A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors

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

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.

Study Overview

• Status: Recruiting
• Conditions: ALK Fusion-positive Solid or CNS Tumors
• Intervention / Treatment: Drug: Alectinib

• Study Type: Interventional
• Enrollment (Estimated): 42
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Reference Study ID Number: GO42286 https://forpatients.roche.com/; Phone Number: 888-662-6728; Email: global-roche-genentech-trials@gene.com
• Study Contact Backup: Name: Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry

Study Locations

• Australia
  • New South Wales
    • Randwick, New South Wales, Australia, 2031
      • Recruiting
      • Sydney Children's Hospital
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • Recruiting
      • Royal Children's Hospital
• Brazil
  • São Paulo
    • Barretos, São Paulo, Brazil, 14784-400
      • Recruiting
      • Hospital de Cancer de Barretos
    • São Paulo, São Paulo, Brazil, 04038-030
      • Recruiting
      • Graacc-Grupo de Apoio ao adolescente e a crianca com cancer
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • Recruiting
      • The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • Recruiting
      • CHU Sainte-Justine
• China
  • Beijing, China, 100045
    • Recruiting
    • Beijing Children's Hospital, Capital Medical University
  • Shanghai, China, 200092
    • Recruiting
    • Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
• Denmark
  • København Ø, Denmark, 2100
    • Recruiting
    • Rigshospitalet
• France
  • Lyon, France, 69373
    • Recruiting
    • Centre Léon Bérard, Institut d?Hémato-Oncologie Pédiatrique
  • Marseille, France, 13385
    • Recruiting
    • Hôpital de la Timone, Oncologie Pédiatrique
  • Paris, France, 75248
    • Recruiting
    • Institut Curie - Centre de Lutte Contre le Cancer (CLCC) de Paris
• Germany
  • Heidelberg, Germany, 69120
    • Withdrawn
    • Universitätsklinikum Heidelberg
• Italy
  • Liguria
    • Genoa, Liguria, Italy, 16147
      • Active, not recruiting
      • Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
  • Lombardy
    • Milan, Lombardy, Italy, 20133
      • Recruiting
      • Istituto Nazionale Tumori di Milano
  • Piedmont
    • Turin, Piedmont, Italy, 10126
      • Recruiting
      • Dipartimento di Scienze Pediatriche Adolescenza
• South Korea
  • Seoul, South Korea, 05505
    • Recruiting
    • Asan Medical Center
  • Seoul, South Korea, 03080
    • Recruiting
    • Seoul National University Hospital
  • Seoul, South Korea, 06351
    • Recruiting
    • Samsung Medical Center
• Spain
  • Madrid, Spain, 28009
    • Recruiting
    • Hospital Infantil Universitario Nino Jesus
  • Seville, Spain, 41 41013
    • Recruiting
    • Hospital Universitario Virgen del Rocio
  • Valencia, Spain, 46026
    • Recruiting
    • Hospital Universitari i Politecnic La Fe
• United Kingdom
  • London, United Kingdom, WC1N 3JH
    • Recruiting
    • Great Ormond Street Hospital
  • Manchester, United Kingdom, M13 9WL
    • Recruiting
    • Royal Manchester Childrens Hospital
  • Newcastle upon Tyne, United Kingdom, NE1 4LP
    • Recruiting
    • Great North Children's Hospital
  • Sutton, United Kingdom, SM2 5PT
    • Withdrawn
    • Royal Marsden Hospital (Sutton)
• United States
  • California
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Lucile Packard Children's Hospital
  • Florida
    • St. Petersburg, Florida, United States, 33701
      • Recruiting
      • Johns Hopkins All Children's Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Recruiting
      • University of Michigan, C.S. Mott Children's Hospital
  • Minnesota
    • Minneapolis, Minnesota, United States, 55404
      • Recruiting
      • Children's Minnesota
  • New York
    • New York, New York, United States, 10065
      • Active, not recruiting
      • Memorial Sloan Kettering Cancer Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Recruiting
      • Cincinnati Children's Hospital Medical Center
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Recruiting
      • St. Jude Children's Research Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria

