ITP - Immune ThrombocytopeniaPhase 1/2NCT06352281
Efficacy and Safety of CAR-T Cells Therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP)
A Phase 1/2 study testing CAR-T cells in people with ITP - Immune Thrombocytopenia, run by 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China at 1 site in China.
Trial summary
It is a single-center, single-arm, open-labeled clinical trial to evaluate the efficacy and safety of CAR-T cells therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP). Read more about this study
- Who is it for?
- Ages 8 to 75 years
- Conditions the study is for
- ITP - Immune Thrombocytopenia
- What is being tested?
- CAR-T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2024to 31 Dec 2027
- 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 8 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willingness to complete the informed consent process and to comply with study procedures and visit schedule;
- Men and women aged 8-75;
- Participants diagnosed with chronic (>12 months duration) or refractory (a documented intolerance or insufficient response to the first and second line standard treatment of ITP) ITP;
- The results of physical, instrumental, and laboratory examination of patients not suggest any disease which may cause thrombocytopenia other than ITP;
- Platelet count <30 x 109 / L;
- If the patient is taking corticosteroids, the treatment regimen/dose should be stable (at least 2 weeks prior to screening);
- The results of physical, instrumental, and laboratory examination of patients should be within the normal range or deviations should be regarded by the researcher as clinically insignificant;
- Willingness to use effective and reliable methods of contraception throughout the entire study period;
Who cannot join
- All subjects with diseases which may cause secondary immune thrombocytopenia
- Patients with preventive splenectomy;
- Hemostatic disorders other than chronic thrombocytopenia;
- Subject treated with drugs that affect platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDs) or anti-coagulants for > 3 consecutive days within 2 weeks of the study start and until the end of the study;
- History of platelet agglutination abnormality that prevents reliable measurement of platelet counts;
- Concurrent malignant disease and/or history of cancer treatment with cytotoxic chemotherapy and/or radiotherapy;
Show all 18Show fewer
- Grade III-IV heart failure or myocardial infarction, cardiac angioplasty or stenting, unstable angina pectoris, or other clinically prominent heart disease within one year prior to enrollment;
- History of thrombosis or presence of significant risk factors for thrombosis;
- Persons with acute or exacerbation of chronic diseases of the gastrointestinal tract associated with the risk of bleeding, acute infectious diseases, pathologies of the respiratory system;
- Any clinically significant hepatic impairment (increase of serum transaminase levels by more than 3 times the upper limit of normal);
- Serum creatinine levels are more than two times higher than the upper limit of normal for a given age and sex;
- Any other concomitant decompensated diseases or acute conditions, the presence of which, according to the researcher, may significantly affect the results of the study;
- Human immunodeficiency virus (HIV) seropositivity, Hepatitis B surface antigen positive or hepatitis B core antibody positive and HBV-DNA positive, Patients with hepatitis C (HCV-RNA quantitative test results positive), Or the presence of other serious active viral or bacterial infections or uncontrolled systemic fungal infections;
- Patients with severe history of allergy or allergic constitution;
- Pregnancy and lactation;
- History of mental illness and known alcohol/drug addiction;
- Poor compliance due to physiological, family, social, geographical and other factors, unable to cooperate with the study protocol and follow-up plan;
- Had undergone other clinical trials in the 4 weeks prior to participating in this trial;
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, 650000
Sanbin Wang, Doctor, 13187424131 ext. +86, sanbin1011@163.com
About this study
Official title: An Investigator-initiated Trial to Evaluate the Efficacy and Safety of CAR-T Cells Therapy in the Treatment of Chronic or Refractory Primary Immune Thrombocytopenia (ITP)
It is a single-center, single-arm, open-labeled clinical trial to evaluate the efficacy and safety of CAR-T cells therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP).
Read the detailed description
This open label and single-arm study aims to evaluate the efficacy and safety of CAR-T cells therapy in patients with Chronic or Refractory Primary Immune Thrombocytopenia (ITP). After enrollment, a leukapheresis procedure will be performed to manufacture chimeric antigen receptor (CAR) modified T cells. Patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide, then the CAR-T cells will be infused by vein. After infusion, subjects will be followed for safety and efficacy evaluation up to 12 weeks. For those with a durable remission 12 weeks after infusion, the follow-up will last for at least 12 months for disease control.
- Conditions
- ITP - Immune Thrombocytopenia
- Being tested
- CAR-T cells
- Keywords
- CAR-T, Immune Thrombocytopenia (ITP)
- 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.
