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Molecularly Stratified Trial for Newly Diagnosed Infant-Type Hemispheric Glioma

29 oktober 2026 bijgewerkt door: St. Jude Children's Research Hospital

A Molecularly Stratified Phase II Trial for Newly Diagnosed Infant-Type Hemispheric Glioma (IHG)

The purpose of this study is to test whether giving treatment before surgery helps babies who are newly diagnosed with Infant-Type Hemispheric Glioma (IHG) have safer and better surgery results.

Studie Overzicht

Toestand

Nog niet aan het werven

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

Primary Objective

  • To estimate the complete tumor response rate (CTR) associated with molecularly directed neoadjuvant treatment (entrectinib for NTRK1/2/3 and ROS1, alectinib for ALK, and chemotherapy for tumors with other fusions and no fusions). (Strata A, B, and C)
  • To estimate the safe surgical resection rate associated with molecular-directed neo-adjuvant treatment entrectinib for NTRK1/2/3 and ROS1, alectinib for ALK, and chemotherapy for tumors with other fusions and no fusions. (Strata A, B, and C)

Secondary Objectives

  • To estimate the stratum-specific and study-wide event-free survival (EFS) and overall survival (OS) for patients enrolled in SJIHG25 (Strata A, B, C, and D)
  • To estimate the change in neurodevelopment and cognitive performance pre- and post-primary surgery in infants with newly diagnosed IHG treated with molecularly directed neoadjuvant treatment. (Strata A, B, and C)

This is a phase II clinical trial evaluating the use of molecularly directed neoadjuvant treatment for patients with newly diagnosed Infant-Type Hemispheric Glioma (IHG) to improve surgical outcome (efficacy and safety).

Bridge Therapy is recommended for participants who are being screened (participants who have signed the screening consent) for the following reasons:

  • Participant can be enrolled on the stratum bridge and 1-4 courses of cytotoxic chemotherapy CCE (carboplatin IV day 2, cyclophosphamide IV day1, and etoposide IV days 1 and 2), which may be given prior to surgical intervention to stabilize the patient. Each cycle of CCE treatment cycle is approximately 28 days in length.
  • If the waiting period for diagnostic work-up is deemed unsafe for clinical reasons, the participant may enroll in the stratum bridge and receive one course of bridge chemotherapy with CCE before enrollment to stratum A, B, or C.
  • Where a molecular target has been identified prior to screening and confirmation of diagnosis by central pathology review, the participant can be enrolled on stratum bridge and started on treatment based on the molecular target (alectinib po day1-28, entrectinib po day1-28, or chemotherapy (CCE), with pending central pathology review, if waiting for completion of the central pathology review is deemed to put the patient at increased risk of tumor progression.

Decisions regarding bridge therapy need to be discussed and approved by the study PI/team before starting treatment.

Enrollment in strata A, B, C, and D can occur only after central pathology review at St. Jude Children's Research Hospital confirms the diagnosis. Participants will be assigned to treatment strata based on whether residual tumor is present and types of RTK fusion.

Participants with ALK fusion will receive alectinib as neoadjuvant treatment (Stratum A), participants with NTRK 1/2/3 or ROS1 fusions will receive entrectinib as neoadjuvant treatment (Stratum B), and participants with no RTK fusion or RTK fusions other than ALK, ROS1 or NTRK1/2/3 (including MET fusion) will receive chemotherapy with carboplatin, etoposide, and cyclophosphamide as neoadjuvant treatment (Stratum C). Post primary surgery all participants on Strata A, B and C will be treated with 4 courses (1 course = 28 days) of adjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide.

The participants who have no residual disease at the time of enrollment will be treated with 4 courses (1 course = 28 days) of adjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide on stratum D.

Participants will be followed for five years following enrollment.

Studietype

Ingrijpend

Inschrijving (Geschat)

50

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Locaties

  • Verenigde Staten
    • Tennessee
      • Memphis, Tennessee, Verenigde Staten, 38105
        • St. Jude Children's Research Hospital
        • Contact:
        • Hoofdonderzoeker:
          • Aditi Bagchi, M.D., Ph.D.

