NY · Get directions →

    This is a double-blind active placebo controlled clinical trial for individuals with moderate to severe depression. The purpose of this study is to assess if nebulized ketamine can reduce depressive symptoms.

    ✓ Study care freePhase 3Ages 18–88Learn more →
  • Sickle cell disease

    The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

    Testing rilzabrutinib for safety and effectiveness in sickle-cell disease

    📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →

    This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).

    ✓ Study care freePhase 3Ages 10–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 7–21Learn more →
  • DepressionAnxiety

    Creatine for Depressed Male and Female Methamphetamine Users

    Testing whether creatine helps depression and anxiety in people who use meth

    📍 Montana State University College of Nursing (Missoula campus) · Missoula, MT · Get directions →

    * Assess the antidepressant/anxiolytic effect of creatine in male and female methamphetamine users * Assess creatine's effect on methamphetamine use * Assess the safety of creatine in male methamphetamine users with depression

    ✓ Study care freePhase 3Ages 18–59Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 1+Learn more →
  • Anxiety

    📍 Servicios Especializados En Ensayos Clinicos, Seec, Sc · Mexico City, Mexico City · Get directions →

    To evaluate the superiority of cognitive behavioral therapy using the Aurora® digital medical device + conventional drug treatment, compared to conventional drug treatment alone, in reducing anxiety symptoms, as assessed by the change in the GAD-7 scale at 12 weeks of treatment, relative to baseline.

    ✓ Study care freePhase 3Ages 18–65Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 12–35Learn more →
  • DepressionAnxiety

    Open-Label Psilocybin Study in Transdiagnostic Population

    Testing psilocybin for safety and symptom changes in people with depression or anxiety

    📍 Connecticut Mental Health Center - Yale School of Medicine · New Haven, CT · Get directions →

    The primary objective of this study is to investigate the safety, feasibility, and tolerability of psilocybin treatment in individuals with functional impairment due to psychiatric symptoms. The secondary objective of this study is to determine whether individuals with functional impairments due to psychiatric symptoms will experience statistically significant symptom reduction and functional improvement from baseline symptom measurements (Visit 3) to 1-week (Visit 7), 4-weeks (Visit 8), and 6-weeks (Visit 9) post dosing.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • DepressionAnxiety

    Use of a Generative AI (Gen-AI) Chatbot for Anxiety and Depression Among Persons With Cannabis Use

    Testing a chatbot to support people using cannabis with anxiety or depression

    📍 46 Centerra Parkway, Suite 300, Office #333S · Lebanon, NH · Get directions →

    This study tests a chatbot called Therabot-CALM to help people who use cannabis with anxiety and/or low mood. Participants will do questionnaires, try the chatbot for 4 weeks, and join virtual interviews to give feedback on how usable and helpful it feels. The goal is to see if the chatbot is acceptable to users and can improve symptoms.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Anxiety

    The Impact of Focused Ultrasound Thalamotomy of the Anterior Nucleus for Focal-Onset Epilepsy on Anxiety

    Testing whether focused ultrasound thalamotomy can improve anxiety

    📍 The Ohio State University · Columbus, OH · Get directions →

    The purpose of this study is to evaluate the feasibility, safety, and effects on anxiety of high intensity focused ultrasound ablation (FUSA) in patients suffering from treatment-refractory focal epilepsy and anxiety. FUSA is a non-invasive neurosurgical procedure that uses ultrasound waves, sent directly through the scalp and skull, to precisely target small abnormal areas of the brain.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–24Learn more →
  • Sickle cell disease

    Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

    Testing a single pneumococcal vaccine dose in children with sickle cell disease

    📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →

    The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • DepressionAnxiety

    Enhancing Transdiagnostic Mechanisms of Cognitive Dyscontrol (R33)

    Testing a cognitive training program to see brain and thinking changes in people with depression or

    📍 UC San Diego · San Diego, CA · Get directions →

    The proposed project aims to test the cognitive and neural effects of a cognitive training in a sample of individuals seeking treatment for anxiety, depression, or traumatic stress symptoms. Participants will be randomly assigned to one of two groups.

    ✓ Study care freePhase 2Ages 21–55Learn more →
  • Sickle cell disease

    A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

    Testing long-term treatment with a new medicine for sickle cell or thalassaemia

    📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →

    Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.

    ✓ Study care freePhase 3Ages 2+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 4–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 13–40Learn more →
  • Anxiety

    Cannabidiol-Assisted Learning for Managing Generalized Anxiety Disorder

    Testing cannabidiol added to talk therapy for adults with generalized anxiety

    📍 Wayne State University School of Medicine, Tolan Park Medical Building · Detroit, MI · Get directions →

    This randomized, double-blind, placebo-controlled clinical trial investigates the use of Food and Drug Administration (FDA)-approved cannabidiol (EPIDIOLEX®) as an adjunct to cognitive behavioral therapy (CBT) in adults with generalized anxiety disorder (GAD). The study aims to evaluate whether cannabidiol-assisted CBT enhances emotion regulation via dorsomedial prefrontal cortex (dmPFC) activation and improves anxiety symptom outcomes compared to CBT with placebo.