CAR-T cellsBiological
CAR-T cells will be administered by vein. Before CAR-T infusion，patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide.
| Study group | Role | What they receive |
|---|---|---|
| CAR-T Cells Therapy | Experimental | Subjects who meet the enrollment conditions will receive intravenous infusion of CAR-T cells after lymphodepletion. Receives: Biological: CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Portion of patients with response (R)
platelet count>30x10^9/L and at least 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
Measured: At least 2 weeks after infusion
Secondary outcomes (6)
Portion of patients with complete response (CR)
platelet count>100x10^9/L, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
Measured: At least 2 weeks after infusion
Portion of patients with relapse
platelet count below 30x10^9/L or less than 2-fold increase of baseline platelet count or bleeding after treating effectively; platelet counts confirmed on at least 2 separate occasions approximately 1 day apart
Measured: At least 2 weeks after infusion
Time (in days) from treatment start to response (R)
Time calculated from infusion to the day when the response (R) criteria are achieved
Measured: At least 2 weeks after infusion
Time (in days) from treatment to complete response (CR)
Time calculated from infusion to the day when the complete response (CR) criteria are achieved
Measured: At least 2 weeks after infusion
Duration (in days) of response (R)
Time calculated from the day when the response (R) criteria are achieved, to the day when loss of response (R) criteria is achieved
Measured: At least 2 weeks after infusion
Incidence of adverse events(AE) after infusion
The frequency, severity, and laboratory findings of all adverse events/serious adverse events are included. Description, time, classification, and outcome of AE events resulted from the investigational medical product, delivery method, or emergency measures will be recorded in the case report form.
Measured: Up to 12 months after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2024
- Study start1 Feb 2024
- Primary completion1 Jan 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ITP - Immune ThrombocytopeniaStudies 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
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sanbin Wang, Doctor, Principal investigator, 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06352281
- Sponsor's protocol ID
- KM-007
- Status last verified
- Jul 2024
- First submitted
- 2 Apr 2024
- Last updated
- 22 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator-initiated Trial to Evaluate the Efficacy and Safety of CAR-T Cells Therapy in the Treatment of Chronic or Refractory Primary Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT06352281
- Acronym / sponsor ID
- KM-007
- Conditions
- ITP - Immune Thrombocytopenia
- Keywords
- CAR-T, Immune Thrombocytopenia (ITP)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of ChinaUniversity, hospital or other
- Study start
- 1 Feb 2024 (actual)
- Primary completion
- 1 Jan 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 8 Apr 2024 (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.
It is a single-center, single-arm, open-labeled clinical trial to evaluate the efficacy and safety of CAR-T cells therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP).
Detailed description
This open label and single-arm study aims to evaluate the efficacy and safety of CAR-T cells therapy in patients with Chronic or Refractory Primary Immune Thrombocytopenia (ITP). After enrollment, a leukapheresis procedure will be performed to manufacture chimeric antigen receptor (CAR) modified T cells. Patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide, then the CAR-T cells will be infused by vein. After infusion, subjects will be followed for safety and efficacy evaluation up to 12 weeks. For those with a durable remission 12 weeks after infusion, the follow-up will last for at least 12 months for disease control.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAR-T Cells Therapy | Experimental | Biological: CAR-T cellsSubjects who meet the enrollment conditions will receive intravenous infusion of CAR-T cells after lymphodepletion. |
Who may qualify
- Ages
- 8 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Willingness to complete the informed consent process and to comply with study procedures and visit schedule;
- Men and women aged 8-75;
- Participants diagnosed with chronic (>12 months duration) or refractory (a documented intolerance or insufficient response to the first and second line standard treatment of ITP) ITP;
- The results of physical, instrumental, and laboratory examination of patients not suggest any disease which may cause thrombocytopenia other than ITP;
- Platelet count <30 x 109 / L;
Key exclusion criteria (18 listed)
- All subjects with diseases which may cause secondary immune thrombocytopenia
- Patients with preventive splenectomy;
- Hemostatic disorders other than chronic thrombocytopenia;
- Subject treated with drugs that affect platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDs) or anti-coagulants for > 3 consecutive days within 2 weeks of the study start and until the end of the study;
- History of platelet agglutination abnormality that prevents reliable measurement of platelet counts;
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 |
|---|---|---|
| CAR-T cellsCAR-T cells will be administered by vein. Before CAR-T infusion，patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide. | Biological | CAR-T Cells Therapy |
Primary outcome
Portion of patients with response (R)
Time frame: At least 2 weeks after infusion
platelet count>30x10^9/L and at least 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
6 secondary outcomes
Portion of patients with complete response (CR)
Time frame: At least 2 weeks after infusion
Portion of patients with relapse
Time frame: At least 2 weeks after infusion
Time (in days) from treatment start to response (R)
Time frame: At least 2 weeks after infusion
Time (in days) from treatment to complete response (CR)
Time frame: At least 2 weeks after infusion
Duration (in days) of response (R)
Time frame: At least 2 weeks after infusion
Incidence of adverse events(AE) after infusion
Time frame: Up to 12 months after 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 22 Jul 2024.
Related trials for ITP - Immune Thrombocytopenia
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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.
Sanbin Wang, Doctor principal investigator
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CAR-T cells in people with ITP - Immune Thrombocytopenia, run by 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China at 1 site in China.
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 8 inclusion and 18 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 22 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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 Sanbin Wang, Doctor (13187424131 ext. +86, sanbin1011@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Portion of patients with response (R) (time frame: At least 2 weeks after 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 22 Jul 2024.