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NCT03893487 | SNCTP000003508

Eine Zielvalidierungsstudie von Fimepinostat bei Kindern und Jugendlichen mit neu diagnostiziertem diffusem intrinsischen Ponsgliom (DIPG), rezidivierendem Medulloblastom oder rezidivierendem hochgradigem Gliom (HGG).

Data source: BASEC (Imported from 25.02.2020), WHO (Imported from 23.02.2020)
Changed: 23.02.2020
Disease category: Erkrankungen des Nervensystems

Brief description of trial (Source of data: BASEC)

Diese Studie wird durchgeführt, um herauszufinden, wieviel von dem Medikament Fimepinostat (CUDC-907) im Gehirn ankommt. Ausserdem wollen wir herausfinden, welche Wirkung Fimepinostat auf den Tumor und den Organismus hat. Alle Patienten werden mit dem Medikament behandelt

Health conditions investigated (Source of data: BASEC)

Neu diagnostiziertes diffuses intrinsisches Ponsgliom (DIPG), rezidivierendes Medulloblastom und rezidivierendes hochgradiges Gliom (HGG)

Health conditions (Source of data: WHO)

Diffuse Intrinsic Pontine Glioma;Recurrent Anaplastic Astrocytoma;Recurrent Glioblastoma;Recurrent Malignant Glioma;Recurrent Medulloblastoma

Rare disease (Source of data: BASEC)

Yes

Intervention investigated (e.g. drug, therapy or campaign) (Source of data: BASEC)

Orale Aufnahme von Fimepinostat

Interventions (Source of data: WHO)

Drug: Fimepinostat;Procedure: Therapeutic Conventional Surgery

Criteria for participation in trial (Source of data: BASEC)

Alter: zwischen 3 und 39 Jahren (in der Schweiz bis 21 Jahre)
Patienten, bei denen neu ein diffuser, intrinsischer Tumor im Hirnstamm, ein wiederkehrendes Medulloblastom (Tumor des Kleinhirns) oder ein wiederkehrender hochgradiger Tumor des Gehirns diagnostiziert wurde
Patienten müssen in der Lage sein, Tabletten zu schlucken

Exclusion criteria (Source of data: BASEC)

Personen, die bereits zuvor eine therapeutische Behandlung mit einem Wirkstoff, der gegen dieselben Zielmoleküle gerichtet wurde, erhalten haben
Patienten, die sich noch nicht von Nebenwirkungen anderer Medikamente erholt haben
Patienten mit einer HIV Infektion oder Diabetes Diagnose.

Inclusion/Exclusion Criteria (Source of data: WHO)


Inclusion Criteria:

- Patients must have one of the following histologically confirmed diagnoses (histologic
confirmation from initial diagnosis acceptable, as appropriate):

- Stratum A: Newly diagnosed diffuse intrinsic pontine glioma (DIPG) (World Health
Organization [WHO] grade II-IV)- this stratum does not require tissue
confirmation at time of enrollment, but diagnostic confirmation will be required
to continue on study after biopsy

- Stratum B: Recurrent medulloblastoma (WHO grade IV), any molecular subtype

- Stratum C: Recurrent high-grade glioma (HGG), including anaplastic astrocytoma
(WHO grade III) and glioblastoma (WHO grade IV)

- Stratum B & C: Patients in the recurrent medulloblastoma or recurrent HGG
arm can have locally recurrent or disseminated disease, provided
resection/biopsy would still be clinically indicated. Disseminated disease
can be diagnosed by imaging or cerebrospinal fluid (CSF) cytology

- Patients must be able to swallow intact fimepinostat capsules or mini-tabs without
chewing or crushing

- Patients must have body surface area (BSA) >= 0.5 m^2

- Patients must undergo tumor tissue collection as part of their standard of care

- Minimum possible tissue collected must be equivalent to about 4-6 stereotactic
core biopsies

- Strata B & C: Patients in the medulloblastoma and HGG strata will be allowed to have
undergone prior therapy including surgery, chemotherapy, and radiation therapy.
Patients in the DIPG stratum are not allowed to have prior therapy before the
initiation of fimepinostat. Patients must have fully recovered from acute side effects
related to previous anti-cancer therapies. Patients undergoing radiation during
protocol therapy will not be permitted to receive other concomitant agents with
radiation and pending initiation of maintenance with fimepinostat

- Myelosuppressive chemotherapy: At least 21 days after last dose of
myelosuppressive chemotherapy (42 days if prior nitrosourea)

- Hematopoietic growth factors: At least 14 days after last dose of a long-acting
growth factor or 7 days after short-acting growth factor or beyond time during
which adverse events are known to occur

- Biologic (anti-neoplastic agent): At least 7 days after last dose of a biologic
agent or beyond time during which adverse events are known to occur

- Monoclonal antibodies: At least 21 days after last dose of monoclonal antibody

- Radiotherapy:

- At least 2 weeks after local palliative radiotherapy (XRT)

- At least 3 months from craniospinal XRT, or XRT to > 50% pelvis

- Surgery:

