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

August 12, 2026 updated by: Hoffmann-La Roche

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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
42

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

Study Contact Backup

Study Locations

  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia, 2031
        • Sydney Children's Hospital
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Royal Children's Hospital
  • Brazil
    • São Paulo
      • Barretos, São Paulo, Brazil, 14784-400
        • Hospital de Cancer de Barretos
      • São Paulo, São Paulo, Brazil, 04038-030
        • Graacc-Grupo de Apoio ao adolescente e a crianca com cancer
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • CHU Sainte-Justine
  • China
      • Beijing, China, 100045
        • Beijing Children's Hospital, Capital Medical University
      • Shanghai, China, 200092
        • Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
  • Denmark
      • København Ø, Denmark, 2100
        • Rigshospitalet
  • France
      • Lyon, France, 69373
        • Centre Léon Bérard, Institut d?Hémato-Oncologie Pédiatrique
      • Marseille, France, 13385
        • Hôpital de la Timone, Oncologie Pédiatrique
      • Paris, France, 75248
        • Institut Curie - Centre de Lutte Contre le Cancer (CLCC) de Paris
  • Italy
    • Liguria
      • Genoa, Liguria, Italy, 16147
        • Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
    • Lombardy
      • Milan, Lombardy, Italy, 20133
        • Istituto Nazionale Tumori di Milano
    • Piedmont
      • Turin, Piedmont, Italy, 10126
        • Dipartimento di Scienze Pediatriche Adolescenza
  • South Korea
      • Seoul, South Korea, 03080
        • Seoul National University Hospital
      • Seoul, South Korea, 05505
        • Asan Medical Center
      • Seoul, South Korea, 06351
        • Samsung Medical Center
  • Spain
      • Madrid, Spain, 28009
        • Hospital Infantil Universitario Nino Jesus
      • Seville, Spain, 41 41013
        • Hospital Universitario Virgen del Rocio
      • Valencia, Spain, 46026
        • Hospital Universitari i Politecnic La Fe
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Childrens Hospital
      • Newcastle upon Tyne, United Kingdom, NE1 4LP
        • Great North Children's Hospital
  • United States
    • California
      • Palo Alto, California, United States, 94304
        • Lucile Packard Children's Hospital
    • Florida
      • St. Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan, C.S. Mott Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Minnesota
    • New York
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude Children's Research Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Incidence of Participants with Dose-Limited Toxicities (DLTs)
Time Frame: Cycle 1 (cycle length = 28 days)
Cycle 1 (cycle length = 28 days)
Percentage of Participants with Adverse Events
Time Frame: Up to 10 years
Up to 10 years
Plasma Concentration of Alectinib
Time Frame: Up to 10 years
Up to 10 years
Plasma Concentration of Alectinib Metabolite (M4)
Time Frame: Up to 10 years
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
Up to 10 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Confirmed ORR as Determined by the Investigator
Time Frame: Up to 10 years
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)
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)
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
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)
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)
From the first dose of alectinib to the date of death due to any cause (up to 10 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Trials, Hoffmann-LaRoche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 14, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 28, 2032

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 28, 2032

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 10, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 25, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 1, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 14, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 12, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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