ATRA for Management of Primary ITP

Official title

All-trans Retinoic Acid for Management of Primary Immune Thrombocytopenia : a Randomized, Double-blind, Placebo-controlled Study

Overview

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).

Detailed description

The investigators are undertaking a parallel-group, multicenter, randomized controlled trial of 192 adults with corticosteriod-resistant/relapsed primary ITP. Patients were randomized to all-trans etinoic acid and placebo group. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Interventions

• Drug all-trans retinoic acid (ATRA)
  10mg twice daily ×24 weeks
• Drug Placebo
  10mg twice daily ×24 weeks

Primary outcome measures

• Durable platelet response [Time frame: Up to week 24]

Secondary outcome measures (12)

• 24-week overall response rate [Time frame: Up to week 24]
• 12-week overall response rate [Time frame: Up to week 12]
• Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30×10^9/L) [Time frame: Up to week 24]
• Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50×10^9/L) [Time frame: Up to week 24]
• Quality of Life Score [Time frame: Up to week 24]
• Adverse Events [Time frame: Up to week 28]
• Complete response rate [Time frame: Up to week 24]
• Duration of response [Time frame: Up to 24 weeks]
• Time to response [Time frame: Up to week 24]
• Initial response [Time frame: Up to week 4]
• Peak platelet count [Time frame: Up to week 24]
• Bleeding events [Time frame: 0-12 weeks and 0-24 weeks]

Eligibility criteria

Inclusion criteria

• 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.

Exclusion criteria

• 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;
• WBC < 2500/µL, neutrophil count < 1200/µL, lymphocyte count < 750/µL, hemoglobin < 9 g/dL;
• eGFR < 50 mL/min/1.73m²;
• Other patients deemed unsuitable for enrollment in this study by the investigator.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

China · 1 center

• Peking University Institute of hematology, People's Hospital — Beijing

Identifiers

NCT: NCT07597395 · 2025PHD007-001

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