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Hemoglobinopathies: early-phase trial registry
8 of 18 indexed Hemoglobinopathies trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #953 of 28,707 conditions by registered trial count.
18 US clinical trials · 8 currently recruiting · #953 of 28,707 conditions by registered trial count
Hemoglobinopathies: 18 US clinical trials tracked, 8 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.
Enrollment posture brief
Registry condition hemoglobinopathies
OPEN 8 | RECR 44% | LATE 3 | EARLY 10 | RANK #953 | SPON 3
Volume-matched condition peers
Nearest open-share peer: Acute Ischemic Stroke (44% recruiting · 27 trials)
Hemoglobinopathies lists 8 open slots (44% of 18 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Hemoglobinopathies on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Hemoglobinopathies is recruiting below its size-band peers.
8 of 18 indexed trials (44.4%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Hemoglobinopathies research.
Vertex Pharmaceuticals Incorporated accounts for 3 of 18 trials (16.7%). 13 distinct sponsors appear on at least one study for this condition label.
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Most Hemoglobinopathies trials plan to enrol fewer than 100 people.
Median target enrolment is 29 among the 18 trials that report a genuine target (sentinel values excluded). 16 of those (88.9%) plan for fewer than 100 participants.
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Hemoglobinopathies research is mostly interventional.
15 interventional and 3 observational studies make up this index (83.3% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
18 total, page 1 of 1
Study of the Role of Genetic Modifiers in Hemoglobinopathies
Cyprus Institute of Neurology and Genetics
NCT05799118
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
Washington University School of Medicine
NCT00920972
Allo HSCT for High Risk Hemoglobinopathies
Masonic Cancer Center, University of Minnesota
NCT06872333
Discarded Bone Marrow for Hematology Research
St. Jude Children's Research Hospital
NCT04671212
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
Memorial Sloan Kettering Cancer Center
NCT04644016
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
Masonic Cancer Center, University of Minnesota
NCT01666080
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Washington University School of Medicine
NCT03128996
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
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
Children's Hospital of Philadelphia
NCT01050855
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Editas Medicine
NCT04853576
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Vertex Pharmaceuticals Incorporated
NCT05356195
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Vertex Pharmaceuticals Incorporated
NCT05329649
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
Editas Medicine
NCT05444894
A Blood Stem Cell Transplant for Sickle Cell Disease
City of Hope Medical Center
NCT03249831
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Baylor College of Medicine
NCT05236764
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
University of California, San Francisco
NCT03609827
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Duke University
NCT00744692
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
Dana-Farber Cancer Institute
NCT00153985
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 6 |
| Phase 2 | 4 |
| Phase 3 | 3 |
Top Sponsors
Named interventions
Most-linked intervention names on Hemoglobinopathies records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Hemoglobinopathies trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Hemoglobinopathies page
18 registered trials, 8 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 8 Hemoglobinopathies trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Vertex Pharmaceuticals Incorporated sponsors the most Hemoglobinopathies trials on record, review their full trial history before evaluating a specific study. See Vertex Pharmaceuticals Incorporated's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Hemoglobinopathies condition card totals 18 registered trials; most-linked intervention Fludarabine; top US site state Tennessee. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.