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

NCT06291415 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: 2022-523-GLOB1
• Study Type: interventional
• Target: N/A
• Locations: 5 countries
• Sites: 28

Brief Summary

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

Conditions

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

Keywords

ITP; sovleplenib

Outcome Measures

Primary Outcomes (2)

• Safety and tolerability of HMPL-523 in adult subjects with primary ITP

  Calculated as the number and percent incidence of participants experiencing adverse events (AE).

  week 1 - week 24

• Dose Limiting Toxicities

  Defined as an adverse event AE that meets protocol defined Dose Limiting Toxicities (DLT) criteria during the DLT assessment window (first 28 days), unless clearly unrelated to ITP drugs.

  week 1 - week 4

Secondary Outcomes (4)

• Cmax (maximum plasma drug concentration)

  week 1 and week 3

• AUCtau (area under the concentration-time curve over a dosage interval)

  week 1 and week 3

• Tmax (time to reach maximum plasma drug concentration)

  week 1 and week 3

• Cmin (minimum plasma drug concentration)

  week 1 - week 20

Study Arms (2)

Dose escalation

EXPERIMENTAL

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

Drug: HMPL-523

Dose optimization stage

EXPERIMENTAL

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

Drug: HMPL-523

Interventions

HMPL-523 DRUG

Syk inhibitor

Also known as: sovleplenib

Dose escalation; Dose optimization stage

Eligibility Criteria

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

You may qualify if:

• Subjects may be enrolled in this study only if they satisfy all the following criteria:
• Adult male or female subjects ≥18 years of age
• Diagnosis of ITP, with a duration of disease of at least 3 months prior to randomization or enrollment
• Intolerance or insufficient response or recurrence after at least 1 prior ITP treatment (excluding splenectomy)
• Response (defined as achieved a platelet count ≥50 × 109/L) to at least 1 prior ITP therapy (including splenectomy)
• Adequate hematologic, hepatic and renal function

You may not qualify if:

• Subjects are not eligible for enrollment into this study if any one of the following criteria are met:
• Evidence of the presence of secondary causes of ITP
• Clinically serious hemorrhage requiring immediate adjustment of platelets
• Known history of vital organ transplantation or hematopoietic stem-cell transplantation or chimeric antigen receptor T-cells (CAR-T) therapy
• Splenectomy within 12 weeks prior to enrollment
• Presence of active malignancy unless deemed cured by adequate treatment.
• History of serious cardiovascular disease corrected QT interval (QTcF) ≥450 ms
• Uncontrolled hypertension
• Being unsuitable to participate in this study as considered by investigators

Sponsors & Collaborators

• Hutchmed: lead

Study Sites (28)

Childrens Hospital of California

Irvine, California, 92868, United States

Location

Georgetown University Medical Center - Georgetown Lombardi Comprehensive Cancer Center

Georgetown, Delaware, 20007, United States

Location

Center for Cancer and Blood Disorders

Bethesda, Maryland, 20817, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

San Juan Oncology Associates

Farmington, New Mexico, 87401, United States

Location

East Carolina University, Brody School of Medicine

Greenville, North Carolina, 27834, United States

Location

Taussig Cancer Institute

Cleveland, Ohio, 44106, United States

Location

Oklahoma Cancer Specialists and Research Institute

Tulsa, Oklahoma, 74146, United States

Location

Texas Oncology San Antonio Medical Center

San Antonio, Texas, 78240, United States

Location

University of Washington (UW) Medical Center

Seattle, Washington, 98195, United States

Location

Peninsula Private Hospital

Frankston, Victoria, Australia

Location

The Perth Blood Institute (PBI) Hollywood Specialist Centre

West Perth, Western Australia, Australia

Location

Royal Adelaide Hospital

Adelaide, Australia

Location

Canberra Hospital

Canberra, Australia

Location

Charite university

Berlin, 10117, Germany

Location

Marien Hospital Dusseldorf

Düsseldorf, 40479, Germany

Location

UMG Gottingen Hämatologie

Göttingen, Germany

Location

University Hospital of Schleswig-Holstein, Department of Haematology and Oncology

Lübeck, Germany

Location

Sykehuset Ostfold Kalnes (fosta) / Osfold Hospital Trust (MSL)

Grålum, 1714, Norway

Location

Oslo University Hospital

Oslo, Norway

Location

Hospital del Mar Barcelona

Barcelona, 08003, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, Spain

Location

University de Burgos

Burgos, 09001, Spain

Location

Hospital Gregorio Maranon Madrid

Madrid, 28007, Spain

Location

Clinica Universidad de Navarra

Madrid, 28027, Spain

Location

Hospital Infanta Leonor

Madrid, 28031, Spain

Location

Fundacion Jimenez Diaz

Madrid, 28040, Spain

Location

Hospital Morales Meseguer

Murcia, 30008, Spain

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Hematologic Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Skin Manifestations; Thrombocytopenia

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Cytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Study Officials

• William Schelman, MD, PhD

  Hutchmed

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1: Dose escalation stage - 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) Part 2: subjects will be randomized in a 1:1 ratio between the 2 dose levels recommended by the SRC to better understand the exposure/efficacy/toxicity relationship. At the end of Part 2, the SRC will evaluate the safety, tolerability, preliminary efficacy, and PK data to determine the Recommended Phase 3 dose (RP3D) of HMPL-523.

Study Record Dates

• First Submitted: February 13, 2024
• First Posted: March 4, 2024
• Study Start: April 2, 2024
• Primary Completion: April 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: September 16, 2025

Record last verified: 2025-09

Locations

US (10); AU (4); ES (8); DE (4); NO (2)