Clinical Trials Register

Summary
EudraCT Number:	2021-004740-24
Sponsor's Protocol Code Number:	INCB54828-209
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-03-18
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-004740-24

A.3

Full title of the trial

A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations (FIGHT-209)

Studio multicentrico di fase 2, in aperto, a braccio singolo, per valutare l’efficacia e la sicurezza di pemigatinib in partecipanti con glioblastoma o altri tumori primitivi del sistema nervoso centrale precedentemente trattati che presentano alterazioni attivanti di FGFR1-3 (FIGHT-209)

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

INCB54828-209

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	INCYTE CORPORATION
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Incyte Corporation
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Incyte Corporation
B.5.2	Functional name of contact point	Clinical Trials Information
B.5.3	Address:
B.5.3.1	Street Address	1801 Augustine Cut-Off
B.5.3.2	Town/ city	Wilmington
B.5.3.3	Post code	DE 19803
B.5.3.4	Country	United States
B.5.4	Telephone number	000000
B.5.5	Fax number	+13024252734
B.5.6	E-mail	RA@incyte.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Pemazyre
D.2.1.1.2	Name of the Marketing Authorisation holder	Incyte Biosciences Distribution B.V. EU/1/21/1535/005
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Pemigatinib
D.3.2	Product code	[INCB054828]
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PEMIGATINIB
D.3.9.1	CAS number	1513857-77-6
D.3.9.2	Current sponsor code	INCB054828
D.3.9.3	Other descriptive name	INCB054828
D.3.9.4	EV Substance Code	SUB194579
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	13
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Pemazyre
D.2.1.1.2	Name of the Marketing Authorisation holder	Incyte Biosciences Distribution B.V. EU/1/21/1535/001
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Pemigatinib
D.3.2	Product code	[INCB054828]
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PEMIGATINIB
D.3.9.1	CAS number	1513857-77-6
D.3.9.2	Current sponsor code	INCB054828
D.3.9.3	Other descriptive name	INCB054828
D.3.9.4	EV Substance Code	SUB194579
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	4
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Pemazyre
D.2.1.1.2	Name of the Marketing Authorisation holder	Incyte Biosciences Distribution B.V. EU/1/21/1535/003
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Pemigatinib
D.3.2	Product code	[INCB054828]
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PEMIGATINIB
D.3.9.1	CAS number	1513857-77-6
D.3.9.2	Current sponsor code	INCB054828
D.3.9.3	Other descriptive name	INCB054828
D.3.9.4	EV Substance Code	SUB194579
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	9
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Male and female participants at least 18 years of age who have recurrent GBM or other recurrent primary CNS tumors harboring an FGFR1-3 rearrangement or fusion, who have progressed on at least one line of standard of care therapy, eg chemotherapy and/or radiation therapy

Partecipanti di sesso maschile e femminile di almeno 18 anni di età con recidive di GBM o recidive di altri tumori primitivi del SNC che presentano un riarrangiamento o una fusione di FGFR1-3, che hanno mostrato progressione su almeno una linea di terapia standard, ad es. chemioterapia e/o radioterapia

E.1.1.1

Medical condition in easily understood language

Participants with brain tumors harboring FGFR1-3 mutation, who have progressed after at least one standard of care therapy

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10065147
E.1.2	Term	Malignant solid tumor
E.1.2	System Organ Class	100000004864

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To determine the efficacy of pemigatinib in participants with recurrent GBM or other primary CNS tumors with an activating FGFR1-3 mutation or fusion/rearrangement.

determinare l’efficacia di pemigatinib in partecipanti con recidive di GBM o di altri tumori primitivi del SNC con una mutazione attivante o fusione/riarrangiamento di FGFR1-3.