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

Niet ouder dan 5 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria: Screening

  • Patient with presumptive or suspected (new) diagnosis of Infant-Type Hemispheric Glioma (IHG)
  • Patient age at diagnosis is from birth to ≤ 60 months
  • Patient has MRI/CT scan imaging of a supra-tentorial, hemispheric tumor avail-able for review before screening consent.
  • Patient is willing to undergo a biopsy procedure to confirm tissue diagnosis.
  • Adequate tumor tissue for central review of pathology, at the St. Jude Children's Research Hospital, DNA methylation array analysis, and NGS testing should be provided, if available from previous surgery, but is not required for screening eligibility.
  • The parent or legal guardian of the patient should be able to understand and be willing to sign a written informed consent document according to institutional guidelines

Inclusion Criteria: Enrollment

  • Without a central pathology confirmed diagnosis of IHG

    • Stratum-Bridge

      • The patient should meet all screening criteria and should have signed the screening consent.
      • Meets one of the exception/modification criteria

  • Centrally confirmed diagnosis of IHG

    • Strata A and B

      • The tumor has confirmed in-frame gene fusions involving ALK (Stratum A)
      • The tumor has confirmed in-frame gene fusions involving NTRK1/2/3 or ROS1 (Stratum B).
      • A measurable primary residual tumor at the time of enrollment

    • Stratum C

      • Central pathology review doesn't detect in-frame gene fusion involving ALK, NTRK 1/2/3, or ROS1in the tumor
      • Central pathology review confirms the presence of in-frame MET gene fusion or any other in frame fusion other than those involving ALK, NTRK1/2/3, or ROS1 gene fusions
      • A measurable primary residual tumor at the time of enrollment

    • Stratum D

      • No measurable primary residual tumor at the time of enrollment

Exclusion Criteria: (All Strata)

  • Age > 60 months
  • Any prior cancer-directed therapy
  • Organ Dysfunction, or a medical condition that, in the principal investigator's judgment, could compromise their ability to tolerate or absorb protocol therapy or would interfere with study procedures and results.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Stratum A
  • Neoadjuvant Treatment: Participants with ALK fusions will receive neoadjuvant treatment (4 Courses) with alectinib.
  • Surgery
  • Adjuvant Therapy: Participants will receive adjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide (4 courses).
Geneesmiddel: Alectinib

By mouth, nasogastric, or gastronomy/stomach tube, Day 1-28

Andere namen:
  • RO5424802
Procedure: Surgery

Primary surgery (Strata A, B, and C) post Neoadjuvant therapy with a goal of attaining gross total (GTR) or near total resection (NTR) of the tumor.

Andere namen:
  • Chirurgische resectie
Geneesmiddel: Cyclophosphamide

IV (intravenous) day 1

Andere namen:
  • Cytoxaan
Geneesmiddel: Etoposide

IV (intravenous) days 1 and 2

Andere namen:
  • VP-16
Geneesmiddel: Carboplatin

IV (intravenous) day 2

Andere namen:
  • Paraplatine
Biologisch: Filgrastim

Subcutaneous (under the skin) or IV (intravenous) day 3

Andere namen:
  • G-CSF
Biologisch: Pegfilgrastim

Subcutaneous (under the skin) day 3

Andere namen:
  • Neulasta
Experimenteel: Stratum B
  • Neoadjuvant Treatment: Participants with NTRK 1/2/3 or ROS1 fusions will receive neoadjuvant (4 Courses) treatment with entrectinib.
  • Surgery
  • Adjuvant Therapy: Participants will receive adjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide (4 courses).
Procedure: Surgery

Primary surgery (Strata A, B, and C) post Neoadjuvant therapy with a goal of attaining gross total (GTR) or near total resection (NTR) of the tumor.

Andere namen:
  • Chirurgische resectie
Geneesmiddel: Cyclophosphamide

IV (intravenous) day 1

Andere namen:
  • Cytoxaan
Geneesmiddel: Etoposide

IV (intravenous) days 1 and 2

Andere namen:
  • VP-16
Geneesmiddel: Carboplatin

IV (intravenous) day 2

Andere namen:
  • Paraplatine
Biologisch: Filgrastim

Subcutaneous (under the skin) or IV (intravenous) day 3

Andere namen:
  • G-CSF
Biologisch: Pegfilgrastim

Subcutaneous (under the skin) day 3

Andere namen:
  • Neulasta
Geneesmiddel: Entrectinib

By mouth, nasogastric, or gastronomy/stomach tube, Day 1-28

Andere namen:
  • RO7102122
Experimenteel: Stratum C
  • Neoadjuvant Treatment: Participants no RTK fusion or RTK fusions other than ALK, ROS1 or NTRK1/2/3 (including MET fusion) will receive neoadjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide (4 courses).
  • Surgery
  • Adjuvant Therapy: Participants will receive adjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide (4 courses).
Procedure: Surgery

Primary surgery (Strata A, B, and C) post Neoadjuvant therapy with a goal of attaining gross total (GTR) or near total resection (NTR) of the tumor.

