What this study is
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).
What's being tested
- CTX112
Intervention · BIOLOGICAL
CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Study details
- 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
- Sponsor
- CRISPR Therapeutics AG
- Conditions
- Warm Autoimmune Hemolytic Anemia (WAIHA) · ITP - Immune Thrombocytopenia · Warm Autoimmune Hemolytic Anemia · Immune Thrombocytopenic Purpura
- Enrolling
- 60 people
- Starts
- 2026-07-17
- Est. completion
- 2033-12
- Design
- Open-label, single group, Phase 1, Phase 2
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 18 Years and overSex ALLHealthy volunteers Not acceptedYou may qualify if
- 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)
You can't join if
- 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.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (12)
12 openBoston Medical Center
Boston, Massachusetts, United States
- Fabio Petrocca, MD · Principal investigator
RecruitingMayo Clinic in Rochester
Rochester, Minnesota, United States
- Jithma Abeykoon, MD · Principal investigator
RecruitingUniversity of Nebraska Medical Center
Omaha, Nebraska, United States
- Marcel Devetten, MD · Principal investigator
Recruiting
9 sites outside the US
Universitaetsklinikum Koeln
Cologne, Germany
- Ruth Flümann, MD · Principal investigator
RecruitingUniversitaetsklinikum Carl Gustav Carus Dresden
Dresden, Germany
- Karolin Trautmann-Grill, MD · Principal investigator
RecruitingMedizinische Hochschule Hannover (MHH)
Hanover, Germany
- Christian Schultze-Florey, MD · Principal investigator
RecruitingUCT University Medical Center Mainz
Mainz, Germany
- Eva Wagner-Drouet, MD · Principal investigator
RecruitingHospital Clinic Barcelona
Barcelona, Spain
- Anna Gaya, MD · Principal investigator
RecruitingHospital Universitario Reina Sofia
Córdoba, Spain
- Clara Aparicio Perez, MD · Principal investigator
RecruitingGregorio Marañón General University Hospital
Madrid, Spain
- Cristina Pascual Izquierdo, MD · Principal investigator
RecruitingHospital Universitario La Paz
Madrid, Spain
- Ana Mendoza Martínez, MD · Principal investigator
RecruitingHospital Universitario Virgen del Rocio
Seville, Spain
- Maria Eva Mingot Castellano, MD · Principal investigator
Recruiting
Study team
Sponsor
CRISPR Therapeutics AG
Study ID CRSP-AID-501
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.