Found 6,933 clinical trials
A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
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 …
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
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.
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
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. Participants will be treated …
Auto-antibody Dosage From Blood Spots for Diagnosis of Type 1 Diabetes and Celiace Disease
Early diagnosis of type 1 diabetes and celiac disease is very useful, allows early therapy and prevents deaths from the onset of diabetic ketoacidosis. This is a pilot study on screening of autoantibodies of type 1 diabetes and celiac disease in tuscany patients. The study aims to evaluate the concordance …
HEALEY ALS Platform Trial - Master Protocol
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Platform Trial to Assess the Efficacy of Multiple Drugs in Amyotrophic Lateral Sclerosis (ALS)
The objective of this phase III, placebo-controlled platform study is to investigate the efficacy of drugs for patients with ALS (Amyotrophic lateral sclerosis).
French-German Cohort Study to Determine Factors Associated With Weight Loss in Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease. Studies have shown the importance of weight loss at the time of diagnosis and during the progression of the disease. However, the pathophysiological mechanisms behind weight loss remain unknown. Identifying these mechanisms could make it possible to propose an effective therapeutic strategy …
Using the EHR to Advance Genomic Medicine Across a Diverse Health System
Given the expansion of indications for genetic testing and our understanding of conditions for which the results change medical management, it is imperative to consider novel ways to deliver care beyond the traditional genetic counseling visit, which are both amenable to large-scale implementation and sustainable. The investigators propose an entirely …
Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).
