CTX001
VERTEX PHARMACEUTICALS INC VRTX · Hemoglobinopathies; Beta-Thalassemia; Genetic Diseases, Inborn; Hematologic Diseases; Thalassemia
Key figures
Next catalyst
None scheduled
Only dates with a source are listed
Phase
Phase 3
Not recorded
Linked trials
5
From ClinicalTrials.gov
Latest FDA action
None on record
From Drugs@FDA
Recalls, shortages, patents and EU status at a glance
Key information
- Sponsor
- VERTEX PHARMACEUTICALS INC VRTX Source: Company record for VERTEX PHARMACEUTICALS INC (opens in a new tab)
- Indications
- Hemoglobinopathies; Beta-Thalassemia; Genetic Diseases, Inborn; Hematologic Diseases; Thalassemia Source: Conditions from ClinicalTrials.gov (opens in a new tab)
- Designations
- None recorded
- Next catalyst
- None scheduled
Development timeline
- Preclinical Completed
- Phase 1 Completed
- Phase 2 CompletedFirst trial start 14 Sep 20181 linked trial
- Phase 3 Current stageFirst trial start 14 Sep 20185 linked trials
- Filed Not yet
- Approved Not yet
Overview
- Mechanism
- Not recorded
- Route
- Not recorded
- Modality
- Not recorded
- Specialties
- Not recorded
Overview text is written by HealthTrade editors.
Key dates
No catalysts yet. Editors add each date together with its source.
FDA designations
No FDA designations are on record for CTX001. Editors add each one with its source.
Filed application
No filed application is on record for CTX001. Approvals are listed under FDA actions.
FDA advisory committees
No FDA advisory committee meetings are on record for CTX001.
Clinical trials
| Trial | Title | Phase | Status | Enrollment | Primary completion | Results posted |
|---|---|---|---|---|---|---|
| NCT05477563ClinicalTrials.gov record for NCT05477563 (opens in a new tab) | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | Phase 3 | Recruiting | 26 (estimated) | 9 Jun 2027 (estimated) | No |
| NCT05356195ClinicalTrials.gov record for NCT05356195 (opens in a new tab) | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | Phase 3 | Active, not recruiting | 16 (actual) | 14 Nov 2027 (estimated) | No |
Publications listing NCT05356195 (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT05329649ClinicalTrials.gov record for NCT05329649 (opens in a new tab) | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | Phase 3 | Active, not recruiting | 13 (actual) | 6 Jun 2027 (estimated) | No |
Publications listing NCT05329649 (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT04208529ClinicalTrials.gov record for NCT04208529 (opens in a new tab) | A Long-term Follow-up Study in Participants Who Received CTX001 | Phase 3 | Enrolling by invitation | 160 (estimated) | 30 Sep 2039 (estimated) | No |
Publications listing NCT04208529 (3), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT03655678ClinicalTrials.gov record for NCT03655678 (opens in a new tab) | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia | Phase 2/3 | Completed | 59 (actual) | 13 Nov 2025 (actual) | No |
Publications listing NCT03655678 (5), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.
Trial quality
About CT-VCALoading trial quality…
FDA actions
No FDA approvals on record for this drug.
FDA recalls load here.
FDA drug shortages load here.
Orange Book patents load here.
FAERS adverse event counts load here.
Papers and preprints load here.
Community discussion loads here.
EU status (EMA)
EMA's medicines data lists no human medicine with the same active substance as CTX001.
Source: European Medicines Agency (EMA), medicines data download.
Related news
No recent headlines mention this drug.
Sources
- ClinicalTrials.gov NCT04208529 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT05356195 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT05477563 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT05329649 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT03655678 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
Record updated .
Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.