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Evaluating Alectinib for Children with ALK Fusion-Positive 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

Phase1; Phase2 Interventional Hoffmann-La Roche · NCT04774718

This study is testing a drug called alectinib to see if it can help children and teens with certain tumors that have not responded to other treatments.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment42 (estimated)
AgesN/A to 17 Years
SexAll
SponsorHoffmann-La Roche Industry-sponsored
Drugs / interventionsalectinib
Locations33 sites (Palo Alto, California and 32 other locations)
Trial IDNCT04774718 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety, pharmacokinetics, and efficacy of alectinib in pediatric patients diagnosed with ALK fusion-positive solid or CNS tumors. It targets children and adolescents whose previous treatments have failed or who lack satisfactory standard treatment options. The study will assess the drug's effectiveness and safety through a series of evaluations and measurements based on established criteria for tumor response.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents with confirmed ALK fusion-positive solid or CNS tumors who have not responded to prior treatments.

Not a fit: Patients with tumors that do not have ALK gene fusions or those who have not undergone prior treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for children with difficult-to-treat ALK fusion-positive tumors.

How similar studies have performed: Other studies have shown promising results with targeted therapies for ALK fusion-positive tumors, indicating potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Palo Alto, California and 32 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions ALK Fusion-positive Solid or CNS Tumors
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.