ATRA for Management of Primary ITP

NCT07597395 | Not Yet Recruiting Phase 3

• 1 other identifier: 2025PHD007-001
• Study Type: interventional
• Target: 192
• Locations: 1 country
• Sites: 1

Brief Summary

A multicenter, randomized, double-blind placebo-controlled study to report the efficacy and safety of all-trans etinoic acid compared to placebo for the treatment of adults with corticosteriod-resistant/relapsed primary immune thrombocytopenia (ITP).

Conditions

Immune Thrombocytopenia

Keywords

Immune thrombocytopenia;; Corticosteriod resistance resistant/relaped; ATRA

Outcome Measures

Primary Outcomes (1)

• Durable platelet response

  Platelet count of ⩾50 × 10\^9/L or between ⩾30 × 10\^9/L and \<50 × 10\^9/L and at least doubled from baseline on at least four of six scheduled visits between weeks 14 and 24

  Up to week 24

Secondary Outcomes (14)

• 24-week overall response rate

  Up to week 24

• 12-week overall response rate

  Up to week 12

• Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30×10^9/L)

  Up to week 24

• Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50×10^9/L)

  Up to week 24

• Quality of Life Score

  Up to week 24

Study Arms (2)

ATRA

EXPERIMENTAL

ATRA 10mg twice daily

Drug: all-trans retinoic acid (ATRA)

Placebo

PLACEBO COMPARATOR

Placebo 10mg twice daily

Drug: Placebo

Interventions

all-trans retinoic acid (ATRA) DRUG

10mg twice daily ×24 weeks

Also known as: tretinoin

ATRA

Placebo DRUG

10mg twice daily ×24 weeks

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Primary ITP if aged ⩾18 years;
• With an average of two platelet counts ⩾1 day apart of \<30×10\^9/L during screening and no single platelet count \>35×10\^9/L within 2 weeks before study treatment;
• Patients who have previously received at least one first-line standard therapy for ITP (corticosteroid and/or intravenous immunoglobulin) with unsustained efficacy, relapse, intolerance to standard therapy, or insufficient response.

You may not qualify if:

• Pregnant or lactating women, and who were possibly pregnant, planning to become pregnant, or who had partners planning to become pregnant;
• With active malignancy or a history of malignant tumor;
• Having experienced severe bacterial, viral, fungal or parasitic infection within the past 4 weeks;
• With a history of symptomatic herpes zoster infection within 12 weeks prior to screening;
• Active or chronic HBV, HCV or HIV infection;
• Evidence of active tuberculosis; or previous evidence of active tuberculosis without appropriate and documented treatment; or household contact with patients with active tuberculosis without appropriate and documented tuberculosis prophylaxis;
• Receipt of live vaccines within the past 12 weeks, or planned live vaccination during the study period;
• Prior ATRA therapy;
• History of solid organ transplant or planned surgery;
• Myelodysplastic syndrome, aplastic anemia or myelofibrosis;
• Patients with other diseases were undergoing treatment with immunosuppressants;
• Clinically significant thromboembolic events within the past 24 weeks, or ongoing anticoagulant treatment, who are deemed ineligible for the study by the investigator;
• History or presence of myocardial infarction, unstable ischemic heart disease, stroke, or NYHA Class IV heart failure;
• History or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, neuropsychiatric or any other severe and/or unstable diseases that, in the opinion of the investigator, may pose an unacceptable risk with the investigational product or interfere with the interpretation of study data;
• AST \> 2 times the upper limit of normal (ULN), ALT \> 2×ULN, TBIL ≥ 1.5×ULN;

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Peking University First Hospital: collaborator
• Beijing Friendship Hospital: collaborator
• Beijing Tongren Hospital: collaborator
• Peking University Third Hospital: collaborator
• Beijing Hospital: collaborator

Study Sites (1)

Peking University Institute of hematology, People's Hospital

Beijing, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Tretinoin

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Vitamin A; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Diterpenes; Pigments, Biological; Biological Factors

Central Study Contacts

Xiaohui Zhang, Prof.

CONTACT

861088326001; zhangxh@bjmu.edu.cn

Haixia Fu, Dr.

CONTACT

861088326002; fuhaixia_210@163.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking University Institute of Hematology

Study Record Dates

• First Submitted: May 13, 2026
• First Posted: May 19, 2026
• Study Start (Estimated): May 30, 2026
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2028
• Last Updated: May 19, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)