CTX112: CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
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).
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
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:
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.
Primary outcome measure(s)
To evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA. — From CTX112 infusion up to 28 days post infusion. Incidence of dose-limiting toxicities.
Trial sites (10)
Facility
City
Region
Status
Mayo Clinic in Rochester
Rochester
Minnesota
Recruiting
University of Nebraska Medical Center
Omaha
Nebraska
Recruiting
Universitaetsklinikum Koeln
Cologne
Germany
Recruiting
Medizinische Hochschule Hannover
Hannover
Germany
Not Yet Recruiting
Medizinische Hochschule Hannover (MHH)
Hanover
Germany
Not Yet Recruiting
UCT University Medical Center Mainz
Mainz
Germany
Not Yet Recruiting
Hospital Universitario Reina Sofia
Córdoba
Spain
Recruiting
Gregorio Marañón General University Hospital
Madrid
Spain
Recruiting
Hospital Universitario La Paz
Madrid
Spain
Not Yet Recruiting
Hospital Universitario Virgen del Rocio
Seville
Spain
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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