    ✓ Study care freePhase 3Ages 18–45Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    📍 University Of Alabama · Birmingham, AL · +13 more US sites · Get directions →

    A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

    ✓ Study care freePhase 3Ages 12–100Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 12–55Learn more →
  • 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.

    ✓ Study care freePhase 1Learn more →
  • Sickle cell disease

    A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

    Testing how well etavopivat lowers painful sickle cell crises and organ damage

    📍 Uni of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →

    This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo.

    ✓ Study care freePhase 3Ages 12+Learn more →
  • DepressionAnxiety

    Affect of Duavive on Mood & Anxiety Symptoms

    Testing how Duavive affects mood and anxiety symptoms in early menopause women

    📍 St Joseph's Healthcare · Hamilton, Ontario · Get directions →

    This study evaluates the impact of conjugated estrogens/ bazedoxifene (CE/ BZA) on the mood (depression and anxiety) in peri- and early menopausal women.

    ✓ Study care freePhase 1Ages 45–60WomenLearn more →
  • 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.

    ✓ Study care freePhase 1Ages 8–17Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 12–45Learn more →
  • Sickle cell disease

    Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

    Testing a new transplant plan using immune-suppressing treatment before a blood stem cell transplant

    📍 City of Hope Medical Center · Duarte, CA · Get directions →

    This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

    ✓ Study care freePhase 1Ages 1–30Learn more →
  • Sickle cell disease

    T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

    Testing whether using mismatched donor stem cells works for severe sickle cell disease and other

    📍 Children's Hospital of Pittsburgh of UPMC · Pittsburgh, PA · Get directions →

    The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease.

    ✓ Study care freePhase 1Ages 5–40Learn more →
  • Sickle cell disease

    A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease

    Testing how oral medicines may help people with high-risk sickle cell disease

    📍 University of Illinois at Chicago College of Medicine · Chicago, IL · Get directions →

    A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

    ✓ Study care freePhase 1Ages 18+Learn more →
  • DepressionAnxiety

    PSilocybin for psYCHological and Existential Distress in PALliative Care (PSYCHED-PAL)

    Testing daily low-dose psilocybin to treat anxiety and depression in advanced illness patients

    📍 The Ottawa Hospital · Ottawa, Ontario · Get directions →

    The goal of this multi-centre phase I/II open-label, single-arm study is to determine the safety, feasibility, therapeutic dose, and preliminary efficacy of psilocybin microdosing to treat psychological distress among patients with advanced illness. Forty patients will receive psilocybin drug product (1-3mg per day, Mon-Fri) for 4 weeks to be administered via oral capsules by the participant.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

    Testing gene correction in a person’s own blood-forming stem cells for severe sickle cell disease

    📍 Children's Hospital Los Angeles · Los Angeles, CA · +5 more US sites · Get directions →

    This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

    ✓ Study care freePhase 1Ages 12–50Learn more →
  • Sickle cell disease

    PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD

    Using PET scans to find blood vessel blockage markers in sickle cell disease

    📍 UPMC · Pittsburgh, PA · Get directions →

    The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET).

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    A Phase 2/3 Study in Adult and Adolescent Participants With SCD

    Testing safety and how well a treatment works for sickle cell in teens and adults

    📍 Smilow Cancer Hospital · New Haven, CT · +23 more US sites · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

    ✓ Study care freePhase 2Ages 12+Learn more →
  • DepressionAnxiety

    Combination Therapy for Treatment of Sleep Disturbance in Patients With Advanced Cancer

    Testing combined sleep treatments to improve sleep and mood in people with advanced cancer

    📍 MD Anderson Cancer Center · Houston, TX · Get directions →

    To learn if Cognitive Behavior Therapy (called CBT), combined with either Bright Light Therapy (called BLT), methylphenidate, and/or melatonin, can help improve sleep and other related symptoms such as fatigue, anxiety, and depression in cancer patients. This is an investigational study.

    ✓ Study care freePhase 2Ages 18+Learn more →
  • Sickle cell disease

    Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

    Testing the safety and effectiveness of a one-time gene-edited cell treatment for severe sickle cell

    📍 New York Presbyterian Hospital - Morgan Stanley Children's Hospital · New York, NY · +2 more US sites · Get directions →

    This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

    ✓ Study care freePhase 3Ages 12–35Learn more →