The Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of HMPL-523 in Immune Thrombocytopenia Patients

The Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of HMPL-523, a Syk Inhibitor in Adult Patients of Immune Thrombocytopenia: a Randomized, Double Blinded, Placebo Controlled Phase Ib Study

Sponsors

Lead Sponsor: Hutchison Medipharma Limited

Source Hutchison Medipharma Limited
Brief Summary

This is a randomized, double blinded, placebo-controlled phase Ib clinical trial in adult patients with immune thrombocytopenia. Cross-over treatment will be allowed during the study.

Detailed Description

Approximate 51 to 60 patients will be enrolled in dose escalation (3 cohorts, 8-20 subjects each with the ratio of 3:1 vs Placebo) .

Overall Status Recruiting
Start Date August 12, 2019
Completion Date December 31, 2021
Primary Completion Date December 31, 2021
Phase Phase 1
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of Participants with any Adverse Event From first dose to within 28 days after the last dose
Secondary Outcome
Measure Time Frame
Maximum plasma concentration (Cmax) Day 15, 16, 29, 43 and 47
Area under the concentration-time curve in a selected time interval (AUC0-t) Day 15, 16, 29, 43 and 47
Rate of Clinical Remission Day 1 to 8 weeks treatment
Enrollment 60
Condition
Intervention

Intervention Type: Drug

Intervention Name: HMPL-523

Description: HMPL-523 will be oral administrated once daily for 8 weeks and 16 weeks open-label treatment.

Intervention Type: Drug

Intervention Name: Placebo

Description: HMPL-523 matching placebo will be oral administrated once daily for 8 weeks and 16 weeks open-label treatment.

Arm Group Label: placebo arm

Eligibility

Criteria:

Inclusion Criteria: 1. Signed informed consent form 2. 18~75 years old male of female 3. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 4. Diagnosed immune thrombocytopenia before randomization with platelet decrease for more than 6 months. 5. Patients with refractory or relapsed ITP who have been treated with 1st line anti-ITP regimen or have experienced splenectomy. 6. Relative stable disease with World Health Organization (WHO) bleeding score of 0-1 and no rescue treatment needed within 2 weeks based on investigator's judgment. 7. Laboratory tests meet the following conditions: - During screening stage, twice PLT<30x10^9/L(exceed 24 hours) - Hb≥90g/L(if iron-deficiency anemia,Hb>80g/L),WBC>2.5x10^9/L, NEU>1.8x10^9/L - Crea≤1.5xULN and CCR≥50mL/min - TBIL、ALT、AST≤1.5xULN - Amylase、lipase

Gender: All

Minimum Age: 18 Years

Maximum Age: 75 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Hongyan Yin Study Director Hutchison MediPharma
Overall Contact

Last Name: jiayi Mai

Phone: 086-021-20673063

Email: Jiayim@hmplglobal.com

Location
Facility: Status: Contact: Blood diseases hospital, Chinese academy of medical university Renchi Yang rcyang65@163.com
Location Countries

China

Verification Date

September 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: treatment arm

Type: Active Comparator

Description: Eligible subjects will be treated with planned dose of 100 mg, 200 mg and 300 mg HMPL-523 once daily for 8 weeks and 16 weeks open-label treatment.

Label: placebo arm

Type: Placebo Comparator

Description: Eligible subjects will be treated with HMPL-523 matching placebo once daily for 8 weeks and 16 weeks open-label treatment.

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov