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📍 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.

📍 Northern Arizona Healthcare · Flagstaff, AZ · +34 more US sites · Get directions →

This goal of this study is to compare three medications used for migraine preventive treatment. This study will compare atogepant, a newer migraine preventive medication, with two older preventive medications, topiramate and propranolol.

📍 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.

📍 Rehabilitation & Neurological Services /ID# 248517 · Huntsville, AL · +39 more US sites · Get directions →

A migraine is a moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms.

📍 The Belinga Clinic · Fort Smith, AR · +39 more US sites · Get directions →

The purpose of this study is to compare the efficacy and safety of rimegepant to placebo as a preventative treatment for migraine in children and adolescents ≥ 6 to \<18 years with episodic migraine.

📍 TrialSphere Corp, Merz Investigational Site #0010513 · Chandler, AZ · +39 more US sites · Get directions →

In this clinical trial, participants with episodic migraine will receive injections with Xeomin or Placebo into muscles of the head and neck. The purpose is to measure the change in monthly migraine days with Xeomin injections compared to Placebo injections.

📍 Mayo Clinic Hospital · Phoenix, AZ · +31 more US sites · Get directions →

The purpose of this study is to evaluate the efficacy and safety of rimegepant when administered during the peri-menstrual period (PMP) for intermittent prevention of migraine in women who experience menstrual migraine attacks.

📍 TrialSphere Corp, Merz Investigational Site #0010513 · Chandler, AZ · +39 more US sites · Get directions →

In this clinical trial, participants with chronic migraine will receive injections with Xeomin or Placebo into muscles of the head and neck. The purpose is to measure the change in monthly migraine days with Xeomin injections compared to Placebo injections.

📍 Rehabilitation and Neurological Services /ID# 270782 · Huntsville, AL · +30 more US sites · Get directions →

Migraine is a disease that most often causes moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms.

📍 Yale-New Haven Children's Hospital - PIN · New Haven, CT · +21 more US sites · Get directions →

To find out if eptinezumab is better than placebo (normal saline solution) in lowering the number of days with migraine in young people ages 12 to 17 with chronic migraine.

📍 Xenoscience Inc · Phoenix, AZ · +26 more US sites · Get directions →

The purpose of this study is to learn about the effect of a study medicine called rimegepant in adolescents who have frequent migraine attacks. Rimegepant is a tablet that dissolves when you put it on or under your tongue.

📍 Arizona Liver Health - Chandler · Chandler, AZ · +30 more US sites · Get directions →

Rivus Pharmaceuticals. Inc. is sponsoring this research study to assess the safety and tolerability of HU6 as a possible treatment for patients diagnosed with metabolic dysfunction-associated steatohepatitis (MASH). The study will also asse

📍 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).

📍 Central Research Associates /ID# 260161 · Birmingham, AL · +39 more US sites · Get directions →

A migraine is a moderate to severe headache typically on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms.

📍 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.

📍 Arizona Liver Health · Chandler, AZ · +39 more US sites · Get directions →

The primary objective of this trial is to evaluate the dose-dependent and comparative effects of ECC4703 (low and high dose), ECC0509 (low and high dose), and their combination on hepatic fat reduction as assessed by change in magnetic resonance imaging proton density fat fraction (MRI-PDFF) at Week 12.

📍 Yale University School of Medicine · New Haven, CT · +19 more US sites · Get directions →

The main goal of the study is to assess the long-term safety of eptinezumab on children and adolescents ages 6 to 17 with chronic or episodic migraine.

📍 Ki Health Partners LLC DBA New England Institute for Clinical Research · Stamford, CT · +14 more US sites · Get directions →

The main goal of this trial is to learn whether eptinezumab helps reduce the number of days with episodic migraine in pediatric participants.

📍 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.

📍 Upsher-Smith Clinical Trial Site #5 · Birmingham, AL · +14 more US sites · Get directions →

A Phase 4 study to evaluate Qudexy XR for the prevention of migraine in children 6 to 11 years of age.

📍 Arizona Liver Health · Chandler, AZ · +39 more US sites · Get directions →

This study is researching an investigational drug, ALN-HSD called "study drug". This study is focused on participants who are known to have Metabolic dysfunction-Associated SteatoHepatitis (MASH).

📍 Central Research Associates, LLC (CRA) dba Flourish Research · Birmingham, AL · +39 more US sites · Get directions →

This is a double-blind, randomized, multicenter, outpatient evaluation of the efficacy, safety, and tolerability of elismetrep, as compared with placebo, in the acute treatment of migraine.

📍 University of California San Diego · La Jolla, CA · +15 more US sites · Get directions →

A Phase 2 double-blind, randomized, placebo-controlled study to evaluate resmetirom in 2 cohorts of subjects with moderate to advanced fibrosis, consistent with stage F2 and F3 fibrosis, who have undergone liver transplant. Cohort 1 will consist of patients who have undergone liver transplant for MASH cirrhosis who developed recurrent MASH.

📍 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.

📍 Central Research Associates · Birmingham, AL · +39 more US sites · Get directions →

A migraine is a headache with severe throbbing pain or a pulsating sensation, usually on one side of the head. It is often accompanied by feeling or being sick and a sensitivity to bright lights and sound.

📍 Flourish - Birmingham · Birmingham, AL · +15 more US sites · Get directions →

The purpose of this Phase 2 study is to evaluate the safety and efficacy of SC MEDI0618 compared to placebo in participants with episodic migraine.

