ThrombocytopeniaEarly Phase 1NCT07905144
A Clinical Study Evaluating the Safety and Preliminary Effectiveness of Induced Pluripotent Stem Cell-derived Platelets in Patients With Thrombocytopenia Caused by Allogeneic Hematopoietic Stem Cell Transplantation
A Early Phase 1 study testing iPSC-derived platelets in people with Thrombocytopenia, run by Ruijin Hospital.
Trial summary
A multicenter open-label study with dose-escalation and multiple expansion cohorts, aimed at evaluating the safety and preliminary efficacy of IPSC-derived platelets in patients with thrombocytopenia caused by allogeneic hematopoietic stem cell transplantation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia
- What is being tested?
- iPSC-derived platelets
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- No sites listed yet
- Study dates
- 30 Dec 2026to 30 Dec 2027
- Phase
- Early Phase 1
- 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
- Participants must be 18 years old or older, with no gender restrictions;
- First-time allogeneic hematopoietic stem cell transplantation patients;
- Platelet count between 20×10*9/L ;
- Absence of peripheral blood platelet transfusions in the three days prior to receiving human-induced pluripotent stem cell-derived platelets;
- ECOG score of ≤ 2;
- Participants and/or their guardians (if applicable) must comprehend and sign the informed consent form.
Who cannot join
- History of ineffective platelet transfusion ;
- Platelet antibody positive, and/or donor-specific antibody (DSA) positive ；
- Individuals with severe transfusion related allergic reactions in the past; Patients with recurrent malignant hematological tumors that have not yet improved;
- Within 7 days prior to receiving the investigational drug;
- Patients with existing severe bleeding (such as intracranial hemorrhage, severe gastrointestinal bleeding, or severe hemoptysis);
- For patients with hemophilia and coagulation system diseases;
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- History of pulmonary embolism and hepatic vein thrombosis;
- Patients with disseminated intravascular coagulation;
- Use of anticoagulant medications within 7 days before screening;
- Individuals with positive results in screening for antibodies against the human immunodeficiency virus or specific antibodies against Treponema pallidum (the causative agent of syphilis); or those who test positive for hepatitis C antibodies, hepatitis B surface antigen, or have a history of hepatitis B, or individuals with positive hepatitis B core antibodies and HBV-DNA ≥2000 IU/mL in the past 3 months;
- Individuals with uncontrolled active infections;
- Body weight exceeding 80 kg;
- Have undergone hematopoietic stem cell transplantation and have an acute graft-versus-host disease that requires control;
- Women of childbearing age who are pregnant or breastfeeding, and male participants who plan to donate sperm within 180 days after medication administration;
- The investigator believes that due to any medical condition, the assessment cannot be part of this study, or the participant is unable to complete the follow-up investigation.
About this study
A multicenter open-label study with dose-escalation and multiple expansion cohorts, aimed at evaluating the safety and preliminary efficacy of IPSC-derived platelets in patients with thrombocytopenia caused by allogeneic hematopoietic stem cell transplantation.
Read the detailed description
Dose escalation This section aims to assess the safety, preliminary effectiveness, and immunogenicity of IPSC-derived platelets in patients with thrombocytopenia caused by allogeneic hematopoietic stem cell transplantation.
