Hemoglobinopathies
📧 Sign up to get email alerts when something new happens for Hemoglobinopathies.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-07-21Clinical Trial Results: Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders [Phase 2]
Phase 2 trial evaluating a Reduced Intensity Conditioning regimen for patients with non-malignant disorders; results completed and posted in…
-
CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders [Phase 2]
Phase 2 clinical trial evaluating cord blood transplant in children and young adults with blood cancers and select non-malignant disorders; …
-
CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) [Phase 3]
A Phase 3 clinical trial update reports ongoing evaluation of CTX001 for safety and efficacy in pediatric patients with transfusion-dependen…
-
CLINICAL_TRIALS research · 2026-01-07Clinical Trial Update: Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders [Phase 1, Phase 2]
A Phase 1/2 clinical trial at Washington University evaluates reduced-intensity conditioning and familial HLA-mismatched bone marrow transpl…
-
CLINICAL_TRIALS trial_result · 2025-12-17Clinical Trial Update: A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia [Phase 2, Phase 3]
A Phase 2/3 safety and efficacy study of CTX001 in transfusion-dependent beta-thalassemia has completed.
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2025-12-08Clinical Trial Update: Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases [Phase 2]
A Phase 2 trial comparing treosulfan-based conditioning to busulfan-based conditioning in pediatric patients with non-malignant diseases has…
-
CLINICAL_TRIALS trial_result · 2025-10-30Clinical Trial Update: Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion [NA]
Clinical trial update from Baylor College of Medicine reports results for haploidentical hematopoietic cell transplantation using TCR alpha/…
-
CLINICAL_TRIALS research · 2025-09-18Clinical Trial Update: Prevalence and Hematological Characteristics of Hemoglobinopathies
This completed clinical trial reports on the prevalence and hematological characteristics of hemoglobinopathies.
-
CLINICAL_TRIALS trial_result · 2025-08-11Clinical Trial Update: A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease [Phase 2, Phase 3]
A safety and efficacy study of CTX001 in subjects with severe Sickle Cell Disease has completed Phase 2 and Phase 3 trials.
-
CLINICAL_TRIALS research · 2025-07-24Clinical Trial Update: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) [NA]
ClinicalTrials.gov entry for a second or greater allogeneic hematopoietic stem cell transplant using reduced intensity conditioning, current…
-
CLINICAL_TRIALS research · 2024-07-25Clinical Trial Update: Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders [Phase 2]
Phase 2 clinical trial update describing a reduced-intensity conditioning regimen for patients with non-malignant hematologic disorders, inc…
-
CLINICAL_TRIALS trial_result · 2024-03-25Clinical Trial Update: Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases [Phase 1]
Phase 1 safety study of gene-modified donor T cell infusion after stem cell transplantation for non-malignant diseases has been terminated; …
-
CLINICAL_TRIALS trial_result · 2024-01-10Clinical Trial Update: Non-Myeloablative Conditioning and Bone Marrow Transplantation [NA]
Clinical trial update involving non-myeloablative conditioning and bone marrow transplantation for sickle cell disease and related hemoglobi…
-
CLINICAL_TRIALS trial_result · 2023-09-26Clinical Trial Update: Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study [Phase 1, Phase 2]
Long-term follow-up update from BP-004 study (Phase 1/Phase 2) by Bellicum Pharmaceuticals; study status TERMINATED with results posted in 2…
-
CLINICAL_TRIALS trial_result · 2023-01-04Clinical Trial Update: Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant [Phase 1, Phase 2]
A phase 1/2 safety study evaluating gene-modified donor T-cells after TCRαβ+ depleted stem cell transplant was terminated; results were post…
-
CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria Research
The study collected blood from individuals with hemoglobinopathies and erythrocyte variants to support laboratory malaria research; the tria…
-
CLINICAL_TRIALS trial_result · 2017-05-11Clinical Trial Update: Hepatitis C Virus Infection in Patients With Hemoglobinopathies
A completed clinical trial update regarding Hepatitis C infection in patients with hemoglobinopathies.
-
CLINICAL_TRIALS trial_result · 2017-02-23Clinical Trial Update: Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload [Phase 4]
Novartis-sponsored Phase 4 trial assessing heart and liver iron load by MRI in patients with transfusion-related iron overload; the study st…
-
CLINICAL_TRIALS trial_result · 2013-03-12Clinical Trial Update: Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies [Phase 2]
Phase 2 clinical trial update on allogeneic stem cell transplantation after chemotherapy in patients with hemoglobinopathies. The study, inc…
-
CLINICAL_TRIALS trial_result · 2009-04-23Clinical Trial Update: Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies [Phase 4]
Phase 4 clinical trial update examining interferon and ribavirin treatment in patients with hemoglobinopathies.