NCT07287228

The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia

An Ib/II Phase Clinical Study Evaluating the Safety and Preliminary Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 37 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study consists of two phases, phase Ib and phase II. The primary objective of phase Ib is to evaluate the safety and tolerability of multiple doses of daratumumab in patients with refractory aplastic anemia. The primary objective of phase II is to preliminarily assess the efficacy of multiple doses of daratumumab in patients with refractory aplastic anemia. Secondary objectives include evaluation of other efficacy endpoints, safety (phase II), and pharmacodynamic characteristics of multiple doses of daratumumab in these patients. The exploratory objective is to assess biological functional changes in peripheral blood and bone marrow before and after daratumumab treatment.

Detailed description

This study is a seamless phase Ib/II design. The target population comprises adult patients with refractory aplastic anemia (AA) who meet the criteria for refractory AA at enrollment, with platelet counts (PLT) \< 30×10⁹/L or hemoglobin (HGB) \< 90 g/L. A Safety Review Committee (SRC), led by the principal investigator, will be established. If the phase Ib results demonstrate sufficient safety and allow determination of the maximum tolerated dose (MTD), the phase II study will proceed directly. The phase Ib study serves as the dose-escalation stage, with three dose cohorts: low-dose (8 mg/kg), medium-dose (12 mg/kg), and high-dose (16 mg/kg), administered once weekly for six consecutive doses. Dose escalation follows the "3+3" design.

Conditions

• Aplastic Anemia
• Refractory
• Relapse

Interventions

Type	Name	Description
DRUG	Daratumumab	In Phase I, eligible subjects who meet the inclusion criteria will receive daratumumab at doses of 8 mg/kg, 12 mg/kg, or 16 mg/kg. The treatment period will last 6 weeks, with intravenous infusion administered once weekly (QW) for a total of 6 consecutive doses. During the conduct of the clinical trial, based on the clinical trial data obtained (safety, MTD, etc.) and after discussion by the SRC, the RP2D for Phase II will be determined. In Phase II, eligible subjects who meet the inclusion criteria will receive intravenous daratumumab once weekly (specific dose based on the RP2D determined from Phase Ib clinical trial data after SRC discussion) for a total of 6 consecutive doses.

Timeline

• Start date: 2025-12-10
• Primary completion: 2027-12-31
• Completion: 2028-12-31
• First posted: 2025-12-17
• Last updated: 2025-12-17

Locations

1 site across 1 country: China