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Recruiting NCT07549698

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

Phase I / Phase II Interventional Warm Autoimmune Hemolytic Anemia (WAIHA) ITP - Immune Thrombocytopenia Warm Autoimmune Hemolytic Anemia Immune Thrombocytopenic Purpura

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CTX112.
Who it may be relevant to
Registry conditions: Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia, Immune Thrombocytopenic Purpura. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Germany, Spain
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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).

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.

Interventions

  • 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

Primary outcome measures

  • 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.]
Secondary outcome measures (3)
  • 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]
  • 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.]
  • 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]

Eligibility criteria

Inclusion criteria

  • 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)

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Germany · 4 centers
  • Universitaetsklinikum Koeln — Cologne
  • Medizinische Hochschule Hannover — Hannover
  • Medizinische Hochschule Hannover (MHH) — Hanover
  • UCT University Medical Center Mainz — Mainz
Spain · 4 centers
  • Hospital Universitario Reina Sofia — Córdoba
  • Gregorio Marañón General University Hospital — Madrid
  • Hospital Universitario La Paz — Madrid
  • Hospital Universitario Virgen del Rocio — Seville
United States · 2 centers
  • Mayo Clinic in Rochester — Rochester
  • University of Nebraska Medical Center — Omaha

Identifiers

NCT: NCT07549698 · CRSP-AID-501

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