• Histologically confirmed diagnosis of CNS or solid tumors with documented evidence of ALK gene fusions as assessed centrally through the use of the investigational F1CDx assay or based on pre-existing NGS test results
• Disease status: prior treatment proven to be ineffective (i.e. relapsed or refractory), or for whom there is no satisfactory standard treatment available. Disease should be measurable and evaluable as defined by Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1, or Response Assessment in Neuro-oncology criteria (RANO) +/- bone marrow criteria for primary CNS tumors or International Neuroblastoma Response Criteria (INRC)
• Available tumor tissue for submission to the Sponsor from active disease, obtained subsequent to last anti-cancer therapy regimen administered and obtained prior to study enrollment (preferred option), or archival tumor tissue from original diagnosis, or willingness to undergo a core or excisional biopsy sample collection prior to enrollment
• For participants < 16 years old, Lansky Performance Status >/= 50%
• For participants >/= 16 years old, Karnofsky Performance Status >/= 50%
• Adequate bone marrow function as defined by the protocol within at least 28 days prior to initiation of study drug
• Participant and/or caregiver willingness and ability to complete clinical outcome assessments throughout the study using either electronic, paper, or interviewer methods
• For females of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception, and agreement to refrain from donating eggs, as defined by the protocol
• For males who are not surgically sterile: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception, and agreement to refrain from donating sperm, as defined by the protocol

Exclusion Criteria

• Medical history of: prior use of ALK inhibitors; diagnosis of Anaplastic Large Cell Lymphoma (ALCL); any gastrointestinal disorder that may affect absorption of oral medications, such as mal-absorption syndrome or status post-major bowel resection; history of organ transplant; stem cell infusions as defined by the protocol
• Substance abuse within 12 months prior to screening
• Familial or personal history of congenital bone disorders, bone metabolism alterations, or osteopenia
• Treatment with investigational therapy 28 days prior to initiation of study drug
• Liver or kidney disease as defined by the protocol
• National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 grade >/=3 toxicities attributed to any prior therapy such as radiotherapy (excluding alopecia), which have not shown improvement and are strictly considered to interfere with alectinib
• Co-administration of anti-cancer therapies other than those administered in this study
• Active hepatitis B or C virus (HBV, HBC), or known HIV-positivity or AIDS-related illness
• Any clinically significant concomitant disease or condition that could interfere with, or for which the treatment might interfere with, the conduct of the study or the absorption of oral medications or that would, in the opinion of the Principal Investigator, pose an unacceptable risk to the participant in this study
• Any psychological, familial, sociological, or geographical condition potentially hampering compliance with the study protocol requirements and/or follow-up procedures; such conditions should be discussed with the participant before trial entry
• Planned procedure or surgery during the study except as permitted treatment as defined by the protocol
• Infection considered by the investigator to be clinically uncontrolled or of unacceptable risk to the participant upon induction of neutropenia, including participants who are, or should be, on antimicrobial agents for the treatment as active infection
• Pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the final dose of alectinib

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: ALK-Fusion Positive; Part 1 is a dose-confirmation phase to confirm the recommended phase 2 dose (RP2D). In Parts 2 and 3, participants will receive alectinib at the RP2D on Days 1-28 of each 28-day cycle	Drug: Alectinib; Participants will receive twice-daily alectinib capsules on Days 1-28 of each 28-day cycle

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of Participants with Dose-Limited Toxicities (DLTs)	Cycle 1 (cycle length = 28 days)
Percentage of Participants with Adverse Events	Up to 10 years
Plasma Concentration of Alectinib	Up to 10 years
Plasma Concentration of Alectinib Metabolite (M4)	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)	Up to 10 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Confirmed ORR as Determined by the Investigator	Up to 10 years
Duration of Response (DOR) as Determined by BICR and the Investigator	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	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	6 months after the first dose of alectinib
Progression-Free Survival (PFS) as Determined by BICR and the Investigator	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)	From the first dose of alectinib to the date of death due to any cause (up to 10 years)

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche
• Investigators: Study Director: Clinical Trials, Hoffmann-LaRoche

Publications and helpful links

General Publications

• 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.

Helpful Links

• Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.

Study record dates

Study Major Dates

• Study Start (Actual): September 14, 2021
• Primary Completion (Estimated): February 28, 2032
• Study Completion (Estimated): February 28, 2032

Study Registration Dates

• First Submitted: February 10, 2021
• First Submitted That Met QC Criteria: February 25, 2021
• First Posted (Actual): March 1, 2021

Study Record Updates

• Last Update Posted (Actual): June 4, 2026
• Last Update Submitted That Met QC Criteria: June 3, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Nervous System Neoplasms; Central Nervous System Neoplasms; Tyrosine Kinase Inhibitors; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; alectinib
• Other Study ID Numbers: GO42286; 2020-004239-25 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing

Drug and device information, study documents

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