Actively Recruiting
ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
Led by Nationwide Children's Hospital · Updated on 2026-03-27
60
Participants Needed
20
Research Sites
520 weeks
Total Duration
On this page
Sponsors
N
Nationwide Children's Hospital
Lead Sponsor
A
Accendatech USA Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
CONDITIONS
Official Title
ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 12 months to 39 years at enrollment
- For Cohort A: Newly diagnosed DIPG with typical MRI findings, completed radiation therapy within 28 to 35 days prior to treatment start
- Radiation therapy started within 42 days of diagnosis or surgery for non-biopsied DIPG
- No prior anti-cancer therapy other than surgery, radiation, or steroids before enrollment for Cohort A
- For Cohort B: Progressive, refractory, or recurrent DIPG or pathologically confirmed H3K27-altered HGG after frontline radiation therapy
- Measurable disease by MRI for Cohort B; metastatic disease allowed
- Karnofsky performance score ≥50% for patients >16 years; Lansky score >50% for patients ≤16 years
- Fully recovered from prior treatment adverse events to grade 0 or 1 (except alopecia) for Cohort B
- Adequate bone marrow, renal, liver, cardiac, and neurologic function
- Signed informed consent and assent when appropriate
- Absence of other clinically significant active medical disorders as judged by investigator
You will not qualify if you...
- Patients with metastatic disease in Cohort A
- Receiving other anti-cancer agents or investigational drugs currently
- Use of enzyme-inducing anticonvulsants or LHRH agonists/antagonists
- Use of high dose Biotin (B7) supplements
- Uncontrolled infection
- Prior solid organ transplantation
- Pregnant or breastfeeding women; must use effective contraception if of reproductive potential
- Unable to comply with safety monitoring requirements
- Prior treatment with ACT001 or parthenolide
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 20 locations
1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
2
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
3
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Not Yet Recruiting
4
Emory University/Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Not Yet Recruiting
5
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
Not Yet Recruiting
6
St. Louis Children's Hospital
St Louis, Missouri, United States, 63110
Not Yet Recruiting
7
Duke University Medical Center
Durham, North Carolina, United States, 27708
Not Yet Recruiting
8
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
Actively Recruiting
9
Nationwide Children's Hospital
Columbus, Ohio, United States, 43235
Actively Recruiting
10
Children's Hospital of Philidelphia
Philidelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
11
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
12
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
13
Sydney Children's Hospital
Randwick, New South Wales, Australia, 2031
Not Yet Recruiting
14
Queensland Children's Hospital
South Brisbane, Queensland, Australia, 4101
Not Yet Recruiting
15
Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
Not Yet Recruiting
16
Perth Children's Hospital
Perth, Western Australia, Australia, 6000
Not Yet Recruiting
17
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada, M5G1X8
Not Yet Recruiting
18
Montreal Children's Hospital
Montreal, Quebec, Canada, H4A3J1
Not Yet Recruiting
19
Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)
Heidelberg, Baden-Wurttemberg, Germany, 69120
Not Yet Recruiting
20
Starship Children's Hospital
Auckland, Grafton, New Zealand, 1023
Not Yet Recruiting
Research Team
K
Kelsey H Troyer, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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