A Phase 3 study of Hetrombopag in patients with low blood platelet count (thrombocytopenia) caused by chemotherapy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Jiangsu Hengrui Pharmaceuticals Co. Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Not possible to specify

Trial duration

20 May 2026 → ongoing

Decision date (initial)

2026-04-27

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Jiangsu Hengrui Pharmaceuticals Co. Ltd.

External identifiers

EU CT number

2025-524209-34-00

ClinicalTrials.gov

NCT07286032

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

Part A
• To evaluate the pharmacokinetic (PK) characteristics of hetrombopag in non-Asian participants with chemotherapy-induced thrombocytopenia (CIT).
Part B
• To evaluate the efficacy of hetrombopag treatment in participants with CIT.

Secondary objectives 2

1. Part A • To evaluate the efficacy and safety of hetrombopag treatment in nonAsian participants with CIT.
2. Part B • To evaluate the safety of hetrombopag treatment in participants with CIT.

Conditions and MedDRA coding

Chemotherapy induced thrombocytopenia

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

Plan to share IPD

No

IPD plan description

Currently the option to share IPD is being investigated, considering important ethical questions regarding participant consent and autonomy. Hengrui is currently evaluating the implications of informed consent processes and IPD sharing which may need further discussion with ECs, including consultation with key stakeholders (investigators, data protection officers, patient advocacy groups). Any update to the IPD Sharing plan will be submitted for review through CTIS in due time.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Male or female gender, age ≥18 years at screening.
2. Histologically or cytologically confirmed solid tumor
3. Receiving platinum- and/or gemcitabine-containing chemotherapy regimens on 21-day treatment cycles.
4. PC < 75×109/L at enrollment.
5. Experiencing a protocol-defined treatment delay of ≥7 days per chemotherapy cycle due to CIT with PC <75×109/L (Part B only).
6. Eastern Cooperative Oncology Group performance status (ECOG PS) 0-2.
7. Adequate organ and hematologic function
8. Having non-Asian race and ethnicity (Part A only).

Exclusion criteria 7

1. PC <25×109/L at screening.
2. Hematopoietic diseases other than CIT (e.g., primary immune thrombocytopenia).
3. Hematologic malignancies
4. Untreated brain metastases; or with leptomeningeal metastasis.
5. Bone marrow involvement or bone marrow metastasis on routine imaging.
6. Have arterial/venous thrombosis within 6 months prior to Study Day 1
7. Severe hemorrhage (e.g., gastrointestinal, or intracranial hemorrhage) within 28 days prior to Study Day 1.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Part A: PK parameters (e.g., Cmax, AUC0-tau, Cmin) of hetrombopag in non-Asian participants with CIT.
2. Proportion of treatment responders meeting these criteria: 1. Platelet count (PC) ≥100×109 /L within 14 days after initiating IMP treatment (incl Day 14); 2. Complete two consecutive on-study chemotherapy cycles (C1 and C2) without thrombocytopenia-induced modification of any myelosuppressive agent; 3. No use of any rescue therapy for thrombocytopenia during the treatment period from the initiation of IP treatment until Day 21 of C2 (C2D21; window period: +4 days).

Secondary endpoints 13

1. Proportion of participants who completed two consecutive on-study chemotherapy cycles without thrombocytopenia-induced modification of any myelosuppressive agent.
2. Proportion of participants achieving PC ≥100×109 /L without the use of rescue therapy within 14 days after initiating the IP treatment (including Day 14)
3. Proportion of participants with at least a single incidence of rescue therapy.
4. Time to rescue therapy free first platelet response of PC ≥100×109/L defined as the interval between the initiation of IP treatment and the first platelet response.
5. Cumulative duration of platelet response of PC ≥100×109/L without the use of rescue therapy.
6. PC nadir from C1D1 until C2D21 (window period: +4 days).
7. Cumulative duration of severe thrombocytopenia, defined as the number of consecutive days with a PC of ≤50×109/L.
8. Proportion of participants who completed two consecutive on-study chemotherapy cycles without treatment regimen modification.
9. Proportion of participants with all cause events leading to treatment regimen modifications including dose reduction, delay, omission, or discontinuation.
10. Proportion of participants who are free from serious bleeding events, defined as Grade >=2 per the World Health Organization (WHO) bleeding scale, during the treatment period from the initiation of IP treatment until C2D21 (window period: +4 days).
11. Number and proportion of participants with adverse events (AEs)/serious adverse events (SAEs), safety laboratory parameters, vital signs, etc.
12. Proportion of participants with neutropenia during the treatment period from the initiation of IP treatment until C2D21 (window period: +4 days).
13. Proportion of non-Asian treatment responders who meet the following criteria (Part A only): 1) Platelet count (PC) of 100×109/L within 14 days after initiating the IP treatment (incl. Day 14); 2) Complete two consecutive on-study chemotherapy cycles (C1 and C2) without thrombocytopenia-induced modification of any myelosuppressive agent; 3) No use of any rescue therapy for thrombocytopenia during the treatment period from initiation of IMP treatment until Day 21 of C2 (C2D21; window period:+4d)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Jiangsu Hengrui Pharmaceuticals Co. Ltd.

Sponsor organisation

Jiangsu Hengrui Pharmaceuticals Co. Ltd.

Address

7 Kunlunshan Road, Economic And Technological Development Zone Economic And Technological Development Zone

City

Lianyungang

Postcode

222047

Country

China

Scientific contact point

Organisation

Jiangsu Hengrui Pharmaceuticals Co. Ltd.

Contact name

Hengrui Clinical Trials EU

Public contact point

Organisation

Jiangsu Hengrui Pharmaceuticals Co. Ltd.

Contact name

Hengrui Clinical Trials EU

Third parties 13

Locations

5 EU/EEA countries · 9 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 68 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications