The Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of HMPL-523 in Adult Subjects With Immune Thrombocytopenia (ITP)

HUTCHMED

Status and phase: Enrolling; Phase 1

Conditions: Blood Platelet Disorder; Thrombocytopenia; Hemorrhage; Thrombotic Microangiopathies; Immune System Diseases; Hemorrhagic Disorders; Primary Immune Thrombocytopenia; Purpura, Thrombocytopenic; Pathologic Processes; Autoimmune Diseases; Purpura, Thrombocytopenic, Idiopathic; Skin Manifestations; Immune Thrombocytopenia; Purpura; ITP - Immune Thrombocytopenia; Blood Coagulation Disorder; Hematologic Diseases

Treatments: Drug: HMPL-523

Study type: Interventional

Funder types: Industry

Identifiers

NCT06291415

2022-523-GLOB1

Details and patient eligibility

About

This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.

Full description

This study is a Phase 1b, open-label, multicenter, single-arm study to evaluate the safety, tolerability, and preliminary efficacy of HMPL-523 in adult subjects with primary ITP diagnosed at least 3 months prior to enrollment or randomization.

In the dose escalation stage (Part 1), subjects will receive one of 3 dose levels of HMPL-523 to determine the recommended dose of HMPL-523 for the randomized dose optimization- stage (Part 2).

At the end of Part 1, 2 dose levels will be selected to be used in the dose-optimization stage (Part 2) of the study. In Part 2 of the study, subjects will be randomized in a 1:1 ratio between the 2 dose levels to better understand the exposure/efficacy/toxicity relationship.

At the end of Part 2, the Recommended Phase 3 dose (RP3D) of HMPL-523 will be determined based on the safety, efficacy and PK data.

Enrollment: 48 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Subjects may be enrolled in this study only if they satisfy all the following criteria:

1. Adult male or female subjects ≥18 years of age
2. Diagnosis of ITP, with a duration of disease of at least 3 months prior to randomization or enrollment
3. Intolerance or insufficient response or recurrence after at least 1 prior ITP treatment (excluding splenectomy)
4. Response (defined as achieved a platelet count ≥50 × 109/L) to at least 1 prior ITP therapy (including splenectomy)
5. Adequate hematologic, hepatic and renal function

Exclusion criteria

Subjects are not eligible for enrollment into this study if any one of the following criteria are met:

1. Evidence of the presence of secondary causes of ITP
2. Clinically serious hemorrhage requiring immediate adjustment of platelets
3. Known history of vital organ transplantation or hematopoietic stem-cell transplantation or chimeric antigen receptor T-cells (CAR-T) therapy
4. Splenectomy within 12 weeks prior to enrollment
5. Presence of active malignancy unless deemed cured by adequate treatment.
6. History of serious cardiovascular disease corrected QT interval (QTcF) ≥450 ms
7. Uncontrolled hypertension
8. Being unsuitable to participate in this study as considered by investigators

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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48 participants in 2 patient groups

Dose escalation

Experimental group

Description:

Part 1 will consist of the following 3 dose levels: 300, 400, and 500 mg once daily (QD).

Treatment:

Drug: HMPL-523

Dose optimization stage

Experimental group

Description:

In part 2 subjects will be randomized in a 1:1 ratio between the 2 dose levels selected at the end of part 1.

Treatment:

Drug: HMPL-523