Clinical trials: Neonatal Anemia, open to volunteers
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
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.
- Conditions
- Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders
- Trial name
- FAM BMT (NCT03128996)
- Testing
- RIC regimen, GVHD prophylaxis regimen
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell…
- Trial name
- TRANSPLANTORN2 (NCT06351462)
- Testing
- Spermogram, Hospital Anxiety and Depression Scale (HADS), SF36 Quality of life questionnaire
- Volunteers
- 220 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Sickle Cell Disease
Sickle Cell Clinical Research and Intervention Program
Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation.
- Trial name
- NCT02098863
- Where
- 6 sites in United States
- Volunteers
- 10,000 people
- Sponsor
- St. Jude Children's Research Hospital
Dengue (Virus); Fever, Sandfly, Sickle Cell Disease
Molecular and Cellular Basis of Severe Forms of Dengue in Sickle Cell Patients
Dengue virus (DENV) belongs to the genus of Flavivirus transmitted by the mosquito Aedes aegypti and is responsible for an infectious disease associated with different forms and severities such as dengue hemorrhagic fever or…
- Trial name
- DREPADENGUE (NCT07000747)
- Where
- Guadeloupe
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Sickle Cell Disease
CS-206 in Patients With Sickle Cell Disease
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
- Trial name
- NCT06565026
- Testing
- CS-206
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Gaucher Disease Type 1
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate…
- Trial name
- GD (NCT06818838)
- Testing
- LY-M001
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Lingyi Biotech Co., Ltd.
Chronic Myeloproliferative Disorders, Leukemia
Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer
RATIONALE: Collecting and storing samples of tissue, blood, and body fluid from patients with cancer to study in the laboratory may help the study of cancer in the future.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder
- Trial name
- NCT01137643
- Testing
- biologic sample preservation procedure, cytology specimen collection procedure
- Where
- United States
- Volunteers
- 15,000 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Sickle Cell Disease
Feasibility and Acceptability of Amani
Adults with sickle cell disease (SCD) experience severe pain, emotional distress, and social isolation that diminish their quality of life.
- Trial name
- Amani (NCT07648043)
- Testing
- Amani
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Massachusetts General Hospital
Bone Marrow Failure Syndrome
French National Registry of Bone Marrow Failures
This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France (multicenter registry), from diagnosis and…
- Trial name
- RIME (NCT04781790)
- Testing
- Bone Marrow Failure
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD).
- Trial name
- HIBISCUS KIDS (NCT06198712)
- Testing
- Etavopivat
- Where
- 18 sites in Canada, France and 5 more
- Volunteers
- 95 people
- Sponsor
- Forma Therapeutics, Inc.
Beta-Thalassemia
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s).
- Trial name
- NCT03351829
- Testing
- Gene-modified autologous hematopoeitic stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines.
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of
- Trial name
- NCT06092346
- Where
- United States
- Volunteers
- 999 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Vaso-Occlusive Crises, Sickle Cell Disease
Evaluation of the Efficacy of Iloprost in the Management of Vaso-occlusive Crises in Adult Patients With Sickle Cell Disease
Sickle cell disease is a severe monogenic genetic disorder caused by an autosomal recessive mutation of the β-globin gene, leading to production of abnormal hemoglobin (HbS).
- Trial name
- PROSTASICKLE (NCT07498309)
- Testing
- Iloprost
- Where
- France
- Volunteers
- 144 people
- Sponsor
- University Hospital, Rouen
Sickle Cell Disease
MULTIsite Feasibility of MUSIc Therapy to Address Quality Of Life in Sickle Cell Disease
This is a multi-site, multi-visit feasibility RCT of music therapy (MT) among adolescent and adult patients (aged 14 and older) with sickle cell disease (SCD).
- Trial name
- MULTI-MUSIQOLS (NCT06853158)
- Testing
- Music therapy, Health education
- Where
- 2 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, Irvine
Anemia, Sickle Cell, Healthy Volunteers
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
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…
- Trial name
- NCT06481306
- Testing
- BMS-986470, Famotidine, Pantoprazole
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 224 people, healthy volunteers welcome
- Sponsor
- Bristol-Myers Squibb
Thalassemia Majors (Beta-Thalassemia Major)
Risk Factors Affecting Growth in Thalassemic Children at AUCH
Thalassemia major is a hereditary hemoglobinopathy characterized by ineffective erythropoiesis and severe anemia, necessitating lifelong blood transfusions(1,2).
- Trial name
- NCT07509996
- Testing
- Non-interventional assessment of growth and risk factors in thalassemic children
- Volunteers
- 85 people
- Sponsor
- Assiut University
Breath-holding Spell
Breath-holding Spells and Its Management Study
The goal of this prospective population-based study is to evaluate the new disease description and management guidelines for breath-holding spells in children (Hellström Schmidt et al, Acta Paediatrica 2024) below the age of 5…
- Trial name
- BAM (NCT06628271)
- Testing
- Guidelines
- Where
- 12 sites in Sweden
- Volunteers
- 120 people
- Sponsor
- Region Skane
Beta-thalassemia, Glioma
Plasticizer Exposure and Its Consequences on Health
Plasticizers are chemicals commonly found in many everyday items, from food packaging to medical equipment. Although they are pervasive in our daily lives, researchers still don't have a clear picture of their long-term effects…
- Conditions
- Beta-thalassemia, Glioma, Healthy Controls, Transfusion-dependent Beta-Thalassemia
- Trial name
- PEACH (NCT06512298)
- Where
- Austria
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Medical University of Vienna
Sickle Cell Disease, Chronic Pain
Predictors of Pain in Sickle Cell Disease
Sickle cell disease is a painful inherited disorder that affects approximately 100,000 people in the United States, and more than half of these individuals develop chronic or persistent pain that is often severe and disabling.
- Trial name
- NCT06139510
- Testing
- Quantitative sensory testing
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Duke University