Olverembatinib for FGFR1-rearranged Neoplasms

Acute Leukemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05521204
• Other study ID #: SZ-FGFR1
• Status: Recruiting
• Phase: Phase 2
• Start date: September 1, 2022
• Est. completion date: August 31, 2027

Study information

• Verified date: August 2022
• Source: The First Affiliated Hospital of Soochow University
• Contact: Suning Chen
• Phone: +86-13814881746
• Email: chensuning[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: August 31, 2027
• Est. primary completion date: August 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Acute Leukemia
• Myeloproliferative Disorders
• Myeloproliferative Neoplasm
• Neoplasms

Intervention

Drug:
• Olverembatinib
Given PO

Locations

Country	Name	City	State
China	First Affiliated Hospital of Soochow University	Suzhou	Jiangsu

Sponsors (1)

Lead Sponsor	Collaborator
The First Affiliated Hospital of Soochow University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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.
Secondary	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.
Secondary	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.