Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease

NCT07549698 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: CRSP-AID-501
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a single-arm, open-label, multicenter, ascending dose Phase 1/2 trial evaluating the safety and preliminary efficacy of CTX112 or Zugocabtagene geleucel (zugo-cel) in adult participants with relapsed/refractory primary Immune Thrombocytopenia (ITP) and relapsed/refractory primary Warm Autoimmune Hemolytic Anemia (wAIHA).

Conditions

Warm Autoimmune Hemolytic Anemia; Immune Thrombocytopenic Purpura; Warm Autoimmune Hemolytic Anemia (WAIHA); ITP - Immune Thrombocytopenia

Keywords

CD19; CTX112; Zugocabtagene geleucel; zugo-cel; CAR-T; ITP; warm autoimmune hemolytic anemia; wAIHA; Immune Thrombocytopenic Purpura; Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• To evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA.

  Incidence of dose-limiting toxicities.

  From CTX112 infusion up to 28 days post infusion.

Secondary Outcomes (3)

• To assess the pharmacodynamics response to CTX112 in adult participants with ITP or wAIHA.

  From CTX112 infusion up to 60 months post-infusion

• To assess the pharmacokinetics (PK) of CTX112 in adult participants with ITP or wAIHA.

  From CTX112 infusion up to 60 months post-infusion.

• To assess the preliminary efficacy of CTX112 in adult participants with ITP or wAIHA.

  From CTX112 infusion up to 60 months post-infusion

Study Arms (1)

CTX112 (zugo-cel)

EXPERIMENTAL

Administered by IV infusion following lymphodepleting chemotherapy

Biological: CTX112

Interventions

CTX112 BIOLOGICAL

CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components

Also known as: Zugocabtagene geleucel, zugo-cel

CTX112 (zugo-cel)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 years.
• Participants must voluntarily sign a written informed consent and be willing and able to comply with all trial requirements.
• Adequate hematologic, renal, liver, cardiac and pulmonary function.
• Participants must agree to use acceptable methods of contraception.
• Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other trial procedures.
• Diagnosis of relapsed/refractory primary Immune Thrombocytopenic Purpura (ITP) or Warm Autoimmune Hemolytic Anemia (WAIHA)

You may not qualify if:

• Prior treatment with anti-CD19 therapy or any gene therapy or genetically modified cell therapy.
• Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
• Severe active or history of central nervous (CNS) involvement.
• Presence of other active autoimmune disease or other conditions that are likely to pose increased safety risks and/or confound disease assessments, or pose significant risk to those receiving CAR T cell therapy.
• History of primary or secondary immunodeficiency.
• Presence or history of certain bacterial, viral or fungal infection
• Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).
• Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome.
• History or current diagnosis that requires uninterrupted, ongoing anticoagulation.
• Pregnant or lactating.
• Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol.

Sponsors & Collaborators

• CRISPR Therapeutics AG: lead

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Central Study Contacts

Clinical Trials

CONTACT

877-214-4634; medicalaffairs@crisprtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 8, 2026
• First Posted: April 24, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): December 1, 2033
• Study Completion (Estimated): December 1, 2033
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share