📍 GSK Investigational Site · Chandler, AZ · +29 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, preliminary efficacy, and pharmacokinetics (PK) of efimosfermin in participants with metabolic dysfunction associated steatohepatitis (MASH) and compensated cirrhosis consistent with stage F4 fibrosis.

📍 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.

📍 CA Medical Clinic for Headache · Los Angeles, CA · +12 more US sites · Get directions →

This study will evaluate the efficacy and safety of EVO756 against placebo in adults with migraine.

📍 Arkansas Children's Hospital · Little Rock, AR · +12 more US sites · Get directions →

The purpose of the study is to learn about safety and how the body processes the study medicine called Zavegepant (PF-07930207) in children with a history of migraine. This study helps understand how the medicine is changed and removed from the body after taking it.

📍 GSK Investigational Site · Arcadia, CA · +39 more US sites · Get directions →

This study will evaluate the safety and tolerability of Efimosfermin Alfa for participants with known or suspected MASH with fibrosis consistent with stage F2 or F3.

📍 GSK Investigational Site · Arcadia, CA · +39 more US sites · Get directions →

The purpose of this study is to assess the safety and efficacy of efimosfermin alfa in the resolution of steatohepatitis and improvement of liver-related clinical outcome compared to placebo in individuals with MASH and biopsy-confirmed F2- or F3-stage fibrosis.

📍 Perseverance Research Center, LLC · Scottsdale, AZ · +38 more US sites · Get directions →

The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of migraine in children and adolescents (≥ 6 to \< 18 years of age).

📍 Akero Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due to NASH/MASH.

📍 Rehabilitation & Neurological Services /ID# 229969 · Huntsville, AL · +39 more US sites · Get directions →

Migraine is a common neurological disorder typically characterized by attacks of throbbing, moderate to severe headache, often associated with nausea, vomiting, and sensitivity to light and sound. Migraine is extremely common and disabling in children.

📍 Achieve Clinical Research, LLC d/b/a Accel Research Sites-Neurology and Neurodiagnostic of Alabama · Birmingham, AL · +39 more US sites · Get directions →

The purpose of this study is to test the safety and efficacy of BHV-3000 versus placebo in the acute treatment of moderate or severe migraine in children and adolescents.

📍 89bio Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →

The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.

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

This study is open to adults who are at least 18 years old living with obesity and have: * a confirmed liver disease called non-alcoholic steatohepatitis (NASH)/metabolic associated steatohepatitis (MASH) and * moderate or advanced liver fibrosis People with a history of acute or chronic liver diseases other than MASH or chronic alcohol intake cannot take part in this study. The purpose of this study is to find out whether a medicine called survodutide helps people with MASH and moderate or advanced liver fibrosis improve their liver function.

📍 89bio Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →

The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).

📍 University Of Alabama · Birmingham, AL · +6 more US sites · Get directions →

This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.

📍 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.

📍 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).

📍 The Institute for Liver Health II DBA Arizona Clinical Trials · Peoria, AZ · +39 more US sites · Get directions →

This study is open to adults who are at least 18 years old and have: * A confirmed liver disease called non-alcoholic steatohepatitis (NASH) or * A confirmed liver disease called metabolic-associated steatohepatitis (MASH) * BMI of 27 kg/m2 or more or * 25 kg/m2 or more if the participant is Asian. People with a history of other chronic liver diseases or high alcohol intake cannot take part in this study.

📍 Akero Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with non-cirrhotic NASH/MASH and fibrosis stage 2 or 3 (F2 or F3). The study will enroll subjects in two cohorts for a total samples size of 1650 subjects.

📍 Children's National Health System · Washington D.C., DC · +4 more US sites · Get directions →

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

📍 Hope Clinical Research · Canoga Park, CA · +6 more US sites · Get directions →

Acute treatments for migraine may not provide sufficient pain relief after an initial dose, and a second dose of a given medication may be needed to fully abort an attack. International Headache Society (IHS) global practice recommendations for the Acute Treatment of Migraine suggest a second dose of the same medication within the recommended dose limit in people with headache relapse after successful initial treatment of a migraine attack.

📍 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?

📍 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.

📍 Banner Health Center · Phoenix, AZ · +3 more US sites · Get directions →

Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health concern, is commonly associated with obesity, diabetes, and dyslipidemia. MASLD is currently the most common cause of chronic liver disease affecting about 80% of people with obesity, ranging from simple fat deposits in the liver to Metabolic Dysfunction-Associated Steatohepatitis (MASH), cellular injury, advanced fibrosis, cirrhosis, or hepatocellular carcinoma.

📍 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.

📍 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.

📍 University of California Los Angeles · Los Angeles, CA · +3 more US sites · Get directions →

This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS.

📍 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.

📍 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.

📍 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.

📍 GSK Investigational Site · Rialto, CA · +2 more US sites · Get directions →

This study is designed to study the pharmacokinetic (PK) and safety profiles of a single dose of efimosfermin alfa in participants with varying degrees of Hepatic Impairment (HI) (assessed by Child-Pugh score) due to steatotic liver disease, with and without significant alcohol consumption.

📍 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.

📍 Sunwise Clinical Research, Llc · Walnut Creek, CA · +2 more US sites · Get directions →

This study is testing an investigational inhaled migraine medication to see how well it works, how safe it is, and how well people tolerate it. Adults with migraine will receive both the study medication (ASY202) and a placebo (inactive treatment) at different times during the study.

📍 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.

📍 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.