8 completed, 6 recruiting, 3 active not recruiting, 3 unknown. 5 Not applicable, 4 Phase 2, 2 Phase 1, 1 Early Phase 1.
Follow alpha-Thalassemia: new trials by email Literature Review of alpha-Thalassemia trials Research Report on alpha-Thalassemia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Genetic Profile of Alpha Thalassemia Children at Sohag University Hospital . Alpha-Thalassemia | Unknown | Sohag University | 50 | 2024-08-01 | 2024-09-19 | |
| FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases Alpha-Thalassemia, Beta-Thalassemia | Unknown | xCures | 1000 | 2024-06-10 | 2024-11-14 | |
| Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease RM-004 - Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies | Early Phase 1 | Recruiting | The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army | 5 | 2023-10-08 | 2024-06-03 |
| A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia Luspatercept, Placebo - Anemia | Phase 2 | Active not recruiting | Bristol-Myers Squibb | 189 | 2022-12-09 | 2026-09-10 |
| Study of the Role of Genetic Modifiers in Hemoglobinopathies GWAS - Sickle Cell Disease, Thalassemia, Beta | Recruiting | Cyprus Institute of Neurology and Genetics | 30000 | 2022-10-01 | 2024-03-20 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease Etavopivat tablets - Sickle Cell Disease, Thalassemia | Phase 2 | Completed | Forma Therapeutics, Inc. | 53 | 2022-03-28 | 2025-12-16 |
| Uncovering the Etiologies of Non-immune Hydrops Fetalis Whole genome sequencing - Non-Immune Hydrops Fetalis | Not applicable | Enrolling by invitation | University of California, San Francisco | 500 | 2022-03-01 | 2026-04-17 |
| RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) Data collection from EHR. - Sickle Cell Disease, Thalassemia | Recruiting | Hospital Universitari Vall d'Hebron Research Institute | 32564 | 2021-11-30 | 2024-01-19 | |
| In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome in-utero HSCT - Haemoglobin Barts Hydrops | Not applicable | Recruiting | Chinese University of Hong Kong | 10 | 2021-10-01 | 2023-04-12 |
| Multicentric Prospective Study to Screen Inborn Errors of Metabolism in Non-immune Hydrops (NIH) Fetalis by Massively Parallel Sequencing NON-IMMUNE HYDROPS FETALIS diagnosis - Non-Immune Hydrops Fetalis | Not applicable | Completed | Hospices Civils de Lyon | 71 | 2021-04-12 | 2024-11-08 |
| ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 Non applicable, is a patient registry - Hemoglobinopathies, Sickle Cell Disease | Completed | Hospital Universitari Vall d'Hebron Research Institute | 684 | 2020-04-15 | 2025-02-18 | |
| Hydrops: Diagnosing & Redefining Outcomes With Precision Study Exome sequencing - Hydrops Fetalis, Birth Defect | Not applicable | Active not recruiting | University of California, San Francisco | 500 | 2018-10-11 | 2026-04-17 |
| Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal Demise Fetal Magnetocardiogram and Neonatal Electrocardiogram, Substudy only: Maternal/Infant Pharmacogenomic assessment postnatally - High Risk Pregnancy, Congenital Heart Disease | Recruiting | Medical College of Wisconsin | 30 | 2018-07-01 | 2026-03-04 | |
| In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) in utero hematopoietic stem cell transplantation - Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia | Phase 1 | Terminated | University of California, San Francisco | 6 | 2017-10-05 | 2025-01-27 |
| Prospective Observational Cohort Study of Fetal Atrial Flutter & Supraventricular Tachycardia Prospective observational cohorts - Atrial Flutter, Tachycardia, Supraventricular | Completed | Edgar Jaeggi | 330 | 2017-06-08 | 2025-05-31 | |
| Screening for Alpha Thalassemia in Healthy Volunteers Alpha Thalassemia | Active not recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 367 | 2017-04-17 | 2026-04-24 | |
| International Registry of Patients With Alpha Thalassemia Alpha-Thalassemia, Alpha Thalassemia Major | Recruiting | University of California, San Francisco | 500 | 2017-01-01 | 2026-09-02 | |
| Precision Medical Research of Non-immune Fetal Hydrops (NIFH)-From Prenatal Diagnosis to Intrauterine Treatment intrauterine intervention - Fetal Hydrops | Not applicable | Unknown | Shanghai First Maternity and Infant Hospital | 144 | 2016-12-01 | 2016-12-12 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies Enriched Hematopoetic Stem Cell Infusion - Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies | Phase 1 / Phase 2 | Withdrawn | Talaris Therapeutics Inc. | 2011-05-01 | 2023-03-03 |
Showing 20 of 24. Sign in to see all of them.
Hydrops Fetalis (9) · beta-Thalassemia (8) · Anemia, Sickle Cell (7) · Hemoglobinopathies (6) · Thalassemia (6) · Anemia (4) · Anemia, Diamond-Blackfan (2) · Anemia, Hemolytic (2) · Congenital Abnormalities (2) · Cystic Fibrosis (2) · Cystinosis (2) · Gaucher Disease (2) · Genetic Diseases, Inborn (2) · Glycogen Storage Disease Type II (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Hemic and Lymphatic Diseases (24) · Hematologic Diseases (23) · Genetic Diseases, Inborn (22) · Hemoglobinopathies (18) · Thalassemia (18) · Anemia, Hemolytic, Congenital (16) · Anemia, Hemolytic (14) · Anemia (13) · Pathological Conditions, Signs and Symptoms (13) · Immune System Diseases (11) · Female Urogenital Diseases and Pregnancy Complications (10) · Urogenital Diseases (10) · Edema (9) · Erythroblastosis, Fetal (9)
Deferasirox (1) · Exome (1) · luspatercept (1)
University of California, San Francisco (4) · Hospital Universitari Vall d'Hebron Research Institute (2) · Bristol-Myers Squibb (1) · Centre Hospitalier Universitaire de Liege (1) · Chinese University of Hong Kong (1) · Cyprus Institute of Neurology and Genetics (1) · Deborah Rund (1) · Edgar Jaeggi (1) · Forma Therapeutics, Inc. (1) · HaEmek Medical Center, Israel (1) · Hospices Civils de Lyon (1) · Medical College of Wisconsin (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · Novartis Pharmaceuticals (1) · Obstetrix Medical Group (1)
United States (13) · Italy (4) · Spain (4) · Greece (3) · Hong Kong (3) · Israel (3) · Malaysia (3) · United Kingdom (3) · Belgium (2) · Canada (2) · China (2) · Egypt (2) · France (2) · Lebanon (2) · Taiwan (2)
Petros Kountouris, PhD (2) · Amy P Ruhl, M.D. (1) · Deborah Rund, MD (1) · Edgar Jaeggi, MD, FRCPC (1) · Janette F Strasburger, MD (1) · Karrie Francois, MD (1) · Laurent Servais (1) · Mary Norton, MD (1)
PMID 23553596, PMID 25212456, PMID 27862048, PMID 33027564, PMID 36306387, PMID 37606495, PMID 39891377, PMID 41984996
K12HD001262 (1), R01HD107190 (1), R01HL143485 (1)