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New treatment option for Diffuse Intrinsic Pontine Glioma
Official title Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0
ClinicalTrials.gov ID: NCT05476939
What this study is testing
What is Everolimus?
Everolimus is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for diffuse intrinsic pontine glioma.
Also referred to as VOTUBIA, AFINITOR.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The BIOMEDE 2.0 study is the second stage of the BIOMEDE multi-arm, multistage rolling programme (adaptive platform protocol). It is a multicenter, randomized, open-label, controlled phase-3 trial evaluating efficacy of ONC201 in comparison with everolimus (primary objective based on internal comparison) and subsequently to historical controls.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- Eligibility criteria for the inclusion (registration) in BIOMEDE 2.0 study:
- Diagnosis Criteria:
- Diagnosis of DIPG (clinical and radiological). As biopsy is not standard for these tumors, an informed consent is required for the necessary...
- Histological diagnosis of DIPG (i.e. H3K28M or EZHIP positive Diffuse Midline Glioma located in the pons) in case the tumor biopsy was performed...
- Molecular diagnosis of DIPG (i.e. H3K28M) in case a liquid biopsy (CSF or blood) was performed before study entry, in a patient who could not undergo...
See the full eligibility criteria
Who can join
- Eligibility criteria for the inclusion (registration) in BIOMEDE 2.0 study:
- Diagnosis Criteria:
- Diagnosis of DIPG (clinical and radiological). As biopsy is not standard for these tumors, an informed consent is required for the necessary histological verification. [Biopsy-part of BIOMEDE 2.0 trial]. OR
- Histological diagnosis of DIPG (i.e. H3K28M or EZHIP positive Diffuse Midline Glioma located in the pons) in case the tumor biopsy was performed before study entry. The diagnosis will be defined by 1/ diffuse glioma, 2/...
- Molecular diagnosis of DIPG (i.e. H3K28M) in case a liquid biopsy (CSF or blood) was performed before study entry, in a patient who could not undergo a tumor biopsy because it was too dangerous according to the...
- Non-DIPG diffuse midline gliomas (ND-DMG), H3K28M mutant or with H3K28 trimethylation loss together with EZHIP overexpression, will be eligible for the trial after biopsy or surgery. As biopsy and surgery is considered...
- Non-DIPG diffuse midline gliomas (ND-DMG) will be eligible for the trial before the biopsy in case the diagnosis is clinically or radiologically suspected. Informed consent for the biopsy and molecular analysis will be...
- Eligible for a biopsy, or biopsy material available for the biomarker assessment.
- Age \> 6 months, with no upper age limit. Children between 6 months and 3 years will be discussed on a case by case basis for inclusion in the study for the feasibility of the stereotactic biopsy.
- Eligible for cerebral or craniospinal radiotherapy.
- Tumor at diagnosis: no prior chemotherapy for the present cancer; no prior cerebral radiation therapy even for another neoplasm. Surgery is allowed when performed for diagnostic or therapeutic purpose.
- Metastatic diseases or spinal tumors allowed; in this case, patients would receive craniospinal or spinal radiotherapy and medical treatment (everolimus or ONC201) will be postponed and only started after the end of...
- Patients must be affiliated to a social security system or beneficiary of the same according to local requirements.
- Written informed consent from parents/legal representative, patient, and age-appropriate assent before any study-specific procedures are conducted according to local, regional or national guidelines. Non eligibility...
- Uncontrolled spontaneous massive intratumor bleeding. Patients with post-operative bleeding will be allowed to enter the study provided the hemorrhage is controled. Same rule applies for the other post-operative...
- Any other concomitant anti-cancer treatment not foreseen by this protocol is not allowed, except corticosteroids and Bevacizumab which are allowed during the protocol. Bevacizumab is not allowed before and until 15 days...
- Any other cancer diagnosed during the last 5 years.
- Uncontrolled intercurrent illness or active infection.
- Any other co-morbid condition that in the investigator's opinion would impair study participation.