E.2.2

Secondary objectives of the trial

To determine the safety and tolerability of pemigatinib in participants with recurrent GBM or other primary CNS tumors with an activating FGFR1-3 mutation and/or fusion/rearrangement.

determinare la sicurezza e la tollerabilità di pemigatinib in partecipanti con recidive di GBM o di altri tumori primitivi del SNC con una mutazione attivante e/o fusione/riarrangiamento di FGFR1-3.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Ability to comprehend and willingness to sign a written ICF for the study.
2. Male and female participants aged 18 years or older at the time of signing the ICF.
3. Histological, cytological, or molecular confirmation of recurrent GBM or other adult type, diffuse glioma or circumscribed astrocytic tumors.
a. For Cohorts A and C: Prior histopathologically proven WHO grade 4, IDH–wild-type GBM OR molecular diagnosis of IDH–wild-type, diffuse astrocytic glioma with molecular features of WHO grade 4 GBM (astrocytic glioma requires presence of either amplification of EGFR, whole chromosome 7 gain and whole chromosome 10 loss, or TERTpromoter mutation) that has recurred or progressed on or after treatment with at least 1 line of standard of care therapy.
b. For Cohorts B and C: Prior histopathologically proven, per WHO criteria, adult-type diffuse gliomas other than GBM, including IDHmutant astrocytoma and IDH-mutant and 1p/19q codeleted
oligodendroglioma, and circumscribed astrocytic tumors, including pilocytic astrocytomas, that are recurrent or progressed on or after at least 1 line of standard of care therapy. For Cohort C, all gliomas and
glioneuronal and neuronal tumors with a known or likely FGFR 1-3 activating mutation are also eligible.
4. Radiographically measurable disease (per RANO). Tumor lesions located in a previously irradiated area, or in an area subjected to other loco-regional therapy, are considered measurable if progression has been clearly demonstrated in the lesion.
5. Karnofsky performance status = 60.
6. Life expectancy = 12 weeks.
7. Documentation of an FGFR1-3 gene mutation or fusion/rearrangement from tissue.
for the complete list refer to the protocol

1. Capacità di comprensione e disponibilità a firmare un ICF redatto per lo studio.
2. Partecipanti di sesso maschile e femminile di età pari o superiore a 18 anni al momento della firma dell’ICF.
3. Conferma istologica, citologica o molecolare di recidiva di GBM o di altri tipi che si manifestano nell’adulto, glioma ad alto grado di malignità o tumori astrocitici circoscritti.
a. Per le Coorti A e C: GBM di tipo IDH-wild-type di IV grado secondo la classificazione OMS precedentemente comprovata da diagnosi istopatologica OPPURE GBM glioma astrocitico ad alto grado di malignità di tipo IDH-wild-type di IV grado secondo la classificazione OMS da diagnosi molecolare (il glioma astrocitico richiede la presenza sia dell’amplificazione di EGFR, del guadagno dell’intero cromosoma 7 e della perdita dell’intero cromosoma 10, sia della mutazione del promotore della TERT) recidivante o progredito durante o dopo il trattamento con almeno 1 linea di terapia standard.
b. Per le Coorti B e C: Gliomi ad alto grado di malignità che si manifestano nell’adulto diversi dal GBM, precedentemente comprovati da diagnosi istopatologica, secondo i criteri dell’OMS, inclusi l’astrocitoma con mutazioni della IDH, l’oligodendroglioma con mutazioni della IDH e codelezione 1p/19q e i tumori astrocitici circoscritti, incluso l’astrocitoma pilocitico, recidivanti o progrediti durante o dopo almeno 1 linea di terapia standard. Per la Coorte C sono idonei anche tutti i gliomi e i tumori glioneuronali e neuronali con mutazione attivante nota o probabile di FGFR 1-3.
4. Malattia misurabile radiograficamente (secondo i criteri RANO). Le lesioni tumorali situate in una zona precedentemente irradiata o in una zona sottoposta ad altra terapia locoregionale sono considerate misurabili se la progressione è stata chiaramente dimostrata nella lesione.
5. Stato di performance secondo Karnofsky =60.
6. Aspettativa di vita =12 settimane.
7. Documentazione di mutazione o di fusione/riarrangiamento del gene FGFR1-3 dal tessuto.
per l'elenco completo si faccia riferimento al protocollo

