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NCT03213678

Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)

Completed Phase 2 Results posted Last updated 2 April 2026
What this trial tests

Phase 2 trial testing Biospecimen Collection in Advanced Malignant Solid Neoplasm in 18 participants. Completed in 31 December 2024.

Timeline
28 November 2017
Primary endpoint
30 September 2023
31 December 2024

Quick facts

Lead sponsorNational Cancer Institute (NCI)
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment18
Start date28 November 2017
Primary completion30 September 2023
Estimated completion31 December 2024
Sites127 locations across Puerto Rico, United States

Drugs / interventions tested

Conditions studied

Sponsor

National Cancer Institute (NCI)

Who can join

Adults 12 Months to 21, any sex, with Advanced Malignant Solid Neoplasm or Ann Arbor Stage III Non-Hodgkin Lymphoma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Objective Response Rate Primary · Up to 2 years from study entry

A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

GroupValue95% CI
Treatment (Samotolisib)00 – 0
Percentage of Patients Experiencing Grade 3 or 4 Adverse Events Secondary · Up to 2 years from study entry

Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0

GroupValue95% CI
Treatment (Samotolisib)82.456.6 – 96.2
Progression Free Survival (PFS) Secondary · Up to 3 months from study entry

The Kaplan-Meier method will be used to estimate the 3-month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact.

GroupValue95% CI
Treatment (Samotolisib)122 – 31
Pharmacokinetic (PK) of Samotolisib, Area Under the Curve (AUC). Secondary · Up to day 15 of cycle 1

The mean (sd) of the AUC

GroupValue95% CI
Treatment (Samotolisib)2323± 1006

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment (Samotolisib)
Serious: 9/17 (53%)
Deaths: 14/18

Serious adverse events (14 terms)

ReactionSystemTreatment (Samotolisib)
Death NOSGeneral disorders
Lung infectionInfections and infestations
Pain in extremityMusculoskeletal and connective tissue disorders
Heart failureCardiac disorders
Mucositis oralGastrointestinal disorders
Disease progressionGeneral disorders
PainGeneral disorders
Infections and infestations - Other, specifyInfections and infestations
Muscle weakness right-sidedNervous system disorders
HematuriaRenal and urinary disorders
HypoxiaRespiratory, thoracic and mediastinal disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
PneumonitisRespiratory, thoracic and mediastinal disorders
HypotensionVascular disorders
Other adverse events (91 terms — click to expand)

ReactionSystemTreatment (Samotolisib)
FatigueGeneral disorders
AnemiaBlood and lymphatic system disorders
NauseaGastrointestinal disorders
HyperglycemiaMetabolism and nutrition disorders
Alanine aminotransferase increasedInvestigations
Lymphocyte count decreasedInvestigations
HyponatremiaMetabolism and nutrition disorders
ConstipationGastrointestinal disorders
FeverGeneral disorders
White blood cell decreasedInvestigations
HeadacheNervous system disorders
CoughRespiratory, thoracic and mediastinal disorders
VomitingGastrointestinal disorders
Aspartate aminotransferase increasedInvestigations
AnorexiaMetabolism and nutrition disorders
HypophosphatemiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
DiarrheaGastrointestinal disorders
Creatinine increasedInvestigations
Neutrophil count decreasedInvestigations
Platelet count decreasedInvestigations
Weight lossInvestigations
HypoalbuminemiaMetabolism and nutrition disorders
HypomagnesemiaMetabolism and nutrition disorders
Sore throatRespiratory, thoracic and mediastinal disorders
Blurred visionEye disorders
Abdominal painGastrointestinal disorders
Alkaline phosphatase increasedInvestigations
Blood bilirubin increasedInvestigations
Cholesterol highInvestigations
DehydrationMetabolism and nutrition disorders
HypokalemiaMetabolism and nutrition disorders
MyalgiaMusculoskeletal and connective tissue disorders
Sinus tachycardiaCardiac disorders
Oral painGastrointestinal disorders
Upper respiratory infectionInfections and infestations
Urinary tract infectionInfections and infestations
HypermagnesemiaMetabolism and nutrition disorders
HypertriglyceridemiaMetabolism and nutrition disorders
HypocalcemiaMetabolism and nutrition disorders

Most-reported serious reactions: Death NOS, Lung infection, Pain in extremity, Heart failure, Mucositis oral, Disease progression, Pain, Infections and infestations - Other, specify.

Data from ClinicalTrials.gov NCT03213678 adverse events section.

Sponsor's own description

This phase II Pediatric MATCH trial studies how well samotolisib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with TSC or PI3K/MTOR mutations that have spread to other places in the body (metastatic) and have come back (recurrent) or do not respond to treatment (refractory). Samotolisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Targeting PI3K in cancer: mechanisms and advances in clinical trials.
    Yang J, Nie J, Ma X, Wei Y, et al · · 2019 · cited 1142× · PMID 30782187 · DOI 10.1186/s12943-019-0954-x
  2. Role of PI3K/AKT pathway in cancer: the framework of malignant behavior.
    Jiang N, Dai Q, Su X, Fu J, et al · · 2020 · cited 419× · PMID 32333246 · DOI 10.1007/s11033-020-05435-1
  3. Small molecule inhibitors targeting the cancers.
    Liu GH, Chen T, Zhang X, Ma XL, et al · · 2022 · cited 127× · PMID 36254250 · DOI 10.1002/mco2.181
  4. Molecular stratifications, biomarker candidates and new therapeutic options in current medulloblastoma treatment approaches.
    Menyhárt O, Győrffy B. · · 2020 · cited 60× · PMID 31970590 · DOI 10.1007/s10555-020-09854-1
  5. Molecular profiling and targeted therapy in pediatric gliomas: review and consensus recommendations.
    Miklja Z, Pasternak A, Stallard S, Nicolaides T, et al · · 2019 · cited 58× · PMID 30805642 · DOI 10.1093/neuonc/noz022
  6. Integrative discovery of treatments for high-risk neuroblastoma.
    Almstedt E, Elgendy R, Hekmati N, Rosén E, et al · · 2020 · cited 52× · PMID 31900415 · DOI 10.1038/s41467-019-13817-8
  7. Targeted fusion analysis can aid in the classification and treatment of pediatric glioma, ependymoma, and glioneuronal tumors.
    Lake JA, Donson AM, Prince E, Davies KD, et al · · 2020 · cited 49× · PMID 31595628 · DOI 10.1002/pbc.28028
  8. Opportunities and Challenges in Drug Development for Pediatric Cancers.
    Laetsch TW, DuBois SG, Bender JG, Macy ME, et al · · 2021 · cited 45× · PMID 33277309 · DOI 10.1158/2159-8290.cd-20-0779

Verify or expand the search:

Other trials of Biospecimen Collection

Trials testing the same drug.

Other recruiting trials for Advanced Malignant Solid Neoplasm

Currently open trials in the same condition.

Other National Cancer Institute (NCI) trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03213678.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing