ClinicalTrials.gov record NCT07549698
Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This single-arm, open-label Phase 1/2 trial evaluates escalating doses of CTX112 (zugo-cel) in adults with relapsed/refractory primary ITP or primary wAIHA. Participants receive lymphodepleting chemotherapy followed by a single IV infusion of CTX112 and are followed for safety and blood responses for up to 5 years. Up to 60 participants may enroll. The study's main focus is safety and early evidence of effect on platelets or hemoglobin.
What it is studying. The study is testing the safety of CTX112 and its early effects on blood and immune cells. Key scientific measurements include dose-limiting toxicities, changes in B cell levels, the amount of CTX112 detectable in blood over time, and disease-specific responses (platelet counts for ITP; hemoglobin for wAIHA).
What the researchers hope to learn. To evaluate the safety of CTX112 by measuring dose-limiting toxicities; to assess pharmacodynamics (change in B cell levels) and pharmacokinetics (levels of CTX112 in blood); and to gather preliminary evidence of disease response (platelet response in ITP; hemoglobin response in wAIHA) over up to 60 months.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- CTX112
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will receive lymphodepleting chemotherapy followed by an intravenous infusion of CTX112 (zugo-cel). They will have close safety monitoring after infusion (including assessment for dose-limiting toxicities) and scheduled clinic visits and blood tests to measure drug levels and B cell responses. Follow-up and assessments continue long term—up to about 60 months (study duration up to 5 years). Participants must complete consent and follow the study visit and contraception requirements.
How the study is structured. An open-label, single-group Phase 1/2 dose-escalation trial. Adults receive lymphodepleting chemotherapy then one IV infusion of CTX112 (an allogeneic, CRISPR‑engineered anti‑CD19 T-cell product). No placebo or randomization; participants are followed with blood tests and clinical visits for safety, pharmacodynamics, pharmacokinetics, and disease response for up to 60 months.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in Germany, Spain, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
Important dates
- Study start
- Jul 17, 2026
- Primary completion
- December 2033 (estimated)
- Study completion
- December 2033 (estimated)
- Target enrollment
- 60 participants (estimated)
- Last updated on registry
- Sep 22, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- CRISPR Therapeutics AG
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Clinical Trials 📞 877-214-4634 ✉ medicalaffairs@crisprtx.com
Participating sites (10)
- Mayo Clinic in RochesterRecruiting
Rochester, Minnesota, 55905 · United States
- Jithma Abeykoon
- University of Nebraska Medical CenterRecruiting
Omaha, Nebraska, 68198 · United States
- Marcel Devetten
- Universitaetsklinikum KoelnRecruiting
Cologne · Germany
- Ruth Flümann
- Medizinische Hochschule HannoverRecruiting
Hannover · Germany
- Christian Schultze-Florey
- Medizinische Hochschule Hannover (MHH)Recruiting
Hanover, 30625 · Germany
- Christian Schultze-Florey
- UCT University Medical Center MainzRecruiting
Mainz, 55131 · Germany
- Eva Wagner-Drouet
- Hospital Universitario Reina SofiaRecruiting
Córdoba, 14004 · Spain
- Clara Aparicio Perez
- Gregorio Marañón General University HospitalRecruiting
Madrid · Spain
- Cristina Pascual Izquierdo
- Hospital Universitario La PazRecruiting
Madrid · Spain
- Ana Mendoza Martínez
- Hospital Universitario Virgen del RocioRecruiting
Seville · Spain
- Maria Eva Mingot Castellano
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- ascending dose
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- relapsed/refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- CAR T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- relapse
- A condition that improved with treatment and has since returned or worsened.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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