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Not yet recruitingNCT07905144Updated Oct 29, 2026

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

An Early Phase 1 interventional study of iPSC-derived platelets and iPSC-derived platelets in Thrombocytopenia, sponsored by Ruijin Hospital. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-29.

Sponsored by Ruijin Hospital · Early Phase 1, Interventional, and Treatment

Updated Oct 29, 2026Newly registeredGo to Updates ↓
Phase
Early Phase 1
Study type
Interventional
Enrollment
9
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study 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 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.

02

Conditions studied

  • Thrombocytopenia

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03

In context

Thrombocytopenia

705 studies on the registry are indexed under Thrombocytopenia; 153 are open to participants now.

This study's planned enrollment of 9 is below the median of 54 across 477 interventional studies indexed under Thrombocytopenia.

Browse Thrombocytopenia studies →

Lead sponsor

Ruijin Hospital is the lead sponsor of 674 studies on the registry; 382 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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.

Exclusion criteria

Exclusion Criteria:

  • 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;
  • 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.
05

Study design

Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
9 participants (estimated)

Study arms

  • Experimental
    iPSC-derived platelets Dose 1

    iPSC-derived platelets

    Drug: iPSC-derived platelets

  • Experimental
    iPSC-derived platelets Dose 2

    iPSC-derived platelets

    Drug: iPSC-derived platelets

  • Experimental
    iPSC-derived platelets Dose3

    iPSC-derived platelets

    Drug: iPSC-derived platelets

Interventions

  • DrugiPSC-derived platelets

    for injection

  • DrugiPSC-derived platelets

    for injection

  • DrugiPSC-derived platelets

    for injection

06

What researchers measure

Primary outcomes

  1. 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)

    Time frame: within 28 days post-infusion of iPSC-derived platelets(RBT101)

Secondary outcomes

  1. CCI

    Changes in CCI within 7 days after iPSC-derived platelets(RBT101) transfusion

    Time frame: within 7 days post-infusion of iPSC-derived platelets(RBT101)

  2. PPR

    Changes in PPR within 7 days after iPSC-derived platelets(RBT101) transfusion

    Time frame: within 7 days post-infusion of iPSC-derived platelets(RBT101)

  3. TEG

    Changes in TEG within 7 days after iPSC-derived platelets(RBT101) transfusion

    Time frame: within 7 days post-infusion of iPSC-derived platelets(RBT101)

07

Study locations

No study locations are listed for this record.

08

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Start is estimated for Dec 2026; primary completion for Oct 2027, 10 months later.

Of 28 phase 1 interventional studies under Thrombocytopenia, half reached primary completion within 17 months of starting (the median), counting those still running; a quarter within 6 and three quarters within 41. This study's planned 10 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 23, 2026
First posted
Oct 29, 2026
StartEst.
Dec 30, 2026
Primary completionEst.
Oct 30, 2027
CompletionEst.
Dec 30, 2027
Last update
Oct 29, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Registered
First appeared on the registry. No changes since
Oct 29, 2026
Show all 1 update
  1. Oct 29, 2026
    First appeared on the registry

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

09

Registry details

Key details

Study ID
NCT07905144
Lead sponsor
Ruijin Hospital
Collaborators
Renerval Biotherapeutics
Responsible party
Hu Xiaoxia (Chief Physician, Ruijin Hospital) — Principal investigator
First posted
Oct 29, 2026
Start date
Dec 30, 2026 (estimated)
Primary completion
Oct 30, 2027 (estimated)
Completion
Dec 30, 2027 (estimated)
Last update
Oct 29, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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