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

NCT04774718 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: GO422862020-004239-25
• Study Type: interventional
• Target: 42
• Locations: 12 countries
• Sites: 33

Brief 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.

Conditions

ALK Fusion-positive Solid or CNS Tumors

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (6)

• 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)

Study Arms (1)

ALK-Fusion Positive

EXPERIMENTAL

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

Interventions

Alectinib DRUG

Participants will receive twice-daily alectinib capsules on Days 1-28 of each 28-day cycle

ALK-Fusion Positive

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (33)

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

RECRUITING

University of Michigan, C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

RECRUITING

Children's Minnesota

Minneapolis, Minnesota, 55404, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

ACTIVE NOT RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

St. Jude Children'S Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia

RECRUITING

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

RECRUITING

Hospital de Cancer de Barretos

Barretos, São Paulo, 14784-400, Brazil

RECRUITING

Graacc-Grupo de Apoio ao adolescente e a crianca com cancer

São Paulo, São Paulo, 04038-030, Brazil

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

RECRUITING

Beijing Children's Hospital, Capital Medical University

Beijing, 100045, China

RECRUITING

Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, 200092, China

RECRUITING

Rigshospitalet

København Ø, 2100, Denmark

RECRUITING

Centre Léon Bérard, Institut d?Hémato-Oncologie Pédiatrique

Lyon, 69373, France

RECRUITING

Hôpital de la Timone, Oncologie Pédiatrique

Marseille, 13385, France

RECRUITING

Institut Curie - Centre de Lutte Contre le Cancer (CLCC) de Paris

Paris, 75248, France

RECRUITING

Universitätsklinikum Heidelberg

Heidelberg, 69120, Germany

WITHDRAWN

Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS

Genoa, Liguria, 16147, Italy

ACTIVE NOT RECRUITING

Istituto Nazionale Tumori di Milano

Milan, Lombardy, 20133, Italy

RECRUITING

Dipartimento di Scienze Pediatriche Adolescenza

Turin, Piedmont, 10126, Italy

RECRUITING

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Asan Medical Center

Seoul, 05505, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

Hospital Infantil Universitario Nino Jesus

Madrid, 28009, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, 41 41013, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain

RECRUITING

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

RECRUITING

Royal Manchester Childrens Hospital

Manchester, M13 9WL, United Kingdom

RECRUITING

Great North Children's Hospital

Newcastle upon Tyne, NE1 4LP, United Kingdom

RECRUITING

Royal Marsden Hospital (Sutton)

Sutton, SM2 5PT, United Kingdom

RECRUITING

MeSH Terms

Conditions

Central Nervous System Neoplasms

Interventions

alectinib

Condition Hierarchy (Ancestors)

Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Nervous System Diseases

Study Officials

• Clinical Trials

  Hoffmann-LaRoche

  STUDY DIRECTOR

Central Study Contacts

Reference Study ID Number: GO42286 https://forpatients.roche.com/

CONTACT

888-662-6728; global-roche-genentech-trials@gene.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2021
• First Posted: March 1, 2021
• Study Start: September 14, 2021
• Primary Completion (Estimated): February 28, 2032
• Study Completion (Estimated): February 28, 2032
• Last Updated: May 5, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

GB (4); CA (2); IT (3); US (7); DE (1); BR (2); FR (3); AU (2); CN (2); KR (3); ES (3); DK (1)