Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Genetic Diseases, Inborn: later-stage trial registry
1 of 6 indexed Genetic Diseases, Inborn trials are currently recruiting, with more weight in Phase 3-4.
Corpus placement: #2,866 of 28,707 conditions by registered trial count.
6 US clinical trials · 1 currently recruiting · #2,866 of 28,707 conditions by registered trial count
Genetic Diseases, Inborn: 6 US clinical trials tracked, 1 recruiting.
Genetic Diseases, Inborn is the subject of 6 registered US clinical trials on ClinicalTrials.gov, 1 of them currently open to new participants. 4 are in Phase 3-4 (later-stage) and 1 in Phase 1-2 (earlier-stage). The most active sponsor is Vertex Pharmaceuticals Incorporated, running 2 of these trials.
Enrollment posture brief
Registry condition genetic-diseases-inborn
OPEN 1 | RECR 17% | LATE 4 | EARLY 1 | RANK #2866 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Acute Renal Failure (17% recruiting · 6 trials)
Genetic Diseases, Inborn lists 1 open slots (17% of 6 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 Genetic Diseases, Inborn 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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Genetic Diseases, Inborn is recruiting below its size-band peers.
1 of 6 indexed trials (16.7%) 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 Genetic Diseases, Inborn research.
Vertex Pharmaceuticals Incorporated accounts for 2 of 6 trials (33.3%). 5 distinct sponsors appear on at least one study for this condition label.
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Typical Genetic Diseases, Inborn enrolment sits around 106 participants.
Median target enrolment is 106 among the 6 trials that report a genuine target (sentinel values excluded). 3 of those (50%) plan for fewer than 100 participants.
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Genetic Diseases, Inborn research is mostly interventional.
6 interventional and 0 observational studies make up this index (100% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
6 total, page 1 of 1
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
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Capricor
NCT05126758
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Vertex Pharmaceuticals Incorporated
NCT05356195
NYCKidSeq: Incorporating Genomics Into Clinical Care of Diverse NYC Children
Icahn School of Medicine at Mount Sinai
NCT03738098
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
Kastle Therapeutics
NCT00694109
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Spark Therapeutics
NCT03734588
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 3 | 4 |
Top Sponsors
Named interventions
Most-linked intervention names on Genetic Diseases, Inborn records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Genetic Diseases, Inborn 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 Genetic Diseases, Inborn page
6 registered trials, 1 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 1 Genetic Diseases, Inborn trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Vertex Pharmaceuticals Incorporated sponsors the most Genetic Diseases, Inborn 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 Genetic Diseases, Inborn condition card totals 6 registered trials; most-linked intervention CTX001; top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.