← Browse by Condition
Medical Condition

genetic diseases inborn

Total Trials
1
Recruiting Now
1
Trial Phases
Phase 3

ClinicalMetric tracks all active clinical trials for genetic diseases inborn sourced from ClinicalTrials.gov. Each listing includes the NCT identifier, current recruitment status, phase, primary endpoints, sponsor, estimated enrollment, and participating countries — updated daily as new studies open and status changes.

  • Recruiting trials currently enrolling participants, with eligibility criteria and age requirements
  • Phase 1–4 studies from early safety trials to post-marketing surveillance
  • NIH, pharmaceutical sponsor, and academic medical center studies worldwide
  • AI-powered plain-language summary available for every trial listing
Trial Phases
Phase 3
1
Top Sponsors
Vertex Pharmaceuticals Incorporated 1 trial

Recruiting Clinical Trials

NCT05477563 Phase 3
Recruiting

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

Enrollment
26 pts
Location
United States, Germa...
Sponsor
Vertex Pharmaceuticals Incorpo...
View Trial →
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: April 2026  ·  Data Methodology