Genomics Study in CML Patients With Ponatinib Treatment

NCT06945146 | Completed FDA Drug

• 1 other identifier: KC20MISI0507
• Study Type: observational
• Target: 65
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate whether responsiveness and adverse events (AEs) to second-line or later ponatinib treatment are associated with genetic variations as measured by real-time quantitative polymerase chain reaction (qRT-PCR) and next-generation sequencing (NGS) in patients with CML of any stage who failed prior multiple targeted therapies except ponatinib.

Conditions

Chronic Myelogenous Leukemia, BCR-ABL1 Positive

Keywords

ponatinib; tyrosine kinase inhibitor; resistance; intolerance; genetic variation; next generation sequencing

Outcome Measures

Primary Outcomes (1)

• Dynamics of BCR-ABL1 gene expression by qRT-PCR

  To explore the dynamics of BCR-ABL1 kinetics by Ponatinib

  24 months

Secondary Outcomes (2)

• Type and frequency of novel genetic variations (mutations, gene expressions, CNV, INDEL, etc.)

  24 months

• Cobll1/GCA/novel gene network identification: functional tests using Western blot/Knock-down assay

  24 months

Other Outcomes (1)

• Frequency and severity of skin rash, fever, hypertension, pancreatitis and vascular events as common adverse events and Frequency and severity of rare adverse events

  24 months

Study Arms (1)

CML patients failed to TKIs except Ponatinib

The study will involve 100 patients with BCR-ABL1-positive CML who failed to prior targeted therapy other than ponatinib. Patients receiving ponatinib 45 mg once daily as a second-line or later therapy will be enrolled.

Drug: Ponatinib

Interventions

Ponatinib DRUG

Ponatinib treatment will be initiated per usual treatment procedure at 45 mg once daily p.o., which will be gradually decreased to 30 mg and 15 mg according to the predefined criteria based on responsiveness to treatment and AEs in the course of the treatment.

Also known as: Iclusig

CML patients failed to TKIs except Ponatinib

Eligibility Criteria

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

Study Population

The study will involve 100 patients with BCR-ABL1-positive CML who failed to prior targeted therapy other than ponatinib. Patients receiving ponatinib 45 mg once daily as a second-line or later therapy will be enrolled.

You may qualify if:

• Patients who are willing to and capable of providing informed consent
• Adults with BCR-ABL1-positive CML
• Males and females aged 18 years and above
• Adequate organ function
• Subjects who were resistant or intolerant to prior targeted therapy other than ponatinib and with an indication for ponatinib treatment according to the acceptance criteria by the Ministry of Food and Drug Safety (MFDS)
• Women of childbearing potential (WOCBP) should have a negative serum or urine pregnancy test (with a sensitivity of at least 25 IU/L or equivalent to HCG) within 24 hours before initiating ponatinib treatment
• Female subjects who are not breastfeeding

You may not qualify if:

• Patients who were previously treated with ponatinib
• Patients aged below 18 years of age
• Diagnosis of severe comorbidity at baseline
• Any other cancers within 3 years

Sponsors & Collaborators

• Dong-Wook Kim: lead
• Ulsan National Institute of Science and Technology: collaborator
• Takeda: collaborator

Study Sites (1)

Seoul St. Mary's Hospital

Seoul, 137-701, South Korea

Location

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral blood and bone marrow

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Interventions

ponatinib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Dong-Wook Kim, MD, PhD

  Eulji University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: professor

Study Record Dates

• First Submitted: April 18, 2025
• First Posted: April 25, 2025
• Study Start: September 1, 2020
• Primary Completion: August 31, 2024
• Study Completion: September 25, 2024
• Last Updated: April 25, 2025

Record last verified: 2024-12

Locations

KR (1)