Pharmacogenomic Analysis in Pediatric Acute Lymphoblastic Leukemia

• Sponsor: Cipherome, Inc. (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT04770922
• Collaborator: Stanford University (Other)
• Enrollment: 74
• Location: 1
• Anticipated Duration (Months): 17.1
• Patients Per Site Per Month: 4.3

Study Details

Study Description

Brief Summary

This is a retrospective biobank study evaluating the impact of novel genetic variants in a population of 6-mercaptopurine treated pediatric acute lymphoblastic leukemia patients.

Condition or Disease	Intervention/Treatment	Phase
Acute Lymphoblastic Leukemia, Pediatric; Adverse Drug Event

Detailed Description

The study objective is to clinically validate that the presence of recently discovered novel genetic variation adversely affects a population of 6-mercaptopurine treated pediatric acute lymphoblastic leukemia patients using biobank samples.

Study Design

• Study Type: Observational
• Anticipated Enrollment: 74 participants
• Observational Model: Cohort
• Time Perspective: Retrospective
• Official Title: Pharmacogenomic Analysis of 6-mercaptopurine in Pediatric Acute Lymphoblastic Leukemia
• Actual Study Start Date: Feb 23, 2021
• Actual Primary Completion Date: Aug 30, 2021
• Anticipated Study Completion Date: Jul 30, 2022

Arms and Interventions

Arm	Intervention/Treatment
Pediatric ALL patients on 6-mercaptopurine Pediatric ALL patients treated with 6-mercaptopurine who did not experience neutropenia.
Pediatric ALL patients on 6-mercaptopurine with neutropenia Pediatric ALL patients treated with 6-mercaptopurine who experienced neutropenia.

Outcome Measures

Primary Outcome Measures

1. Novel genetic variants impact on 6-mercaptopurine adverse drug reactions [1 year]

   Objective is to clinically validate the presence of novel genetic variants and its impact on adverse drug reactions in a population of pediatric ALL patients treated with 6-MP

Secondary Outcome Measures

1. Evaluating relationship of genetic variants to ancestry [1 year]

   A secondary objective of this study is to compare the impact of the novel genetic variants with other known genetic variants contributing to ADR risk.

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 21 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Pediatric acute lymphoblastic leukemia (ALL) subjects

• Received 6-mercaptopurine

• Available biobank (bone marrow or blood) sample(s) from which deoxyribonucleic acid (DNA) can be extracted

• White blood cell (WBC) levels

Exclusion Criteria:

• Pediatric ALL subjects who did NOT receive 6-mercaptopurine

• No biobank sample

• No WBC level

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Stanford University	Stanford	California	United States	94304

Sponsors and Collaborators

• Cipherome, Inc.
• Stanford University

Investigators

• Principal Investigator: Kathleen M Sakamoto, MD, PhD, Stanford University
• Study Director: Stephanie M Smith, MD, Stanford University

Study Documents (Full-Text)

None provided.

More Information

Publications

• Bhatia S, Landier W, Hageman L, Chen Y, Kim H, Sun CL, Kornegay N, Evans WE, Angiolillo AL, Bostrom B, Casillas J, Lew G, Maloney KW, Mascarenhas L, Ritchey AK, Termuhlen AM, Carroll WL, Wong FL, Relling MV. Systemic Exposure to Thiopurines and Risk of Relapse in Children With Acute Lymphoblastic Leukemia: A Children's Oncology Group Study. JAMA Oncol. 2015 Jun;1(3):287-95. doi: 10.1001/jamaoncol.2015.0245.
• Park Y, Kim H, Choi JY, Yun S, Min BJ, Seo ME, Im HJ, Kang HJ, Kim JH. Star Allele-Based Haplotyping versus Gene-Wise Variant Burden Scoring for Predicting 6-Mercaptopurine Intolerance in Pediatric Acute Lymphoblastic Leukemia Patients. Front Pharmacol. 2019 Jun 11;10:654. doi: 10.3389/fphar.2019.00654. eCollection 2019.

• Responsible Party: Cipherome, Inc.
• ClinicalTrials.gov Identifier: NCT04770922
• Other Study ID Numbers: C04-001
• First Posted: Feb 25, 2021
• Last Update Posted: Mar 9, 2022
• Last Verified: Mar 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Cipherome, Inc.: adverse drug event; acute lymphoblastic leukemia; mercaptopurine; pharmacogenomics
• Additional relevant MeSH terms: Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid; Drug-Related Side Effects and Adverse Reactions

Study Results

No Results Posted as of Mar 9, 2022