E.4

Principal exclusion criteria

1. Prior receipt of a selective FGFR inhibitor.
2. Receipt of anticancer medications or investigational drugs for any indication or reason within 28 days before first dose of study drug. Participants must have recovered (= Grade 1) from AEs from previously administered therapies.
3. Participants may have had treatment for an unlimited number of prior relapses but must not have had prior bevacizumab or other VEGF/VEGFR inhibitors.
4. Concurrent anticancer therapy.
5. Candidate for potentially curative surgery.
6. Dexamethasone (or equivalent) > 4 mg daily at the time of study registration (higher dose of steroid for symptom control is allowed during the study).
7. Current evidence of clinically significant corneal or retinal disorder as confirmed by ophthalmologic examination.
8. Diffuse leptomeningeal disease.
9. Radiation therapy administered within 12 weeks before enrollment/first dose of study drug. An interval of at least 12 weeks after prior radiotherapy is required unless there is either histopathological confirmation of recurrent tumor or new enhancement on MRI outside the radiotherapy field.
10. Known additional malignancy that is progressing or requires active systemic treatment. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy.

1. Aver ricevuto in precedenza un inibitore selettivo di FGFR.
2. Aver ricevuto farmaci antitumorali o farmaci sperimentali per qualsiasi indicazione o motivo nei 28 giorni precedenti la prima dose del farmaco dello studio.
I partecipanti devono essersi ripresi (ritorno a grado =1) dagli eventi avversi (EA) dovuti a terapie somministrate in precedenza.
3. I partecipanti possono aver ricevuto trattamenti per un numero illimitato di recidive pregresse ma non devono aver assunto in precedenza bevacizumab o altri inibitori di VEGF/VEGFR.
4. Terapia antitumorale concomitante.
5. Candidato per chirurgia potenzialmente curativa.
6. Desametasone (o equivalente) >4 mg al giorno al momento della registrazione dello studio (è consentita una dose più alta di steroidi per il controllo dei sintomi durante lo studio).
7. Evidenza attuale di disturbo corneale o retinico clinicamente significativo, come confermato mediante esame oftalmologico.
8. Malattia leptomeningea ad alto grado di malignità.
9. Radioterapia somministrata nelle 12 settimane precedenti l’arruolamento/la prima dose del farmaco dello studio. È necessario un intervallo di almeno 12 settimane dopo una precedente radioterapia, sempre che non vi sia una conferma istopatologica della recidiva del tumore o un nuovo miglioramento alla RM al di fuori del campo della radioterapia.
10. Altro tumore maligno noto in progressione o che richiede un trattamento sistemico attivo. Le eccezioni comprendono carcinoma basocellulare, carcinoma squamocellulare o tumore della cervice uterina in situ che sia stato sottoposto a terapia potenzialmente curativa.

E.5 End points

E.5.1

Primary end point(s)

• ORR in Cohort A, defined as the proportion of participants in Cohort A who achieve a BOR of CR or PR based on RANO as determined by an ICR.
• ORR in Cohort B, defined as the proportion of participants in Cohort B who achieve a BOR of CR or PR based on RANO as determined by an ICR.

• ORR nella Coorte A, definito come la percentuale di partecipanti nella Coorte A che ottengono una BOR di CR o PR in base ai criteri RANO, come determinato da una ICR.
• ORR nella Coorte B, definito come la percentuale di partecipanti nella Coorte B che ottengono una BOR di CR o PR in base ai criteri RANO, come determinato da una ICR.

E.5.1.1

Timepoint(s) of evaluation of this end point

The schedule for assessments will be performed according to the schedule of activities reported in the protocol.
Adverse events will be monitored from the time the participant signs the ICF until at least 30 days after the last dose of study treatment.

Il programma delle valutazioni sarà svolto secondo il programma delle attività indicato nel protocollo.
Gli eventi avversi saranno monitorati dal momento della firma dell’ICF da parte del/la partecipante fino ad almeno 30 giorni dopo l’ultima dose del trattamento dello studio.

E.5.2

Secondary end point(s)

• ORR in Cohorts A and B combined, defined as the proportion of participants in Cohorts A and B who achieve a BOR of CR or PR based on RANO as determined by an ICR.
• ORR in Cohorts A, B, and C combined, defined as the proportion of participants in Cohorts A, B, and C who achieve a BOR of CR or PR based on RANO as determined by an ICR.
• ORR in Cohort C, defined as the proportion of participants in Cohort C who achieve a BOR of CR or PR based on RANO as determined by an ICR.
• ORR in each cohort as determined by investigator assessment.
• DCR in Cohorts A and B, respectively, described as the proportion of participants who achieve a CR, PR, or SD as assessed by an ICR.
• PFS in Cohorts A and B, respectively, defined as the time from first dose until progressive disease (according to RANO and assessed by an ICR) or death (whichever occurs first).
• DOR in Cohorts A and B, respectively, defined as the time from the date of first assessment of CR or PR until the date of the first progressive disease (according to RANO and
assessed by an ICR), or death (whichever is first).
• OS in Cohorts A and B, respectively, defined as the time from first dose of study drug to death of any cause.
• Safety and tolerability in each cohort, assessed by monitoring the frequency and severity of AEs by performing physical examinations, evaluating changes in vital signs and ECGs,
and evaluating clinical laboratory blood samples according to NCI CTCAE v5.0.
• Impact on study treatment, assessed by monitoring the frequency of treatment interruptions, dose reductions, and withdrawal of study treatment due to AEs.

• ORR nelle Coorti A e B combinate, definito come la percentuale di partecipanti nelle Coorti A e B che ottengono una BOR di CR o PR in base ai criteri RANO, come determinato da una ICR.
• ORR nelle Coorti A, B e C combinate, definito come la percentuale di partecipanti nelle Coorti A, B e C che ottengono una BOR di CR o PR in base ai criteri RANO, come determinato da una ICR.
• ORR nella Coorte C, definito come la percentuale di partecipanti nella Coorte C che ottengono una BOR di CR o PR in base ai criteri RANO, come determinato da una ICR.
• ORR in ciascuna coorte come determinato dalla valutazione dello sperimentatore.
• DCR nelle Coorti A e B, rispettivamente, descritto come la percentuale di partecipanti che raggiungono una CR, PR o SD come valutata da una ICR.
• PFS nelle Coorti A e B, rispettivamente, definita come il tempo trascorso dalla prima dose alla progressione della malattia (secondo i criteri RANO e valutati da una ICR) o al decesso (a seconda di quale evento si verifichi per primo).
• DOR nelle Coorti A e B, rispettivamente, definita come il tempo trascorso dalla data della prima valutazione di CR o PR fino alla data della prima progressione della malattia (secondo i criteri RANO e valutata da una ICR) o al decesso (a seconda di quale evento si verifichi per primo).
• OS nelle Coorti A e B, rispettivamente, definita come il tempo trascorso dalla prima dose del farmaco dello studio al decesso per qualsiasi causa.
• Sicurezza e tollerabilità in ciascuna coorte, valutate monitorando la frequenza e la severità degli EA eseguendo esami obiettivi, valutando le variazioni nei segni vitali e degli ECG e valutando i campioni di sangue di laboratorio clinico secondo i criteri NCI CTCAE v5.0.
• Impatto sul trattamento dello studio, valutato monitorando la frequenza delle interruzioni del trattamento, le riduzioni della dose e il ritiro del trattamento dello studio a causa degli EA.

E.5.2.1

Timepoint(s) of evaluation of this end point

The schedule for assessments will be performed according to the schedule of activities reported in the protocol

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

in aperto

open

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Japan

United States

Denmark

France

Germany

Italy

United Kingdom

Netherlands

Spain

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

132

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

189

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Upon completion of this study, for participants who are continuing to receive study drug and benefiting from treatment with pemigatinib, the rollover study, INCB 54828-801, is available.

Al completamento di questo studio, per i partecipanti che stanno continuando a ricevere il farmaco dello studio e che stanno traendo benefici dal trattamento con pemigatinib, è disponibile lo studio di rollover, INCB 54828-801.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2022-04-13

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-03-17

P. End of Trial
P.	End of Trial Status	Ongoing

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IMP with orphan designation in the indication
Orphan Designation Number:
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