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RecruitingNCT07549698Updated Sep 22, 2026

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

A Phase 1/2 interventional study of CTX112 in Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia, sponsored by CRISPR Therapeutics AG. Recruiting at 10 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-22.

Sponsored by CRISPR Therapeutics AG · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
60
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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).

Read the 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.

02

Conditions studied

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

Keywords

  • CD19
  • CTX112
  • Zugocabtagene geleucel
  • zugo-cel
  • CAR-T
  • ITP
  • warm autoimmune hemolytic anemia
  • wAIHA
  • Immune Thrombocytopenic Purpura
  • Immune Thrombocytopenia
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
60 participants (estimated)

Study arms

  • Experimental
    CTX112 (zugo-cel)

    Administered by IV infusion following lymphodepleting chemotherapy

    Biological: CTX112

Interventions

  • BiologicalCTX112

    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

05

What researchers measure

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.

    Time frame: From CTX112 infusion up to 28 days post infusion.

Secondary outcomes

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

    Change from baseline in B cell levels

    Time frame: From CTX112 infusion up to 60 months post-infusion

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

    Levels of CTX112 in blood over time.

    Time frame: From CTX112 infusion up to 60 months post-infusion.

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

    For participants with ITP, platelet response. For participants with wAIHA, hemoglobin response.

    Time frame: From CTX112 infusion up to 60 months post-infusion

06

Study locations

10 of 10 sites recruiting
  • Mayo Clinic in Rochester
    Rochester, Minnesota 55905, United States
    • Jithma Abeykoon · Principal investigator
    Recruiting
  • University of Nebraska Medical Center
    Omaha, Nebraska 68198, United States
    • Marcel Devetten · Principal investigator
    Recruiting
  • Universitaetsklinikum Koeln
    Cologne, Germany
    • Ruth Flümann · Principal investigator
    Recruiting
  • Medizinische Hochschule Hannover
    Hannover, Germany
    • Christian Schultze-Florey · Principal investigator
    Recruiting
  • Medizinische Hochschule Hannover (MHH)
    Hanover, 30625, Germany
    • Christian Schultze-Florey · Principal investigator
    Recruiting
  • UCT University Medical Center Mainz
    Mainz, 55131, Germany
    • Eva Wagner-Drouet · Principal investigator
    Recruiting
  • Hospital Universitario Reina Sofia
    Córdoba, 14004, Spain
    • Clara Aparicio Perez · Principal investigator
    Recruiting
  • Gregorio Marañón General University Hospital
    Madrid, Spain
    • Cristina Pascual Izquierdo · Principal investigator
    Recruiting
  • Hospital Universitario La Paz
    Madrid, Spain
    • Ana Mendoza Martínez · Principal investigator
    Recruiting
  • Hospital Universitario Virgen del Rocio
    Seville, Spain
    • Maria Eva Mingot Castellano · Principal investigator
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07549698
Lead sponsor
CRISPR Therapeutics AG
Responsible party
Sponsor
First posted
Apr 24, 2026
Start date
Jul 17, 2026
Primary completion
Dec 2033 (estimated)
Completion
Dec 2033 (estimated)
Last update
Sep 22, 2026

Study contacts

Clinical Trials
Contact
medicalaffairs@crisprtx.com
877-214-4634

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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