Andere namen:
  • Chirurgische resectie
Geneesmiddel: Cyclophosphamide

IV (intravenous) day 1

Andere namen:
  • Cytoxaan
Geneesmiddel: Etoposide

IV (intravenous) days 1 and 2

Andere namen:
  • VP-16
Geneesmiddel: Carboplatin

IV (intravenous) day 2

Andere namen:
  • Paraplatine
Biologisch: Pegfilgrastim

Subcutaneous (under the skin) day 3

Andere namen:
  • Neulasta
Experimenteel: Stratum D

Participants who have no measurable residual disease at the time of enrollment.

  • Adjuvant Therapy: Participants will receive adjuvant chemotherapy with carboplatin, etoposide, and cyclophosphamide (4 courses).
Geneesmiddel: Cyclophosphamide

IV (intravenous) day 1

Andere namen:
  • Cytoxaan
Geneesmiddel: Etoposide

IV (intravenous) days 1 and 2

Andere namen:
  • VP-16
Geneesmiddel: Carboplatin

IV (intravenous) day 2

Andere namen:
  • Paraplatine
Biologisch: Filgrastim

Subcutaneous (under the skin) or IV (intravenous) day 3

Andere namen:
  • G-CSF
Biologisch: Pegfilgrastim

Subcutaneous (under the skin) day 3

Andere namen:
  • Neulasta

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Rate of Complete Tumor Response (CTR) among patients in Strata A, B, and C
Tijdsspanne: approximately 4 months after enrollment

The proportion of patients whose extent of tumor resection meets criteria for gross total / near total resection (GTR/NTR) after neoadjuvant therapy and surgery.
approximately 4 months after enrollment
Rate of safe surgical resection among patients in Strata A, B, and C
Tijdsspanne: Up to 72 hours after surgery

Proportion of patients whose surgery is completed without clinically significant intra-operative blood loss/hemorrhage. Success for this outcome measure will include surgery without any of the following:

(i) Initiation of massive transfusion protocol- i.e., requiring more than one volume (80ml/Kg) of transfusion over a period of 24 hours

(ii) Hemorrhage leading to cessation of surgery

(iii) Hemorrhagic complications leading to second neurosurgery within 72 hours of the first surgery.
Up to 72 hours after surgery

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Event free survival (EFS)
Tijdsspanne: 2 years

EFS is defined as time from enrollment for treatment (including bridge therapy) until progression, subsequent malignancy or death for any reason or until last follow-up, whichever is earlier.
2 years
Overall survival (OS)
Tijdsspanne: 2 years

OS is defined as time from enrollment for treatment (including bridge therapy) until death for any reason or until last follow-up, whichever is earlier.
2 years
Change in neurodevelopment and cognitive performance pre- and post-primary surgery in infants with newly diagnosed IHG treated with molecularly directed neoadjuvant treatment: BSID-4
Tijdsspanne: Pre-surgery/baseline and after the intervention/procedure/primary surgery for up to 12 weeks
Pre-surgery/baseline and after the intervention/procedure/primary surgery for up to 12 weeks
Change in neurodevelopment and cognitive performance pre- and post-primary surgery in infants with newly diagnosed IHG treated with molecularly directed neoadjuvant treatment: NIH Baby Toolbox
Tijdsspanne: Pre-surgery/baseline and after the intervention/procedure/primary surgery for up to 12 weeks
Pre-surgery/baseline and after the intervention/procedure/primary surgery for up to 12 weeks

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

St. Jude Children's Research Hospital

Medewerkers

Genentech, Inc.
St. Baldrick's Foundation

Onderzoekers

  • Hoofdonderzoeker: Aditi Bagchi, M.D., Ph.D., St. Jude Children's Research Hospital

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Nuttige links

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 november 2026

Primaire voltooiing (Geschat)

1 november 2034

Studie voltooiing (Geschat)

1 november 2036

Studieregistratiedata

Eerst ingediend

13 oktober 2026

Eerst ingediend dat voldeed aan de QC-criteria

29 oktober 2026

Eerst geplaatst (Werkelijk)

4 november 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

4 november 2026

Laatste update ingediend die voldeed aan QC-criteria

29 oktober 2026

Laatst geverifieerd

1 oktober 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • SJIHG25
  • NCI-2026-07231 (Register-ID: NCI Clinical Trial Registration Program)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

IPD-tijdsbestek voor delen

Data will be made available at the time of article publication.

IPD-toegangscriteria voor delen

Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

IPD delen Ondersteunend informatietype

  • LEERPROTOCOOL
  • SAP
  • ICF

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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