Clinical trials: Anemia, Hemolytic
Sickle Cell Disease
Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
Background: Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood.
- Trial name
- NCT02225132
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Music Therapy in Sickle Cell Transition Study
The purpose of this study is to investigate the effects of the BEATS Music Therapy Program on the self-efficacy, trust, knowledge, and adherence of young adult patients with sickle cell disease during transition.
- Trial name
- NCT02466555
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Anemia, Sickle Cell Disease
Novel Use Of Hydroxyurea in an African Region With Malaria
Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in…
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM (NCT01976416)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 208 people
- Sponsor
- Indiana University
Sickle Cell Disease
Home-Based Intervention for Chronic Pain in Adults With Sickle Cell Disease
This project will evaluate AppliedVR's EaseVRx - a multi-modal, skills-based, 8-week, virtual reality, home intervention - in an exploratory randomized controlled trial for self-management of chronic pain among Black, young…
- Trial name
- NCT04906707
- Testing
- EaseVRx, Audio-only version of EaseVRx
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Emory University
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
- Trial name
- NCT04085601
- Testing
- APL-2
- Where
- 30 sites in Colombia, Hong Kong and 8 more
- Volunteers
- 53 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Sickle Cell Anemia
Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea
The primary goal of the Phase III TWiTCH trial is to compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for pediatric subjects with sickle cell anemia and abnormally high (≥200 cm/sec)…
- Trial name
- TWiTCH (NCT01425307)
- Testing
- Hydroxyurea
- Volunteers
- 159 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Non-transfusion Dependent Thalassemia
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload.
- Trial name
- THALASSA (NCT00873041)
- Testing
- deferasirox
- Where
- 21 sites in United States, Greece and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis Pharmaceuticals
Atypical Hemolytic Uremic Syndrome
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician.
- Trial name
- EVIDENCE (NCT02614898)
- Testing
- Eculizumab
- Where
- 24 sites in United States, Australia and 2 more
- Volunteers
- 67 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center
Sickle Cell Disease, Vaso-occlusive Pain Episode
Arginine Therapy for Sickle Cell Disease Pain
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores…
- Trial name
- NCT02536170
- Testing
- L-arginine, L-arginine Loading Dose
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Emory University
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Sickle Cell Disease
European Sickle Cell Disease Cohort - Hydroxyurea
In the context of the Risk Management Plan (RMP), as requested from Addmedica by the EMEA, to collect information about long-term safety of Siklos® (hydroxycarbamide) when used in patients with Sickle Cell Disease.
- Trial name
- ESCORT-HU (NCT02516579)
- Testing
- Siklos
- Where
- 60 sites in France, French Guiana and 5 more
- Volunteers
- 1,906 people
- Sponsor
- Theravia
Severe Thalassemia
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks…
- Trial name
- URTH (NCT01005576)
- Testing
- Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
- Where
- 18 sites in United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Using Patient-Centered Guidelines in a Technology Platform to Improve Health Care in Adults With Sickle Cell Disease
SCD is an inherited disorder of hemoglobin that affects over 100,000 Americans, most of whom live in low-resourced neighborhoods.
- Trial name
- NCT03629678
- Testing
- mobile health application
- Where
- 2 sites in United States
- Volunteers
- 67 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Warm Autoimmune Hemolytic Anemia
To Assess the Efficacy and Safety of RVT-1401 in the Treatment of Warm Autoimmune Hemolytic Anemia (ASCEND-WAIHA).
This is a Phase 2 non-randomized, open-label study to investigate the efficacy, safety and tolerability of RVT-1401 in patients with Warm Autoimmune Hemolytic Anemia.
- Trial name
- ASCEND-WAIHA (NCT04253236)
- Testing
- RVT-1401 680 mg/weekly, RVT-1401 340 mg/weekly
- Where
- 21 sites in United States, Israel and 4 more
- Volunteers
- 5 people
- Sponsor
- Immunovant Sciences GmbH
Warm Autoimmune Hemolytic Anemia (wAIHA)
Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),
- Trial name
- PATHWAY (NCT05073458)
- Testing
- parsaclisinib
- Where
- 56 sites in United States, Austria and 11 more
- Volunteers
- 13 people
- Sponsor
- Incyte Corporation
Sickle Cell Disease
Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization
This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU).
- Trial name
- NCT04080167
- Testing
- InCharge Health mobile application, HU Toolbox mobile application
- Where
- 7 sites in United States
- Volunteers
- 382 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Cold Agglutinin Disease
A Study to Assess the Efficacy and Safety of BIVV009 (Sutimlimab) in Participants With Primary Cold Agglutinin Disease Without A Recent History of Blood Transfusion
The purpose of Part A was to determine whether sutimlimab administration resulted in a greater than or equal to (>=)1.5 grams per deciliter (g/dL) increase in hemoglobin (Hgb) level and avoidance of transfusion in participants…
- Trial name
- Cadenza (NCT03347422)
- Testing
- sutimlimab (BIVV009)
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 42 people
- Sponsor
- Bioverativ, a Sanofi company