Recruiting

ACT001 for Children and Adolescents with DIPG and H3K27-altered Gliomas

What is being tested

ACT001

Drug

Who is being recruted

Diffuse Intrinsic Pontine Glioma+15

+ Brain Diseases

+ Brain Neoplasms

From 12 Months to 39 Years

+36 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 2

Interventional

Study Start: July 2025

See protocol details

Summary

Principal SponsorNationwide Children's Hospital

Study ContactKelsey H Troyer, PhD

Last updated: February 11, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: July 10, 2025

Actual date on which the first participant was enrolled.

This clinical trial is studying a drug called ACT001, which is being tested for its effectiveness in treating children and adolescents who have certain types of brain tumors known as Diffuse Intrinsic Pontine Gliomas (DIPG) and high-grade gliomas with a specific genetic alteration called H3K27. These tumors are challenging to treat, and there are limited options available, especially for cases that do not respond to current treatments or keep coming back. The goal of this study is to determine if ACT001 can help improve the outcomes for these young patients, potentially providing a new treatment option that could manage the disease more effectively. In the study, participants are divided into two groups based on their condition. Those who are newly diagnosed with DIPG are in one group, while those with difficult-to-treat or recurring forms of the tumors are in another. Participants receive ACT001 in pill form twice a day, with each treatment cycle lasting 28 days. The dose is adjusted based on body size, but it will not exceed a certain limit. The treatment continues for up to two years, or longer if it is helping, unless the disease worsens. The study monitors how well the drug works in stopping the tumor's progression and any benefits or side effects that may occur.

Official TitleA Phase II Trial of ACT001 in Children and Adolescents With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas

NCT06838676

Principal SponsorNationwide Children's Hospital

Study ContactKelsey H Troyer, PhD

Last updated: February 11, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

60 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 12 Months to 39 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Diffuse Intrinsic Pontine GliomaBrain DiseasesBrain NeoplasmsCentral Nervous System DiseasesGliomaNeoplasmsNeoplasms by Histologic TypeNeoplasms by SiteNeoplasms, Germ Cell and EmbryonalNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueNervous System DiseasesNervous System NeoplasmsInfratentorial NeoplasmsCentral Nervous System NeoplasmsNeuroectodermal TumorsNeoplasms, NeuroepithelialBrain Stem Neoplasms

Criteria

22 inclusion criteria required to participate

Patients must have started RT <42 calendar days of radiographic diagnosis (for non-biopsied DIPG patients only) or definitive surgery, whichever is later. It is strongly recommended patients begin RT within 31 days of diagnosis/definitive surgery. * If a biopsy was performed, the date of surgical biopsy will be considered the date of definitive diagnostic surgery; if a patient underwent two upfront surgeries [e.g., biopsy then debulking], this is the date of the second surgery)

Patients must have fully recovered from adverse events due to prior treatment with investigational or conventional agents must have recovered to a severity of Grade 0 or Grade 1 (per Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 or higher), with the exception of alopecia. Notes to the above for Cohort B: Patients with chronic Grade 2 toxicities may be eligible per discretion of the Investigator and Sponsor (e.g., Grade 2 chemotherapy-induced neuropathy). Grade 2 or 3 toxicities from prior anti-tumor therapy that are considered irreversible - defined as having been present and stable for > 6 months (such as ifosfamide-related proteinuria) may be allowed if they are not otherwise described in the exclusion criteria AND there is agreement to allow by both the Investigator and Sponsor.) The wash out period between the prior anti-cancer chemotherapy, and first dose of ACT001 (cycle 1 day 1) must be: 1. Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea). 2. Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor. 3. Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair. 4. Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines. 5. Monoclonal antibodies: > 21 days must have elapsed from the infusion of last dose of antibody and toxicity related to antibody therapy must be recovered to Grade ≤ 1 6. Radiation therapy: * For Cohort B, patients with refractory disease must have received their last fraction of frontline craniospinal or focal RT > 6 months prior to study enrollment. Patients that have received re-irradiation for progression must have received their last fraction of focal radiation >6 weeks or craniospinal radiation >6 weeks prior to study enrollment. 7. Stem Cell Transplant: Patients must be ≥ 3 months since autologous stem cell transplant. Patients who received allogenic stem cell transplant or solid organ transplant are not eligible for study

Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.

Cohort A: patients may have any disease status but must have completed initial radiation therapy (RT) before enrollment.

Show More Criteria

14 exclusion criteria prevent from participating

Patients who are currently receiving another investigational drug are not eligible.

Patients who currently have an uncontrolled infection (in the opinion of the PI) are not eligible.

Concomitant medications used with caution: selective serotonin reuptake inhibitor (SSRI) such as Lexapro, Fluoxetine (Prozac), fluvoxamine (Luvox), paroxetine (Paxil), sertraline (Zoloft), citalopram (Celexa), and escitalopram should be used with caution.

High Dose Biotin (B7) supplements are not permitted

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

2 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Patients with newly-diagnosed DIPG with typical MRI findings

Group II

Experimental

Patients with progressive/refractory/recurrent DIPG or extra-pontine H3K27-altered HGG tumors that have progressed following frontline treatment

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 19 locations

Recruiting

Children's National Medical Center

Washington D.C., United StatesOpen Children's National Medical Center in Google Maps

Recruiting

Cincinnati Children's Hospital

Cincinnati, United States

Recruiting

Nationwide Children's Hospital

Columbus, United States

Recruiting

Texas Children's Hospital

Houston, United States

Recruiting

19 Study Centers