ALK Fusion-positive Solid or CNS TumorsPhase 1/2NCT04774718
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors
A Phase 1/2 study testing Alectinib in people with ALK Fusion-positive Solid or CNS Tumors, run by Hoffmann-La Roche at 31 sites in 11 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- ALK Fusion-positive Solid or CNS Tumors
- What is being tested?
- Alectinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 31 sitesin 11 countries
- Study dates
- 14 Sep 2021to 28 Feb 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
31 sites in 11 countries.
31 sites in 11 countries
United States7 sites
University of Michigan, C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
St. Jude Children'S Research Hospital
Memphis, Tennessee, 38105
Children's Minnesota
Minneapolis, Minnesota, 55404
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Lucile Packard Children's Hospital
Palo Alto, California, 94304
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
France3 sites
Centre Léon Bérard, Institut d?Hémato-Oncologie Pédiatrique
Lyon, 69373
Hôpital de la Timone, Oncologie Pédiatrique
Marseille, 13385
Institut Curie - Centre de Lutte Contre le Cancer (CLCC) de Paris
Paris, 75248
Italy3 sites
Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
Genoa, Liguria, 16147
Istituto Nazionale Tumori di Milano
Milan, Lombardy, 20133
Dipartimento di Scienze Pediatriche Adolescenza
Turin, Piedmont, 10126
South Korea3 sites
Seoul National University Hospital
Seoul, 03080
Asan Medical Center
Seoul, 05505
Samsung Medical Center
Seoul, 06351
Spain3 sites
Hospital Infantil Universitario Nino Jesus
Madrid, 28009
Hospital Universitario Virgen del Rocio
Seville, 41 41013
Hospital Universitari i Politecnic La Fe
Valencia, 46026
United Kingdom3 sites
Great Ormond Street Hospital
London, WC1N 3JH
Royal Manchester Childrens Hospital
Manchester, M13 9WL
Great North Children's Hospital
Newcastle upon Tyne, NE1 4LP
Australia2 sites
Royal Children's Hospital
Parkville, Victoria, 3052
Sydney Children's Hospital
Randwick, New South Wales, 2031
Brazil2 sites
Hospital de Cancer de Barretos
Barretos, São Paulo, 14784-400
Graacc-Grupo de Apoio ao adolescente e a crianca com cancer
São Paulo, São Paulo, 04038-030
Canada2 sites
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
China2 sites
Beijing Children's Hospital, Capital Medical University
Beijing, 100045
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, 200092
About this study
Official 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
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
- Being tested
- Alectinib
- Medical subject headings
- Central Nervous System Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlectinibDrug
Participants will receive twice-daily alectinib capsules on Days 1-28 of each 28-day cycle
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Alectinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Participants with Dose-Limited Toxicities (DLTs)
Measured: Cycle 1 (cycle length = 28 days)
Percentage of Participants with Adverse Events
Measured: Up to 10 years
Plasma Concentration of Alectinib
Measured: Up to 10 years
Plasma Concentration of Alectinib Metabolite (M4)
Measured: 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)
Measured: Up to 10 years
Secondary outcomes (6)
Confirmed ORR as Determined by the Investigator
Measured: Up to 10 years
Duration of Response (DOR) as Determined by BICR and the Investigator
Measured: 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
Measured: 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
Measured: 6 months after the first dose of alectinib
Progression-Free Survival (PFS) as Determined by BICR and the Investigator
Measured: 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)
Measured: From the first dose of alectinib to the date of death due to any cause (up to 10 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2021
- Study start14 Sep 2021
- Primary completion28 Feb 2032 (estimated)
- Study completion28 Feb 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ALK Fusion-positive Solid or CNS TumorsStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-LaRoche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04774718
- Sponsor's protocol ID
- GO42286
- Other IDs
- 2020-004239-25
- Status last verified
- Aug 2026
- First submitted
- 10 Feb 2021
- Last updated
- 14 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
Bagchi A, Chiang J, Pinto S, Dhanda S, Gajjar A. Infant-Type Hemispheric Gliomas: A Review of Clinical, Radiologic, Histopathologic, and Molecular Features. J Natl Compr Canc Netw. 2025 Nov;23(11):e257064. doi: 10.6004/jnccn.2025.7064.
PubMed 41213248
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT04774718
- Acronym / sponsor ID
- GO42286
- Conditions
- ALK Fusion-positive Solid or CNS Tumors
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 14 Sep 2021 (actual)
- Primary completion
- 28 Feb 2032 (estimated)
- Study completion
- 28 Feb 2032 (estimated)
- First posted
- 1 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALK-Fusion Positive | Experimental | Drug: AlectinibPart 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 |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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%
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AlectinibParticipants will receive twice-daily alectinib capsules on Days 1-28 of each 28-day cycle | Drug | ALK-Fusion Positive |
Primary outcome
Incidence of Participants with Dose-Limited Toxicities (DLTs)
Time frame: Cycle 1 (cycle length = 28 days)
Percentage of Participants with Adverse Events
Time frame: Up to 10 years
Plasma Concentration of Alectinib
Time frame: Up to 10 years
Plasma Concentration of Alectinib Metabolite (M4)
Time frame: 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
6 secondary outcomes
Confirmed ORR as Determined by the Investigator
Time frame: 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)
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)
Clinical Benefit Rate (CBR) as Determined by BICR and the Investigator
Time frame: 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)
Overall Survival (OS)
Time frame: From the first dose of alectinib to the date of death due to any cause (up to 10 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2026.
- Derived from this study: PubMed 41213248Bagchi A, Chiang J, Pinto S, Dhanda S, Gajjar A. Infant-Type Hemispheric Gliomas: A Review of Clinical, Radiologic, Histopathologic, and Molecular Features. J Natl Compr Canc Netw. 2025 Nov;23(11):e257064. doi: 10.6004/jnccn.2025.7064.
- Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
Related trials for ALK Fusion-positive Solid or CNS Tumors
Enrolling by invitationObservational study
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Oncogene-addicted Non Small Cell Lung Cancer, EGFR Mutation
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-LaRoche
31 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Alectinib in people with ALK Fusion-positive Solid or CNS Tumors, run by Hoffmann-La Roche at 31 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, France, Italy, South Korea, Spain and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Participants with Dose-Limited Toxicities (DLTs) (time frame: Cycle 1 (cycle length = 28 days)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04774718, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2026.