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SNCTP000003508 | NCT03893487 | BASEC2019-01178

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

Datenbasis: BASEC (Import vom 30.11.2021), WHO (Import vom 18.04.2021)
Geändert: 05.11.2021
Krankheitskategorie: Erkrankungen des Nervensystems

Zusammenfassende Beschreibung der Studie (Datenquelle: 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

Untersuchte Krankheiten (Datenquelle: BASEC)

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

Health conditions (Datenquelle: WHO)

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

Seltene Krankheit (Datenquelle: BASEC)

Ja

Untersuchte Intervention (z.B. Medikament, Therapie, Kampagne) (Datenquelle: BASEC)

Orale Aufnahme von Fimepinostat

Interventions (Datenquelle: WHO)

Drug: Fimepinostat;Procedure: Therapeutic Conventional Surgery

Kriterien zur Teilnahme an der Studie (Datenquelle: 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

Ausschlusskriterien (Datenquelle: 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 (Datenquelle: 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 (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.
Patients with newly diagnosed DIPG will be eligible to enroll before or after
standard of care radiation, but must be eligible for a biopsy. Newly diagnosed
DIPG stratum should not have received prior therapy before the initiation of
fimepinostat, with the exception of those patients who received temozolomide
during radiation therapy or who previously received radiation as per standard of
care and have not yet undergone a biopsy. All patients who have received therapy
other than radiation and temozolomide should be discussed with study chair(s)
prior to enrollment. Patients enrolling after standard of care radiation must be
enrolled within 14 weeks of completion of radiotherapy.

- 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. Recurrent
DIPG will be eligible for stratum C; however, eligibility requires
biopsy/resection is feasible in a region of tumor outside of the pons (i.e.
cerebellar extension or new metastatic site). These patients should be
discussed with study chair(s) prior to enrollment

- 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

- Prior Therapy: Patients in the medulloblastoma and HGG strata will be allowed to have
undergone prior therapy including surgery, chemotherapy, and radiation therapy.
Patients in the newly diagnosed DIPG stratum should not have received prior therapy
before the initiation of fimepinostat, with the exception of those patients who
received temozolomide during radiation therapy or who previously received radiation as
per standard of care and have not yet undergone a biopsy. All patients who have
received therapy other than radiation and temozolomide should be discussed with study
chair(s) prior to enrollment. 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
treati

Weitere Angaben zur Studie im WHO-Primärregister

https://clinicaltrials.gov/show/NCT03893487

Weitere Angaben zur Studie aus der Datenbank der WHO (ICTRP)

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

Weitere Informationen zur Studie

Registrationsdatum der Studie

26.03.2019

Einschluss der ersten teilnehmenden Person

07.08.2019

Rekrutierungsstatus

Recruiting

Wissenschaftlicher Titel (Datenquelle: 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)

Studientyp (Datenquelle: WHO)

Interventional

Design der Studie (Datenquelle: WHO)

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

Phase (Datenquelle: WHO)

Early Phase 1

Primäre Endpunkte (Datenquelle: WHO)

Penetration of fimepinostat across the blood brain barrier (BBB)

Kontakt für Auskünfte (Datenquelle: WHO)

Please refer to primary and secondary sponsors

Ergebnisse der Studie (Datenquelle: WHO)

Zusammenfassung der Ergebnisse

noch keine Angaben verfügbar

Link zu den Ergebnissen im Primärregister

noch keine Angaben verfügbar

Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten

noch keine Angaben verfügbar

Studiendurchführungsorte

Durchführungsorte in der Schweiz (Datenquelle: BASEC)

Zürich

Durchführungsländer (Datenquelle: WHO)

Switzerland, United States

Kontakt für weitere Auskünfte zur Studie

Angaben zur Kontaktperson in der Schweiz (Datenquelle: BASEC)

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

Kontakt für allgemeine Auskünfte (Datenquelle: WHO)

Sabine Mueller, MD, PhD;Aubrie Drechsler
University of California, San Francisco
415-502-1600
PNOC_Regulatory@ucsf.edu

Kontakt für wissenschaftliche Auskünfte (Datenquelle: WHO)

Sabine Mueller, MD, PhD;Aubrie Drechsler
University of California, San Francisco
415-502-1600
PNOC_Regulatory@ucsf.edu

Studienverantwortliche

Hauptsponsor (Datenquelle: WHO)

Sabine Mueller, MD, PhD

Weitere Sponsoren (Datenquelle: WHO)

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

Bewilligung durch Ethikkommission (Datenquelle: BASEC)

Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)

Kantonale Ethikkommission Zürich

Datum der Bewilligung durch die Ethikkommission

28.10.2019

Weitere Studienidentifikationsnummern

Studienidentifikationsnummer der Ethikkommission (BASEC-ID) (Datenquelle: BASEC)

2019-01178

Secondary ID (Datenquelle: WHO)

NCI-2019-00144
PNOC016
18086