A Phase II Study Evaluating the Efficacy and Safety of ABSK021 (Pimicotinib)) in the Treatment of cGvHD Chronic Graft Versus Host Disease (cGvHD)

A Multicenter, Single-arm, Open-label, Phase II Clinical Study. This Study Consisting of Part A and Part B to Evaluate the Efficacy and Safety of ABSK021 (Pimicotinib) in Patients With Chronic Graft Versus Host Disease

This is a multicenter, single arm, open label phase II clinical study in China. This study will evaluate the efficacy and safety of ABSK021 (Pimicotinib) in the treatment of patients with cGvHD who failed first-line therapy.

Study Overview

• Status: Enrolling by invitation
• Conditions: cGvHD
• Intervention / Treatment: Drug: ABSK021

Detailed Description

This is a phase II, open-label study to evaluate safety, tolerability, pharmacokinetics (PK), and clinical benefit of ABSK021 in patients with hormone refractory or relapsed cGvHD. This study consisted of Part A and Part B, all cGvHD patients in this study will receive continuous oral treatment with ABSK021 once a day (QD) in a 28-day cycle, complete the core treatment period and extended treatment period, and receive regular follow-up until the termination of treatment is determined.

• Study Type: Interventional
• Enrollment (Estimated): 64
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China
      • Peking University People's Hospital
  • Chongqing
    • Chongqing, Chongqing, China
      • The Second Affiliated Hospital of the Army Medical University
  • Fujian
    • Fuzhou, Fujian, China
      • Fujian Medical University Union Hospital
  • Guangdong
    • Guangzhou, Guangdong, China
      • Guangdong Provincial Peoplep's Hospital
    • Zhujiang, Guangdong, China
      • Zhujiang Hospital of Southern Medical University
  • Hubei
    • Wuhan, Hubei, China
      • Union Hospital, Tongji Medical College,Huazhong University of Science and Technology
    • Wuhan, Hubei, China
      • Tongji Hospital, Tongji Medical College,Huazhong University of Science and Technology
  • Jiangsu
    • Hangzhou, Jiangsu, China
      • The Fir St Affiliated Hospital,Zhejiang Univer Sity School of Medicine
    • Suzhou, Jiangsu, China, 215000
      • The First Affiliated Hospital of Suzhou University
  • Jiangxi
    • Nanchang, Jiangxi, China
      • The First Affiliated Hospital of Nanchang University
  • Jilin
    • Changchun, Jilin, China
      • The First Hospital of Jilin University
  • Sichuan
    • Chengdu, Sichuan, China
      • West China Hospital of Sichuan University
  • Tianjin
    • Tianjin, Tianjin, China
      • Hematology Hospital of Chinese Academy of Medical Sciences
  • Xinjiang
    • Xinjiang, Xinjiang, China
      • The First Teaching Hospital of Xinjiang Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Sign the informed consent and agree to comply with the requirements and restrictions set out in the informed consent.
2. At the time of signing the informed consent., the patient must be at least 18 years old, regardless of gender ；
3. Allogeneic hematopoietic stem cell transplantation from any donor source using bone marrow, peripheral blood stem cells, or cord blood.
4. Patients who have received at least 1 line of systemic therapy
5. If the patient is being treated with glucocorticoids or calcineurin inhibitor(CNI), the patient should have received a stable dose of the above treatment for not less than 2 weeks prior to the first use of ABSK021.
6. ECOG (Eastern Cooperative Oncology Group Performance Status) physical strength score 0-2;7. The patient had sufficient organ and bone marrow function within 14 days prior to the first use of ABSK021.

8. For patients with Part A only: antifungal drugs that are currently being used in combination with CYP3A4 potent inhibitors should have been continuously used in accordance with regulations for no less than one week before the first use of ABSK021

Exclusion Criteria:

1. In previous treatment, he received highly selective colony stimulating factor 1 receptor (CSF-1R) targeted therapy, including small molecule or large molecule drugs;
2. A known history of allergy to components of the investigational drug composition ;
3. Patients continued to use CYP3A4 in combination with antifungal agents or in the two weeks prior to the initial administration of ABSK021 Strong inducer;
4. The patient has received more than 5 lines of systemic therapy for cGvHD;
5. The patient presented with aGvHD symptoms without cGvHD symptoms ;
6. Any evidence of potential tumor or recurrence of post-transplant lymphoproliferative disease at the screening stage .
7. There are factors that have been determined by the investigators to have a significant influence on oral drug absorption
8. Present with cholestatic disease, or unresolved hepatic sinus obstruction syndrome/venous obstructive disease;
9. active infection.
10. During the screening period, the investigators judged that the patients had insufficient pulmonary function reserve, with FEV1≤ 39% or pulmonary function classification score of 3;
11. Prior treatment (adverse events did not return to ≤ Grade 2 (CTCAE v5.0);
12. Pregnant or lactating women;
13. Patients who are unable to or disagree with contraception.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ABSK021; Patients will be instructed to take a specified dose of ABSK021 at the same time each day.	Drug: ABSK021; Patients in each phase and dose group will receive continuous treatment with oral administration once a day for 28 days/cycle until conditions for treatment termination are met.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose limited toxicity (DLT) at each dose can assess the incidence of DLT in patients during the observation period of DLT (Part A only)	Number of Participants With Adverse Event (AE), Serious Adverse Event, (SAE) and Laboratory Abnormalities Defined as Dose Limiting Toxicities (DLT);	Starting from the first medication, observe for 31 days
Overall response rate after 6 cycles of treatment	Proportion of participants with CR or PR after 6 cycles of treatment as defined by the 2014 NIH Consensus Development Project on Criteria in cGVHD	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Safety assessments will be carried out during the trial.	Through study completion, an average of 2 years

Collaborators and Investigators

• Sponsor: Abbisko Therapeutics Co, Ltd

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2023
• Primary Completion (Estimated): October 30, 2026
• Study Completion (Estimated): December 30, 2026

Study Registration Dates

• First Submitted: November 28, 2023
• First Submitted That Met QC Criteria: December 15, 2023
• First Posted (Actual): January 2, 2024

Study Record Updates

• Last Update Posted (Actual): August 12, 2024
• Last Update Submitted That Met QC Criteria: August 8, 2024
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Immune System Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchitis; Bronchiolitis Obliterans; Bronchiolitis; Organizing Pneumonia; Graft vs Host Disease; Bronchiolitis Obliterans Syndrome
• Other Study ID Numbers: ABSK021-201

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No