NCT07549698 · CRISPR Therapeutics AG
Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
What this study is about
This is a single-treatment group$1, where both patients and doctors know the treatment given, conducted at multiple hospitals, ascending dose Phase 1/2 trial evaluating the safety and preliminary effectiveness 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).
View original scientific description
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).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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.
Where
- Rochester, Minnesota
- Omaha, Nebraska
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 15, 2026 · Source of record for eligibility and locations