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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04774718




Registration number
NCT04774718
Ethics application status
Date submitted
10/02/2021
Date registered
1/03/2021
Date last updated
3/10/2024

Titles & IDs
Public title
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors
Scientific title
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
Secondary ID [1] 0 0
2020-004239-25
Secondary ID [2] 0 0
GO42286
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
ALK Fusion-positive Solid or CNS Tumors 0 0
Condition category
Condition code
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Children's - Brain

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Alectinib

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


Treatment: Drugs: Alectinib
Participants will receive twice-daily alectinib capsules on Days 1-28 of each 28-day cycle
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Participants with Dose-Limited Toxicities (DLTs)
Timepoint [1] 0 0
Cycle 1 (cycle length = 28 days)
Primary outcome [2] 0 0
Percentage of Participants with Adverse Events
Timepoint [2] 0 0
Up to 10 years
Primary outcome [3] 0 0
Plasma Concentration of Alectinib
Timepoint [3] 0 0
Up to 10 years
Primary outcome [4] 0 0
Plasma Concentration of Alectinib Metabolite (M4)
Timepoint [4] 0 0
Up to 10 years
Primary outcome [5] 0 0
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)
Timepoint [5] 0 0
Up to 10 years
Secondary outcome [1] 0 0
Confirmed ORR as Determined by the Investigator
Timepoint [1] 0 0
Up to 10 years
Secondary outcome [2] 0 0
Duration of Response (DOR) as Determined by BICR and the Investigator
Timepoint [2] 0 0
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)
Secondary outcome [3] 0 0
Time to Response (TTR) as Determined by BICR and the Investigator
Timepoint [3] 0 0
From the first dose of alectinib to the first documentation of objective response (CR or PR) (up to 10 years)
Secondary outcome [4] 0 0
Clinical Benefit Rate (CBR) as Determined by BICR and the Investigator
Timepoint [4] 0 0
6 months after the first dose of alectinib
Secondary outcome [5] 0 0
Progression-Free Survival (PFS) as Determined by BICR and the Investigator
Timepoint [5] 0 0
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)
Secondary outcome [6] 0 0
Overall Survival (OS)
Timepoint [6] 0 0
From the first dose of alectinib to the date of death due to any cause (up to 10 years)

Eligibility
Key inclusion 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 regiment administered and obtained prior to study enrollment, 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
Minimum age
No limit
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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 design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/09/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
3/07/2030
Actual
Sample size
Target
42
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
Brazil
State/province [7] 0 0
SP
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
China
State/province [10] 0 0
Beijing City
Country [11] 0 0
China
State/province [11] 0 0
Shanghai
Country [12] 0 0
Denmark
State/province [12] 0 0
København Ø
Country [13] 0 0
France
State/province [13] 0 0
Lyon
Country [14] 0 0
France
State/province [14] 0 0
Marseille
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
Germany
State/province [16] 0 0
Heidelberg
Country [17] 0 0
Italy
State/province [17] 0 0
Liguria
Country [18] 0 0
Italy
State/province [18] 0 0
Lombardia
Country [19] 0 0
Italy
State/province [19] 0 0
Piemonte
Country [20] 0 0
Korea, Republic of
State/province [20] 0 0
Seoul
Country [21] 0 0
Spain
State/province [21] 0 0
Madrid
Country [22] 0 0
Spain
State/province [22] 0 0
Sevilla
Country [23] 0 0
Spain
State/province [23] 0 0
Valencia
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Manchester
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Newcastle upon Tyne
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Sutton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Hoffmann-La Roche
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT04774718
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-LaRoche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Reference Study ID Number: GO42286 https://forpatients.roche.com/
Address 0 0
Country 0 0
Phone 0 0
888-662-6728
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04774718