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

April 17, 2026 updated by: CRISPR Therapeutics AG

A Phase 1/2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease

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).

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

This trial will assess the safety and preliminary efficacy of CTX112 or Zugocabtagene geleucel (zugo-cel) in adults with relapsed or refractory hematologic autoimmune diseases (AID), including primary ITP and primary wAIHA. In these B-cell-mediated conditions, autoantibodies target platelets (ITP) or red blood cells (wAIHA), causing severe thrombocytopenia or anemia. Although several treatments exist, some patients relapse or remain refractory, resulting in significant morbidity, mortality, and reduced quality of life. This underscores the need for new therapeutic options.

B-cell-directed therapies are central to current management, and emerging data show promising activity of anti-CD19 CAR T cell therapies in AID. CTX112 (zugo-cel) is an allogeneic, CD19-targeted CAR T cell product derived from healthy donors and genetically modified ex vivo using CRISPR-Cas9. Similar to autologous CD19 CAR T therapies, CTX112 (zugo-cel) may induce clinical responses after a single treatment and offers the advantages of off-the-shelf availability.

Up to 60 participants may be enrolled. Study duration will be up to 5 years.

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

  1. Prior treatment with anti-CD19 therapy or any gene therapy or genetically modified cell therapy.
  2. Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
  3. Severe active or history of central nervous (CNS) involvement.
  4. 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.
  5. History of primary or secondary immunodeficiency.
  6. Presence or history of certain bacterial, viral or fungal infection
  7. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).
  8. Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome.
  9. History or current diagnosis that requires uninterrupted, ongoing anticoagulation.
  10. Pregnant or lactating.
  11. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CTX112 (zugo-cel)
Administered by IV infusion following lymphodepleting chemotherapy
Biological: CTX112
CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Other Names:
  • Zugocabtagene geleucel
  • zugo-cel

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA.
Time Frame: From CTX112 infusion up to 28 days post infusion.
Incidence of dose-limiting toxicities.
From CTX112 infusion up to 28 days post infusion.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To assess the pharmacodynamics response to CTX112 in adult participants with ITP or wAIHA.
Time Frame: From CTX112 infusion up to 60 months post-infusion
Change from baseline in B cell levels
From CTX112 infusion up to 60 months post-infusion
To assess the pharmacokinetics (PK) of CTX112 in adult participants with ITP or wAIHA.
Time Frame: From CTX112 infusion up to 60 months post-infusion.
Levels of CTX112 in blood over time.
From CTX112 infusion up to 60 months post-infusion.
To assess the preliminary efficacy of CTX112 in adult participants with ITP or wAIHA.
Time Frame: From CTX112 infusion up to 60 months post-infusion
For participants with ITP, platelet response. For participants with wAIHA, hemoglobin response.
From CTX112 infusion up to 60 months post-infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CRISPR Therapeutics AG

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2026

Primary Completion (Estimated)

December 1, 2033

Study Completion (Estimated)

December 1, 2033

Study Registration Dates

First Submitted

April 8, 2026

First Submitted That Met QC Criteria

April 17, 2026

First Posted (Actual)

April 24, 2026

Study Record Updates

Last Update Posted (Actual)

April 24, 2026

Last Update Submitted That Met QC Criteria

April 17, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CRSP-AID-501

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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