- Conditions
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 4
- States / cities
- Charlotte, North Carolina • Philadelphia, Pennsylvania • Memphis, Tennessee + 1 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–7
of 7
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2039
- U.S. locations
- 9
- States / cities
- Palo Alto, California • Chicago, Illinois • New York, New York + 5 more
- Conditions
- Geographic Atrophy Secondary to Age-related Macular Degeneration
- Interventions
- CTx001
- Genetic
- Lead sponsor
- Complement Therapeutics
- Industry
- Eligibility
- 55 Years and older
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 4
- States / cities
- Carmel, Indiana • Reno, Nevada • Dallas, Texas + 1 more
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 3
- States / cities
- New York, New York • Charlotte, North Carolina • Nashville, Tennessee
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 6
- States / cities
- Palo Alto, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Interventions
- Exa-cel
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 63 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 9
- States / cities
- Palo Alto, California • Chicago, Illinois • New York, New York + 4 more
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee