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13 studies
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- Parkinson's disease
A Study of Buntanetap in Participants With PD
Testing the long-term safety of buntanetap in people with Parkinson’s
📍 University of Alabama at Birmingham · Birmingham, AL · +26 more US sites · Get directions →
This study will examine the long-term safety of buntanetap in participants with PD. This will be a 36-month open-label safety study.
- Parkinson's disease
A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease
Testing a Parkinson’s surgery approach to see if it is safe and works
📍 Banner Sun Health Research Institute · Sun City, AZ · +32 more US sites · Get directions →
Study BRT-DA01-301 is a Phase 3 multicenter, randomized, sham surgery-controlled, double-blind study to assess efficacy and safety of bemdaneprocel in approximately 102 adults with Parkinson's Disease (PD).
- 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).
- Parkinson's disease
18F-DOPA II - PET Imaging Optimization
Testing an imaging scan to study Parkinson’s disease using PET
📍 WC Mackenzie Health Science Centre / University of Alberta Hospital · Edmonton, Alberta · Get directions →
A single centre non-randomized, non-blinded phase III prospective cohort study of 18F-DOPA PET/CT imaging in specific patient populations: 1. Pediatric patients (less than 18 years old) with congenital hyperinsulinism.
- 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.
- 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.
- Sickle cell disease
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
Testing a drug infusion versus placebo to treat frequent pain crises in 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.
- Parkinson's disease
A Study to Evaluate the Efficacy and Safety of Intravenous (IV) Prasinezumab in Participants With Early-Stage Parkinson's Disease
Testing an IV drug in people with early-stage Parkinson’s disease
📍 University of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of prasinezumab compared with placebo in participants with early-stage Parkinson's disease (PD) on stable symptomatic monotherapy with levodopa.
- 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.
- Parkinson's disease
A Digital Therapeutic Platform for Swallowing and Drooling Problems in Parkinson's
Testing a wearable sensor to track swallowing and drooling problems in people with Parkinson’s
📍 Northwestern University · Evanston, IL · Get directions →
The purpose of this study is the development and early-stage validation of a wearable sensor for dysphagia in patients with PD.
- 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.
- 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.
- Parkinson's disease
A Study to Determine if BHV-8000 is Effective, Safe and Tolerable as a Treatment for Adults Living With Early Parkinson's Disease
Testing BHV-8000 for safety and effectiveness in adults with early Parkinson's disease
📍 Site-049 · Birmingham, AL · +14 more US sites · Get directions →
A study to determine if BHV-8000 is efficacious, safe and tolerable in adults diagnosed with early Parkinson's disease.