- At least 21 days from major surgery (biopsy and central line
placement/removal are not considered major)

- Corticosteroids: Subjects who are receiving dexamethasone must be on a stable or
decreasing dose for at least 7 days prior to enrollment

- Peripheral absolute neutrophil count (ANC) >= 1000/mm^3

- Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving
platelet transfusions for at least 7 days prior to enrollment)

- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
milliliters (mL)/minute (min)/1.73 m^2 or

- A serum creatinine based on age/gender as follows:

- Age: Maximum Serum Creatinine (mg/dL)

- 3 to < 6 years: 0.8 (male), 0.8 (female)

- 6 to < 10 years: 1 (male), 1 (female)

- 10 to < 13 years: 1.2 (male), 1.2 (female)

- 13 to < 16 years: 1.5 (male), 1.4 (female)

- >= 16 years: 1.7 (male), 1.4 (female)

- Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for
age

- Serum glutamate pyruvate transaminase (SGPT)/alanine aminotransferase (ALT) =< 110 U/L

- Serum albumin >= 2 g/dL

- Neurologic function:

- Subjects with seizure disorder may be enrolled if well controlled

- Gastrointestinal function:

- Diarrhea < grade 2 by Common Terminology Criteria for Adverse Events (CTCAE)
version (v)5.0

- Metabolic function:

- Non-fasting glucose < 125 mg/dL without the use of antihyperglycemic agents

- If non-fasting glucose > 125 mg/dL, a fasting glucose should be done. If
fasting glucose =< 160 mg/dL without the use of antihyperglycemic agents,
patient will meet adequate metabolic function criteria

- Cardiac function: corrected QT (QTc) < 480 msec

- The effects of fimepinostat on the developing human fetus are unknown. For this
reason, women of child-bearing potential and men must agree to use adequate
contraception (hormonal or barrier method of birth control; abstinence) prior to study
entry, for the duration of study participation and 30 days after completion of
fimepinostat administration. Should a woman become pregnant or suspect she is pregnant
while she or her partner is participating in this study, she should inform her
treating physician immediately

- A legal parent/guardian or patient must be able to understand, and willing to sign, a
written informed consent and assent document, as appropriate

Exclusion Criteria:

- Subjects who have not recovered from acute adverse events due to therapeutic agents
administered more than 4 weeks earlier

- Patients must not have received prior therapy with single-agent or combination histone
deacetylase (HDAC) and Phosphatidylinositol-4,5-bisphosphate 3-kinase (PI3K)
inhibitors

- Subjects who are receiving any other investigational agent

- History of allergic reaction to compounds of similar chemical or biological
composition to fimepinostat

- Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situation that would limit compliance with
study requirements

- Patients with active human immunodeficiency virus (HIV) infection and with potential
life-threatening consequences associated with immune-suppressive therapy

- Patients with history of type 1 or 2 diabetes mellitus

- Patients with gastrointestinal condition that could interfere with absorption or
metabolism of fimepino

Further information on the trial in WHO primary registry

https://clinicaltrials.gov/show/NCT03893487

Further information on the trial from WHO database (ICTRP)

http://apps.who.int/trialsearch/Trial2.aspx?TrialID=NCT03893487

Further information on trial

Date trial registered

26.03.2019

Incorporation of the first participant

07.08.2019

Recruitment status

Recruiting

Academic title (Source of data: WHO)

A Target Validation Study of Fimepinostat in Children and Young Adults With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG)

Type of trial (Source of data: WHO)

Interventional

Design of the trial (Source of data: WHO)

Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).

Phase (Source of data: WHO)

Early Phase 1

Primary end point (Source of data: WHO)

Penetration of fimepinostat across the blood brain barrier (BBB)

Contact information (Source of data: WHO)

Please refer to primary and secondary sponsors

Trial sites

Trial sites in Switzerland (Source of data: BASEC)

Zürich

Countries (Source of data: WHO)

Switzerland, United States

Contact for further information on the trial

Details of contact in Switzerland (Source of data: BASEC)

Nicolas Gerber
+41 44 266 3117
nicolas.gerber@kispi.zh.ch

Contact for general information (Source of data: WHO)

Sabine Mueller, MD, PhD;Kelly Hitchner
UCSF Medical Center-Mount Zion
415-502-1600
PNOC_Regulatory@ucsf.edu

Contact for scientific information (Source of data: WHO)

Sabine Mueller, MD, PhD;Kelly Hitchner
UCSF Medical Center-Mount Zion
415-502-1600
PNOC_Regulatory@ucsf.edu

Principal Sponsor/Investigator

Principal sponsor (Source of data: WHO)

Sabine Mueller, MD, PhD

Additional sponsors (Source of data : WHO)

Pacific Pediatric Neuro-Oncology Consortium;Cannonball Kids' Cancer Foundation;Curis, Inc.

Further trial identification numbers

BASEC ID (Source of data: BASEC)

2019-01178

Secondary ID (Source of data: WHO)

18086;PNOC016