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ClinicalTrials.gov 8 recruiting now official registry

Hemoglobinopathies clinical trials

8 of 18 indexed Hemoglobinopathies trials are currently recruiting.

18 US clinical trials · 8 currently recruiting

Hemoglobinopathies is the subject of 18 registered US clinical trials on ClinicalTrials.gov, 8 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 10 in Phase 1-2 (earlier-stage). The most active sponsor is Vertex Pharmaceuticals Incorporated, running 3 of these trials.

The research picture

Hemoglobinopathies has 18 registered US clinical trials, 8 of them open to new participants right now, about 44% of the total.

8
recruiting participants now
44%
of trials open to enrollment
3
in Phase 3–4 (later-stage)
3
top sponsor: Vertex Pharmaceuticals Incorporated
Phase 16Phase 24Phase 33

Active & Recent Trials

18 total, page 1 of 1

RECRUITING 30,000 participants

Study of the Role of Genetic Modifiers in Hemoglobinopathies

Cyprus Institute of Neurology and Genetics

NCT05799118

RECRUITING Phase 1 220 participants

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

Washington University School of Medicine

NCT00920972

RECRUITING Phase 2 62 participants

Allo HSCT for High Risk Hemoglobinopathies

Masonic Cancer Center, University of Minnesota

NCT06872333

RECRUITING 50 participants

Discarded Bone Marrow for Hematology Research

St. Jude Children's Research Hospital

NCT04671212

RECRUITING Phase 2 31 participants

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Memorial Sloan Kettering Cancer Center

NCT04644016

RECRUITING NA 30 participants

Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

Masonic Cancer Center, University of Minnesota

NCT01666080

RECRUITING Phase 1 29 participants

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Washington University School of Medicine

NCT03128996

RECRUITING Phase 3 26 participants

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Vertex Pharmaceuticals Incorporated

NCT05477563

ACTIVE NOT RECRUITING Phase 2 75 participants

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

Children's Hospital of Philadelphia

NCT01050855

ACTIVE NOT RECRUITING Phase 1 45 participants

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

Editas Medicine

NCT04853576

ACTIVE NOT RECRUITING Phase 3 16 participants

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Vertex Pharmaceuticals Incorporated

NCT05356195

ACTIVE NOT RECRUITING Phase 3 13 participants

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Vertex Pharmaceuticals Incorporated

NCT05329649

ACTIVE NOT RECRUITING Phase 1 9 participants

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Editas Medicine

NCT05444894

ACTIVE NOT RECRUITING Phase 1 3 participants

A Blood Stem Cell Transplant for Sickle Cell Disease

City of Hope Medical Center

NCT03249831

ACTIVE NOT RECRUITING NA 3 participants

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

Baylor College of Medicine

NCT05236764

COMPLETED 25 participants

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

University of California, San Francisco

NCT03609827

COMPLETED Phase 1 22 participants

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Duke University

NCT00744692

COMPLETED Phase 2 2 participants

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

Dana-Farber Cancer Institute

NCT00153985

Phase Distribution

PhaseTrial count
Phase 1 6
Phase 2 4
Phase 3 3

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Hemoglobinopathies Trial Landscape

18 indexed Hemoglobinopathies studies · 8 recruiting (44%).

Phase mix: 3 late (III/IV) vs 10 early (I/II).

Top sponsor: Vertex Pharmaceuticals Incorporated (3 trials), 9 others listed. 18 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Hemoglobinopathies?

PlainTrial tracks 18 US clinical trials for Hemoglobinopathies, of which 8 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Hemoglobinopathies?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.