Clinical trials: Hemoglobinopathies
Thalassemia
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels.
- Trial name
- NCT00661726
- Testing
- Decitabine (USAN, INN)
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Carelon Research
Sickle Cell Disease
A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease.
- Trial name
- NCT02314689
- Testing
- Intravenous (IV) citrulline
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Suvankar Majumdar
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
Methadone in Pediatric and Adult Sickle Cell Patients
To determine the pharmacokinetics of methadone in children and adults with SCD who are experiencing a painful episode.
- Trial name
- MSCD (NCT00761085)
- Testing
- Morphine, Methadone
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Washington University School of Medicine
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
Sickle Cell Disease
Study of Vitamin D in Children With Sickle Cell Disease
This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels…
- Trial name
- NCT01276587
- Testing
- Vitamin D3
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Gary M Brittenham, MD
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0…
- Trial name
- NCT02906202
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Stem Cell Transplantation, Bone Marrow Transplantation
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked…
- Conditions
- Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation
- Trial name
- NCT02512679
- Testing
- Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
Sickle Cell Disease
Open-Label Extension of Voxelotor
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.
- Trial name
- NCT04188509
- Testing
- Voxelotor
- Where
- 20 sites in United States, Egypt and 3 more
- Volunteers
- 162 people
- Sponsor
- Pfizer
Sickle Cell Disease, Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia
Evaluation of the AMICUS RBCx System in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease.
- Trial name
- NCT02372877
- Testing
- Amicus Red Cell Exchange in SCD patients
- Where
- 6 sites in United States
- Volunteers
- 83 people
- Sponsor
- Fenwal, Inc.
Sickle Cell Disease
Realizing Effectiveness Across Continents With Hydroxyurea (REACH)
REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for for pediatric patients with sickle cell anemia (SCA).
- Trial name
- REACH (NCT01966731)
- Testing
- Hydroxyurea
- Where
- 4 sites in Angola, Democratic Republic of the Congo and 2 more
- Volunteers
- 635 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati