CTX001 (Ph 3) for Sickle Cell Disease | Vertex Pharmaceuticals Incorporated | BiopharmaWatch

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CTX001

Phase 3

Sickle Cell Disease | Monoclonal antibody | Hematology |Vertex Pharmaceuticals Incorporated|Last Updated: Jul 1, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC

Total Trials2

Total Enrollment76

FDA Designations

No designations recorded

Clinical trial landscape

CTX001 · 6 trials · 10 indications

Phase 3 4Phase 2 2

Indication

NCT05477563Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseaseBeta-Thalassemia

RECRUITING26 Analytics

NCT05356195Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)Beta-Thalassemia

ACTIVE NOT_RECRUITING16 Analytics

NCT05329649Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)Sickle Cell Disease

ACTIVE NOT_RECRUITING13 Analytics

NCT04208529A Long-term Follow-up Study in Participants Who Received CTX001Beta-Thalassemia

ENROLLING BY_INVITATION160 Analytics

PHASE3RECRUITING

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

Beta-Thalassemia

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PHASE3ACTIVE NOT_RECRUITING

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Beta-Thalassemia

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PHASE3ACTIVE NOT_RECRUITING

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sickle Cell Disease

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PHASE3ENROLLING BY_INVITATION

A Long-term Follow-up Study in Participants Who Received CTX001

Beta-Thalassemia

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Study Endpoints

Primary Endpoints

Fetal Hemoglobin (HbF) Concentration Over Time

Up to 12 Months After CTX001 Infusion

Total Hemoglobin (Hb) Concentration Over Time

Up to 12 Months After CTX001 Infusion

Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)

Up to 24 Months After CTX001 Infusion

Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12)

Up to 24 Months After CTX001 Infusion

New malignancies

Signing of informed consent up to 15 years post CTX001 infusion

New or worsening hematologic disorders

Signing of informed consent up to 15 years post CTX001 infusion

All-cause mortality

Signing of informed consent up to 15 years post CTX001 infusion

Serious adverse events (SAEs)

Signing of informed consent up to 15 years post CTX001 infusion

CTX001-related adverse events (AEs)

Signing of informed consent up to 15 years post CTX001 infusion

Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)

From 60 days after last RBC transfusion up to 2 years after CTX001 infusion

Proportion of subjects with engraftment (first day of three consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days)

Within 42 days after CTX001 infusion

Time to engraftment

From CTX001 infusion up to 2 years after CTX001 infusion

Frequency and severity of collected adverse events (AEs)

From screening to 2 years after CTX001 infusion

Incidence of transplant-related mortality (TRM) within 100 days after CTX001 infusion

Within 100 days after CTX001 infusion

Incidence of TRM within 1 year after CTX001 infusion

Within 1 year after CTX001 infusion

Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12)

From 60 days after last RBC transfusion up to 24 months post-CTX001 infusion]

Proportion of participants with engraftment (first day of 3 consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days)

Within 42 days after CTX001 infusion

Time to neutrophil and platelet engraftment

Days post-infusion to engraftment

Incidence of transplant-related mortality (TRM)

Baseline (pre-transfusion) to 100 days and 1 year post-CTX001 infusion

Secondary Endpoints

TDT and SCD: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

From Signing of Informed Consent up to 12 Months After CTX001 Infusion

TDT and SCD: Proportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days)

Within 42 Days After CTX001 Infusion

TDT and SCD: Time to Engraftment

Up to 12 Months After CTX001 Infusion

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Study Design & Arms

AllocationNA

MaskingNONE

ModelSINGLE_GROUP

PurposeTREATMENT

Treatment Arms

Arm	Type	Description
CTX001	EXPERIMENTAL	CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter.

Interventions

Name	Type	Description
CTX001	BIOLOGICAL	Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan

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Eligibility Criteria

Age Range12 Years to 35 Years

SexALL

Healthy VolunteersNo

Study Sites6

Key Inclusion Criteria: * Participants with TDT and SCD: * Eligible for autologous stem cell transplant as per investigator's judgment. * Participants with TDT: * Diagnosis of TDT as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hem...

Countries:United StatesGermanyItalySaudi ArabiaCanadaUnited KingdomBelgiumFrance

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Competitive Landscape -Sickle Cell Disease 27 trials

Company	Ticker	Trials	Lead Phase	Drugs
Novo Nordisk A/S Sponsored ADR Class B	NVO	5	PHASE3	Etavopivat Low dose
Novartis AG Sponsored ADR	NVS	4	PHASE3	Crizanlizumab
Sanofi SA Sponsored ADR	SNY	2	PHASE3	PCV21, 20vPCV
Vertex Pharmaceuticals Incorporated	VRTX	3	PHASE3	CTX001
Agios Pharmaceuticals, Inc.	AGIO	2	PHASE2	Mitapivat
Pfizer Inc.	PFE	1	PHASE2	Osivelotor
Bristol-Myers Squibb Company	BMY	1	PHASE1	BMS-986470, Famotidine, Pantoprazole
Fulcrum Therapeutics, Inc.	FULC	1	PHASE2	Pociredir
Beam Therapeutics, Inc.	BEAM	2	PHASE1	BEAM-101
Editas Medicine, Inc.	EDIT	2	PHASE1	EDIT-301
Disc Medicine, Inc.	IRON	1	PHASE1	DISC-3405
Illumina, Inc.	ILMN	1	-	Undisclosed

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Recent Changes (Last 90 Days)

MEDIUMJul 2, 2026NCT05329649primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05356195primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05329649primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05356195primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05329649primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05356195primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05329649primaryCompletionDate: changed

MEDIUMJul 2, 2026NCT05356195primaryCompletionDate: changed

LOWMay 26, 2026NCT05477563primaryCompletionDate: changed

LOWMay 26, 2026NCT04208529primaryCompletionDate: changed

LOWMay 26, 2026NCT05356195primaryCompletionDate: changed

LOWMay 26, 2026NCT05329649primaryCompletionDate: changed

LOWMay 24, 2026NCT05477563studyFirstPostDate: changed

LOWMay 24, 2026NCT04208529studyFirstPostDate: changed

LOWMay 24, 2026NCT05329649studyFirstPostDate: changed

LOWMay 24, 2026NCT05356195studyFirstPostDate: changed