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

Phase 1

Recruiting

• Conditions: ALK Fusion-positive Solid or CNS Tumors
• Interventions: Drug: Alectinib

• Registration Number: NCT04774718
• Lead Sponsor: Hoffmann-La Roche

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-02-10
• Study First Submitted QC: 2021-02-25
• Study First Posted: 2021-03-01
• Study Start: 2021-09-14
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-01
• Last Update Posted: 2025-05-02
• Primary Completion: 2028-02-28
• Study Completion: 2032-02-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ALK-Fusion Positive	Alectinib	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

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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)
Confirmed ORR as Determined by the Investigator	Up to 10 years
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)
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)
Clinical Benefit Rate (CBR) as Determined by BICR and the Investigator	6 months after the first dose of alectinib

Trial Locations

Locations (33)

Lucile Packard Children's Hospital; 🇺🇸 Palo Alto, California, United States

Children's Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

Hospital de Cancer de Barretos; 🇧🇷 Barretos, São Paulo, Brazil

Rigshospitalet; 🇩🇰 København Ø, Denmark

Institut Curie - Centre de Lutte Contre le Cancer (CLCC) de Paris; 🇫🇷 Paris, France

Universitätsklinikum Heidelberg; 🇩🇪 Heidelberg, Germany

Istituto Nazionale Tumori di Milano; 🇮🇹 Milano, Lombardia, Italy

Dipartimento di Scienze Pediatriche Adolescenza; 🇮🇹 Torino, Piemonte, Italy

Hospital Universitario Virgen del Rocio; 🇪🇸 Sevilla, Spain

Johns Hopkins All Children's Hospital; 🇺🇸 Saint Petersburg, Florida, United States

University of Michigan, C.S. Mott Children's Hospital; 🇺🇸 Ann Arbor, Michigan, United States

Memorial Sloan Kettering Cancer Center; 🇺🇸 New York, New York, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

St. Jude Children'S Research Hospital; 🇺🇸 Memphis, Tennessee, United States

Sydney Children's Hospital; 🇦🇺 Randwick, New South Wales, Australia

Royal Children's Hospital; 🇦🇺 Parkville, Victoria, Australia

Graacc-Grupo de Apoio ao adolescente e a crianca com cancer; 🇧🇷 Sao Paulo, São Paulo, Brazil

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

CHU Sainte-Justine; 🇨🇦 Montreal, Quebec, Canada

Beijing Children's Hospital, Capital Medical University; 🇨🇳 Beijing, China

Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine; 🇨🇳 Shanghai, China

Centre Léon Bérard, Institut d?Hémato-Oncologie Pédiatrique; 🇫🇷 Lyon, France

Hôpital de la Timone, Oncologie Pédiatrique; 🇫🇷 Marseille, France

Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS; 🇮🇹 Genova, Liguria, Italy

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of

Asan Medical Center; 🇰🇷 Seoul, Korea, Republic of

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of

Hospital Infantil Universitario Nino Jesus; 🇪🇸 Madrid, Spain

Hospital Universitari i Politecnic La Fe; 🇪🇸 Valencia, Spain

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom

Royal Manchester Childrens Hospital; 🇬🇧 Manchester, United Kingdom

Great North Children's Hospital; 🇬🇧 Newcastle upon Tyne, United Kingdom

Royal Marsden Hospital (Sutton); 🇬🇧 Sutton, United Kingdom