- Conditions
- Thrombocytopenia
- Being tested
- iPSC-derived platelets
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iPSC-derived plateletsDrug
for injection
iPSC-derived plateletsDrug
for injection
iPSC-derived plateletsDrug
for injection
| Study group | Role | What they receive |
|---|---|---|
| iPSC-derived platelets Dose 1 | Experimental | iPSC-derived platelets Receives: Drug: iPSC-derived platelets |
| iPSC-derived platelets Dose 2 | Experimental | iPSC-derived platelets Receives: Drug: iPSC-derived platelets |
| iPSC-derived platelets Dose3 | Experimental | iPSC-derived platelets Receives: Drug: iPSC-derived platelets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity, and relationship to iPSC-derived platelets(RBT101) of adverse events within 28 days post-infusion of iPSC-derived platelets(RBT101)
within 28 days post-infusion of iPSC-derived platelets(RBT101)
Measured: within 28 days post-infusion of iPSC-derived platelets(RBT101)
Secondary outcomes (3)
CCI
Changes in CCI within 7 days after iPSC-derived platelets(RBT101) transfusion
Measured: within 7 days post-infusion of iPSC-derived platelets(RBT101)
PPR
Changes in PPR within 7 days after iPSC-derived platelets(RBT101) transfusion
Measured: within 7 days post-infusion of iPSC-derived platelets(RBT101)
TEG
Changes in TEG within 7 days after iPSC-derived platelets(RBT101) transfusion
Measured: within 7 days post-infusion of iPSC-derived platelets(RBT101)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2026
- Study start30 Dec 2026 (estimated)
- Primary completion30 Oct 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Renerval Biotherapeutics
- Responsible party
- Principal investigator, Hu Xiaoxia, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07905144
- Sponsor's protocol ID
- RBT101-IIT-04
- Status last verified
- Oct 2026
- First submitted
- 23 Oct 2026
- Last updated
- 29 Oct 2026
- 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 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study Evaluating the Safety and Preliminary Effectiveness of Induced Pluripotent Stem Cell-derived Platelets in Patients With Thrombocytopenia Caused by Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07905144
- Acronym / sponsor ID
- RBT101-IIT-04
- Conditions
- Thrombocytopenia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Collaborators
- Renerval Biotherapeutics
- Study start
- 30 Dec 2026 (estimated)
- Primary completion
- 30 Oct 2027 (estimated)
- Study completion
- 30 Dec 2027 (estimated)
- First posted
- 29 Oct 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.
A multicenter open-label study with dose-escalation and multiple expansion cohorts, aimed at evaluating the safety and preliminary efficacy of IPSC-derived platelets in patients with thrombocytopenia caused by allogeneic hematopoietic stem cell transplantation.
Detailed description
Dose escalation This section aims to assess the safety, preliminary effectiveness, and immunogenicity of IPSC-derived platelets in patients with thrombocytopenia caused by allogeneic hematopoietic stem cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| iPSC-derived platelets Dose 1 | Experimental | Drug: iPSC-derived plateletsiPSC-derived platelets |
| iPSC-derived platelets Dose 2 | Experimental | Drug: iPSC-derived plateletsiPSC-derived platelets |
| iPSC-derived platelets Dose3 | Experimental | Drug: iPSC-derived plateletsiPSC-derived platelets |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Participants must be 18 years old or older, with no gender restrictions;
- First-time allogeneic hematopoietic stem cell transplantation patients;
- Platelet count between 20×10*9/L ;
- Absence of peripheral blood platelet transfusions in the three days prior to receiving human-induced pluripotent stem cell-derived platelets;
- ECOG score of ≤ 2;
Key exclusion criteria (15 listed)
- History of ineffective platelet transfusion ;
- Platelet antibody positive, and/or donor-specific antibody (DSA) positive ；
- Individuals with severe transfusion related allergic reactions in the past; Patients with recurrent malignant hematological tumors that have not yet improved;
- Within 7 days prior to receiving the investigational drug;
- Patients with existing severe bleeding (such as intracranial hemorrhage, severe gastrointestinal bleeding, or severe hemoptysis);
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 |
|---|---|---|
| iPSC-derived plateletsfor injection | Drug | iPSC-derived platelets Dose 1 |
| iPSC-derived plateletsfor injection | Drug | iPSC-derived platelets Dose 2 |
| iPSC-derived plateletsfor injection | Drug | iPSC-derived platelets Dose3 |
Primary outcome
Incidence, severity, and relationship to iPSC-derived platelets(RBT101) of adverse events within 28 days post-infusion of iPSC-derived platelets(RBT101)
Time frame: within 28 days post-infusion of iPSC-derived platelets(RBT101)
within 28 days post-infusion of iPSC-derived platelets(RBT101)
3 secondary outcomes
CCI
Time frame: within 7 days post-infusion of iPSC-derived platelets(RBT101)
PPR
Time frame: within 7 days post-infusion of iPSC-derived platelets(RBT101)
TEG
Time frame: within 7 days post-infusion of iPSC-derived platelets(RBT101)
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 29 Oct 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing iPSC-derived platelets in people with Thrombocytopenia, run by Ruijin Hospital.
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 15 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: opening soon, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity, and relationship to iPSC-derived platelets(RBT101) of adverse events within 28 days post-infusion of iPSC-derived platelets(RBT101) (time frame: within 28 days post-infusion of iPSC-derived platelets(RBT101)). 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 29 Oct 2026.