Warm Autoimmune Hemolytic Anemia (WAIHA)Phase 1/2NCT07549698
Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
A Phase 1/2 study testing CTX112 in people with Warm Autoimmune Hemolytic Anemia (WAIHA) and ITP - Immune Thrombocytopenia, run by CRISPR Therapeutics AG at 12 sites in 3 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia
- What is being tested?
- CTX112
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 12 sitesin 3 countries
- Study dates
- 17 Jul 2026to Dec 2033
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
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- 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.
Trial locations and contacts
12 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 3 countries
Spain5 sites, 5 recruiting
- Recruiting
Hospital Clinic Barcelona
Barcelona
Anna Gaya, MD
- Recruiting
Hospital Universitario Reina Sofia
Córdoba, 14004
Clara Aparicio Perez, MD
- Recruiting
Gregorio Marañón General University Hospital
Madrid
Cristina Pascual Izquierdo, MD
- Recruiting
Hospital Universitario La Paz
Madrid
Ana Mendoza Martínez, MD
- Recruiting
Hospital Universitario Virgen del Rocio
Seville
Maria Eva Mingot Castellano, MD
Germany4 sites, 4 recruiting
- Recruiting
Universitaetsklinikum Koeln
Cologne
Ruth Flümann, MD
- Recruiting
Universitaetsklinikum Carl Gustav Carus Dresden
Dresden
Karolin Trautmann-Grill, MD
- Recruiting
Medizinische Hochschule Hannover (MHH)
Hanover, 30625
Christian Schultze-Florey, MD
- Recruiting
UCT University Medical Center Mainz
Mainz, 55131
Eva Wagner-Drouet, MD
United States3 sites, 3 recruiting
- Recruiting
Boston Medical Center
Boston, Massachusetts, 02118
Fabio Petrocca, MD
- Recruiting
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Marcel Devetten, MD
- Recruiting
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Jithma Abeykoon, MD
About this study
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
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).
Read the 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.
- Conditions
- Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia, Immune Thrombocytopenic Purpura
- Being tested
- CTX112
- Keywords
- CD19, CTX112, Zugocabtagene geleucel, zugo-cel, CAR-T, ITP, warm autoimmune hemolytic anemia, wAIHA, Immune Thrombocytopenic Purpura, Immune Thrombocytopenia
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTX112Biological
Also called Zugocabtagene geleucel, zugo-cel
CTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
| Study group | Role | What they receive |
|---|---|---|
| CTX112 (zugo-cel) | Experimental | Administered by IV infusion following lymphodepleting chemotherapy Receives: Biological: CTX112 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety of CTX112 in adult participants with refractory hematologic autoimmune diseases, including ITP or wAIHA.
Incidence of dose-limiting toxicities.
Measured: From CTX112 infusion up to 28 days post infusion.
Secondary outcomes (3)
To assess the pharmacodynamics response to CTX112 in adult participants with ITP or wAIHA.
Change from baseline in B cell levels
Measured: From CTX112 infusion up to 60 months post-infusion
To assess the pharmacokinetics (PK) of CTX112 in adult participants with ITP or wAIHA.
Levels of CTX112 in blood over time.
Measured: From CTX112 infusion up to 60 months post-infusion.
To assess the preliminary efficacy of CTX112 in adult participants with ITP or wAIHA.
For participants with ITP, platelet response. For participants with wAIHA, hemoglobin response.
Measured: From CTX112 infusion up to 60 months post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2026
- Study start17 Jul 2026
- Primary completionDec 2033 (estimated)
- Study completionDec 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Warm Autoimmune Hemolytic Anemia (WAIHA)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CRISPR Therapeutics AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07549698
- Sponsor's protocol ID
- CRSP-AID-501
- Status last verified
- Oct 2026
- First submitted
- 8 Apr 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07549698
- Acronym / sponsor ID
- CRSP-AID-501
- Conditions
- Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia, Immune Thrombocytopenic Purpura
- Keywords
- CD19, CTX112, Zugocabtagene geleucel, zugo-cel, CAR-T, ITP, warm autoimmune hemolytic anemia, wAIHA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- CRISPR Therapeutics AGIndustry (pharma, biotech, devices)
- Study start
- 17 Jul 2026 (actual)
- Primary completion
- Dec 2033 (estimated)
- Study completion
- Dec 2033 (estimated)
- First posted
- 24 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CTX112 (zugo-cel) | Experimental | Biological: CTX112Administered by IV infusion following lymphodepleting chemotherapy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CTX112Also: Zugocabtagene geleucel, zugo-celCTX112 (zugo-cel): CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components | Biological | CTX112 (zugo-cel) |
Primary outcome
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.
Incidence of dose-limiting toxicities.
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CTX112 in people with Warm Autoimmune Hemolytic Anemia (WAIHA) and ITP - Immune Thrombocytopenia, run by CRISPR Therapeutics AG at 12 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Spain, Germany, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials (877-214-4634, medicalaffairs@crisprtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2026.