Olverembatinib for FGFR1-rearranged Neoplasms

A Study of Olverembatinib in the Treatment of Myeloid/Lymphoid Tumors With FGFR1 Rearrangement

FGFR1-rearranged myeloid/lymphoid neoplasms are a rare hematologic malignancy with very poor outcome despite intensive chemotherapy. The only curative option is thought to be allogeneic hematopoietic stem cell transplantation (HSCT) in remission.

This phase II study is aimed to evaluate the efficacy of Olverembatinib, consolidated with HSCT in the treatment of FGFR1-rearranged myeloid/lymphoid neoplasm.

Study Overview

• Status: Recruiting
• Conditions: Acute Leukemia; Myeloproliferative Neoplasm
• Intervention / Treatment: Drug: Olverembatinib

Detailed Description

FGFR1-rearranged myeloid/lymphoid neoplasms are a rare and highly heterogeneous hematological malignancy, mainly manifested as myeloproliferative neoplasms (MPNs) or acute leukemia, including T cells or B cells Cell lymphoblastic leukemia/lymphoma (T-cell or B-cell-ALL/LBL), acute myeloid leukemia (AML) and mixed cell leukemia (MPAL).

To date, there is no standard treatment. Conventional chemotherapy is frequently ineffective. The only curative option is thought to be allogeneic HSCT at present, TKIs may offer a therapeutic alternative in patients not eligible for allogeneic HSCT or to bridge the time between diagnosis and allogeneic HSCT.

Third-generation TKIs Olverembatinib is a pan- FGFR1 kinase inhibitor, and is supposed to be effective to achieve bone marrow remission in FGFR1-rearranged myeloid/lymphoid neoplasms.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Suning Chen; Phone Number: +86-13814881746; Email: chensuning@sina.com

Study Locations

• China
  • Jiangsu
    • Suzhou, Jiangsu, China, 215000
      • Recruiting
      • First Affiliated Hospital of Soochow University
      • Contact: Suning Chen, PhD; Phone Number: +8613814881746; Email: chensuning@sina.com

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Patients with newly diagnosed, progressed or relapsed myeloid/lymphoid neoplasms with FGFR1 rearrangement according to the WHO-2016 diagnostic criteria. Patients who have received allogeneic hematopoietic stem cell transplantation or ponatinib should be excluded.
2. ECOG score: MPNs patients, 0-3 points; AL patients, 0-2 points.
3. Expected survival period ≥12 weeks.
4. Willingness and ability to comply with study procedures and follow-up examination.

Exclusion Criteria:

1. Patients who have received allogeneic hematopoietic stem cell or ponatinib.
2. Human immunodeficiency virus (HIV) infection, or chronic infection with hepatitis B virus (HBsAg positive) or hepatitis C virus (anti-HCV positive).
3. Patients who are pregnant, planning to become pregnant or breastfeeding.
4. Patients who may not be able to complete all study visits or procedures required by the study protocol, including follow-up visits, and/or fail to comply with all required study procedures.
5. Patients who suffer from any condition or illness that, in the opinion of the investigator, would compromise patient safety or interfere with the evaluation of the safety of the research drug.

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: Treatment Group	Drug: Olverembatinib; Given PO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate	The proportion of participants who achieve Overall Response (ORR) based on response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement	Assessed at protocol-defined timepoints through end of study, up to approximately 48 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Event-free survival (PFS)	The Kaplan-Meier method will be used to assess EFS probabilities.	From the first day of treatment until any failure (resistant disease, relapse, or death), assessed up to 2 to 4 years.
Overall survival (OS)	The Kaplan-Meier method will be used to assess OS probabilities.	From the first day of treatment to time of death from any cause, assessed up 2 to 4 years.
Incidence of adverse events (AEs)	Will be graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0. The proportion of patients with AEs will be estimated, along with the Bayesian 95% credible interval.	Up to approximately 2 to 4 years.

Collaborators and Investigators

• Sponsor: The First Affiliated Hospital of Soochow University
• Investigators: Principal Investigator: Suning Chen, First Affiliated Hospital of Soochow University

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2022
• Primary Completion (Anticipated): August 31, 2024
• Study Completion (Anticipated): August 31, 2027

Study Registration Dates

• First Submitted: August 27, 2022
• First Submitted That Met QC Criteria: August 27, 2022
• First Posted (Actual): August 30, 2022

Study Record Updates

• Last Update Posted (Actual): August 30, 2022
• Last Update Submitted That Met QC Criteria: August 27, 2022
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Neoplasms; Myeloproliferative Disorders
• Other Study ID Numbers: SZ-FGFR1

Drug and device information, study documents

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