- Unable for medical follow-up (geographic, social or mental reasons).
- Patient previously treated with irradiation on the brainstem for another neoplasm.
- Participation in another clinical study with an investigational product while on study treatment.
- Patient under guardianship or deprived of his/her liberty by a judicial or administrative decision or incapable of giving his/her consent. Eligibility criteria for the randomization in BIOMEDE 2.0 study:
- Patient enrolled in the BIOMEDE 2.0 study.
- Life expectancy \> 12 weeks after the start of study treatment.
- Histological diagnosis of DIPG (as per the WHO criteria) confirmed by central pathology review, OR Typical radiology of a DIPG (mandatory central radiological review) as well as the short clinical history (less than...
- Karnofsky performance status scale or Lansky Play Scale \> 50%. The PS should not take the neurologic deficit per se into account. NB: Children and adults with a worse performance status due to glioma-related motor...
- Highly effective and appropriate contraception for patients (male and female) of reproductive potential during their entire participation in the study and during 6 months after the end of treatment.
- Negative pregnancy test (serum beta-HCG or urinary test) evaluated within one week prior randomization in sexually active females of reproductive potential.
- Absolute neutrophil count \> 1.0 x 10\^9/l, Platelets \> 100 x 10\^9/l.
- Total bilirubin \< 1.5 x ULN, AST and ALT\< 2.5 x ULN.
- Serum creatinine \ 1.5 x ULN, creatinine clearance must be \> 70 ml/min/1.73 m² (as per local practice).
- Normal coagulation tests within the local reference ranges.
- Written informed consent from parents/legal representative, patient, and age-appropriate assent before randomization according to local, regional or national guidelines. Non Eligibility criteria for the randomization in...
- Current organ toxicity \> grade 2 according to the NCI-CTCAE version 5.0, especially cardiovascular or renal disease (including but not limited to: congenital long QT syndrome, nephrotic syndrome, glomerulopathy...
- Patients with the following cardiac history cannot take ONC201:
- Prolongation of QT/QTcF interval (QTc interval \> 480 milliseconds) preferably using Frederica's QT correction formula on two ECGs separated by at least 48 hours.
- A history of Torsades de pointes or heart failure, hypokalemia, or family history of prolonged QT Syndrome.
- Required concomitant use of medication(s) known to prolong the QT/QTc interval. In this case, patients will be treated in the Everolimus arm without randomization (except if contra-indication to Everolimus).
- Pregnant or breastfeeding women.
- Patients with chronic HBV disease compatible with the trial are not excluded from the study. These patients assigned by chance to everolimus treatment will have regular viral load monitoring throughout the study.
- Patients taking strong P450 inhibitors or inducers or PgP inhibitors are not excluded from the study but drug concentration of everolimus should be monitored carefully to avoid toxicity. Preferably alternative...
- Patient with known congenital galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption will not be assigned by chance and will be treated in the ONC201 arm (except if contra-indication to...
- Patients with known hypersensitivity to any component of Everolimus (active substance, other rapamycin derivatives or excipients) will not be assigned by chance and will be treated in the ONC201 arm (except if...
- Patients with known hypersensitivity to any component of ONC201 (drug product or excipients) will not be assigned by chance and will be treated in the Everolimus arm (except if contra-indication to Everolimus).
The study team makes the final eligibility decision.
Where it's taking place
- Aarhus, Denmark
- Copenhagen, Denmark
- Odense, Denmark
- Villejuif, Val de Marne, France
- Amiens, France
- Angers, France
- Besançon, France
- Bordeaux, France
- Brest, France
- Caen, France
- Clermont-Ferrand, France
- Dijon, France
- Grenoble, France
- Lille, France
- Limoges, France
- Lyon, France
- Marseille, France
- Montpellier, France
- Nancy, France
- Nice, France
+ 16 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Aarhus, Denmark; Copenhagen, Denmark; Odense, Denmark; Villejuif, Val de Marne, France; Amiens, France; Angers, France and 30 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.