A Phase II Trial of Oral Medication ACT001 to Treat Children and Teens With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
Phase: Recruiting
First Posted: August
Condition(s): Diffuse Intrinsic Pontine Gliomas (DIPG) , High-Grade Gliomas (HGG)
NCT Number: NCT06838676 Other Study ID Number(s): CONNECT2110
What Is the Purpose of This Study?
This is a Phase II open-label study to study the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
Who Can Take Part in This Study?
People who may be eligible for this study:
- Have newly diagnosed DIPG or progressive/refractory/recurrent DIPG or H3K27-altered HGG
- Are 12 months to 39 years old
For a full list of eligibility requirements, visit https://www.clinicaltrials.gov/study/NCT06838676.
What Will Happen During This Study?
Patients will be enrolled on either Cohort A for newly diagnosed DIPG or on Cohort B for those with progressive/refractory DIPG or progressive/recurrent/refractory H3K27-altered HGG.
- Each group of patients in the study will take ACT001 by mouth twice a day.
- The dose will be based on body size (875 mg per square meter of body surface area), with a maximum dose of 1700 mg twice a day.
- One cycle of treatment lasts 28 days (about 4 weeks).
- If the treatment is helping and there are no serious side effects, patients can:
- Continue taking ACT001 for up to 26 cycles (about 2 years), or
- Stop earlier if the tumor grows or the disease gets worse.
- In some cases, patients may be allowed to keep taking ACT001 beyond 2 years if:
- They are still benefiting from the treatment, and
- Their doctor and the study sponsor agree it’s the right choice.
Principal Investigator
Discover More Research
Explore additional studies relating to neuro-oncologyYour Might Also Be Interested In
Neuro-Oncology
Our highly experienced and internationally recognized team of pediatric neuro-oncologists, neurosurgeons and neuroradiologists will care for your child by providing the most advanced and innovative treatments, while at the same time, paying careful attention to all the needs and concerns of your child and family.
High-Grade Gliomas
High-grade gliomas are fast-growing cancers that form in the brain or spinal cord. Surgery, radiation, and targeted therapies can help. High-grade gliomas are more aggressive and can grow quickly. They are the second most common type of malignant brain tumor in children.
Diffuse Intrinsic Pontine Glioma (DIPG)
Diffuse intrinsic pontine glioma, also know as DIPG, is an aggressive, malignant brain tumor that develops in a part of the brainstem called the pons.