Exagamglogene autotemcel clinical trials
Also written CTX001.
How each study uses exagamglogene autotemcel is sorted by a rule, right about nine times in ten. Readers can ask us to read every study one by one.Ask us to read each study
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Approvals
The first approval found in each region, with its record
- United States
- 8 Dec 2023 · Casgevy · BLA 125787 (FDA, Purple Book)
- European Union
- 9 Feb 2024 · Casgevy · EMEA/H/C/005763 (EMA)
From FDA's Drugs@FDA and Purple Book (through openFDA) and the European Medicines Agency, files of Sept 2026 and 27 Oct 2026. National approvals in Europe and other countries are not included. How this is worked out
How studies write it
Studies that test it under each name
Studies by the year they started
8 studies that test it; most in 2022 (3); 1 more planned to start later
| Year | Studies |
|---|---|
| 2018 | 2 |
| 2021 | 1 |
| 2022 | 3 |
| 2026 | 2 |
By phase
A phase 1/2 study counts in both.
What it is tested for
Each study’s main condition, where its own words name it, so a study counts once.
Who runs them
Industry 9 · Other 0
Vertex Pharmaceuticals Incorporated (7) · Cajal Therapeutics Inc. (2)
Often tested with
Tested in the same studies.
Coming up
From the study records. Dates are the sponsors’ own estimates and often move.
Reaching primary completion within a year · 4, 3 in phase 3
Studies that test exagamglogene autotemcel, phase 3 first.
- Jun 2027Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)Phase 3 · Vertex Pharmaceuticals Incorporated
- Jun 2027Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseasePhase 3 · Vertex Pharmaceuticals Incorporated
- Nov 2027Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)Phase 3 · Vertex Pharmaceuticals Incorporated
- Oct 2027Study of CTX001 in Adults With Anemia Due to Non-Dialysis-Dependent Chronic Kidney DiseasePhase 1 · Cajal Therapeutics Inc.
Studies
Open first, then newest update. How each study uses exagamglogene autotemcel is read from its arms, and is right about nine times in ten. Ask us to read each study. These are the studies that list it as an intervention; a search of ClinicalTrials.gov also finds studies that mention it only in a description, such as earlier treatment.
Testing it · 9
- Study of CTX001 in Adults With Anemia Due to Non-Dialysis-Dependent Chronic Kidney DiseaseNot yet recruitingPhase 1Cajal Therapeutics Inc.NCT07901933Written as CTX001
- A Long-term Follow-up Study in Participants Who Received CTX001Enrolling by invitationPhase 3Vertex Pharmaceuticals IncorporatedNCT04208529Written as CTX001
- A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.RecruitingEarly Phase 1Cajal Therapeutics Inc.NCT07577817Written as CTX001 With Iron
- Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseaseRecruitingPhase 3Vertex Pharmaceuticals IncorporatedNCT05477563Written as CTX001
- A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell DiseaseCompletedPhase 2/3Vertex Pharmaceuticals IncorporatedNCT03745287Written as Exa-cel
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)Active, not recruitingPhase 3Vertex Pharmaceuticals IncorporatedNCT05329649Written as CTX001
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)Active, not recruitingPhase 3Vertex Pharmaceuticals IncorporatedNCT05356195Written as CTX001
- Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC GenotypeWithdrawnPhase 3Vertex Pharmaceuticals IncorporatedNCT05951205Written as Exa-cel
- A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-ThalassemiaCompletedPhase 2/3Vertex Pharmaceuticals IncorporatedNCT03655678Written as CTX001
Codes
What other systems call exagamglogene autotemcel · ClinicalTrials.gg INT002066
- MeSH (NLM) C000729927exagamglogene autotemcelConfirmed ClinicalTrials.gov, NLM
- UNII (FDA) S53L777GM8EXAGAMGLOGENE AUTOTEMCELConfirmed FDA, NLM
- NCI Thesaurus C169042Exagamglogene AutotemcelOne source FDA
| System | Code | Name there | Checked by |
|---|---|---|---|
| MeSH (NLM) | C000729927 | exagamglogene autotemcel | Confirmed ClinicalTrials.gov, NLM |
| UNII (FDA) | S53L777GM8 | EXAGAMGLOGENE AUTOTEMCEL | Confirmed FDA, NLM |
| NCI Thesaurus | C169042 | Exagamglogene Autotemcel | One source FDA |
“Confirmed” means two independent publishers link the code to this drug; “one source”, one does and its name agrees.