A Phase I/II, Open-Label, Multicenter, Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors for Whom Prior Treatment Has Proven to be Ineffective or for Whom There is No Satisfactory Treatment Available
Summary
This study will evaluate the safety, pharmacokinetics, and efficacy of alectinib in children and adolescents with ALK fusion-positive solid or CNS tumors for whom prior treatment has proven to be ineffective or for whom there is no satisfactory standard treatment available.
Arms & interventions
- DrugAlectinib
Participants will receive twice-daily alectinib capsules on Days 1-28 of each 28-day cycle
Outcome measures
Primary
Incidence of Participants with Dose-Limited Toxicities (DLTs)
Time frame: Cycle 1 (cycle length = 28 days)
Percentage of Participants with Adverse Events
Time frame: Up to 10 years
Plasma Concentration of Alectinib
Time frame: Up to 10 years
Plasma Concentration of Alectinib Metabolite (M4)
Time frame: Up to 10 years
Confirmed Objective Response Rate (ORR): Defined as the Proportion of Participants with Complete Response (CR) or Partial Response (PR) on two Consecutive Occasions >/= 4 Weeks Apart, as Determined by Blinded Independent Central Review (BICR)
Time frame: Up to 10 years
Secondary
Confirmed ORR as Determined by the Investigator
Time frame: Up to 10 years
Duration of Response (DOR) as Determined by BICR and the Investigator
Time frame: From the first occurrence of a documented objective response (CR or PR) to disease progression or death from any cause, whichever occurs first (up to 10 years)
Time to Response (TTR) as Determined by BICR and the Investigator
Time frame: From the first dose of alectinib to the first documentation of objective response (CR or PR) (up to 10 years)
Clinical Benefit Rate (CBR) as Determined by BICR and the Investigator
Time frame: 6 months after the first dose of alectinib
Progression-Free Survival (PFS) as Determined by BICR and the Investigator
Time frame: From the first dose of alectinib to the first occurrence of disease progression or death from any cause, whichever occurs first (up to 10 years)
Overall Survival (OS)
Time frame: From the first dose of alectinib to the date of death due to any cause (up to 10 years)
Eligibility criteria
Study locations (7)
Lucile Packard Children's Hospital
Palo Alto, California, 94304
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
University of Michigan, C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109
Children's Minnesota
Minneapolis, Minnesota, 55404
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
St. Jude Children'S Research Hospital
Memphis, Tennessee, 38105
References
- Bagchi A, Chiang J, Pinto S, Dhanda S, Gajjar A. Infant-Type Hemispheric Gliomas: A Review of Clinical, Radiologic, Histopathologic, and Molecular Features. J Natl Compr Canc Netw. 2025 Nov;23(11):e257064. doi: 10.6004/jnccn.2025.7064.(PubMed)