Clinical Trials Register

Summary
EudraCT Number:	2021-004934-12
Sponsor's Protocol Code Number:	INCB54828-210
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-03-11
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

Spain - AEMPS

A.2

EudraCT number

2021-004934-12

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 Advanced Non–Small Cell Lung Cancer With an FGFR Alteration Who Progressed on Previous Therapy (FIGHT-210)

Estudio de fase II, abierto, de un solo grupo y multicéntrico para evaluar la eficacia y la seguridad de pemigatinib en participantes con cáncer pulmonar no microcítico avanzado con una alteración del receptor del factor de crecimiento fibroblástico que progresó en el tratamiento anterior (FIGHT-210)

A.3.1

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

A.4.1

Sponsor's protocol code number

INCB54828-210

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	+34 911594080
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.
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	4.5
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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.
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	9.0
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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.
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	13.5
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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 squamous or nonsquamous NSCLC with a documented FGFR1-3 mutations or fusions/rearrangement who have progressed on prior therapies and have no available standard treatment options

Participantes de ambos sexos de 18 años de edad como mínimo, que padezcan CPNM de células escamosas o no escamosas con mutaciones o fusiones/reordenamientos documentados del FGFR1-3 que hayan progresado con tratamientos anteriores y no tengan opciones de tratamiento estándar disponibles.

E.1.1.1

Medical condition in easily understood language

Lung cancer participants with documented FGFR1-3 mutation, who have progressed on prior therapies and have no available standard treatment options

Participantes con cáncer de pulmón con mutación FGFR1-3 documentada, que han progresado con terapias anteriores y no tienen opciones de tratamiento estándar disponibles

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	PT
E.1.2	Classification code	10061873
E.1.2	Term	Non-small cell lung cancer
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To determine the efficacy of pemigatinib in participants in Cohort A

Determinar la eficacia de pemigatinib en los participantes de la cohorte A.

E.2.2

Secondary objectives of the trial

1. To evaluate the efficacy of pemigatinib in participants in Cohort B
2. To evaluate the efficacy of pemigatinib in Cohort A
3. To assess safety and tolerability of pemigatinib in all participants

Evaluar la eficacia de pemigatinib en los participantes de la cohorte B.
Evaluar la eficacia de pemigatinib en la cohorte A.
Evaluar la seguridad y la tolerabilidad de pemigatinib en todos los participantes.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Age 18 years or older, inclusive at the time of signing the ICF.
2. Histologically or cytologically confirmed advanced or metastatic NSCLC (Stage IIIB/C or IV per the AJCC Cancer Staging Manual, 8th Editiion). Both squamous and nonsquamous NSCLC are eligible.
3. Radiographically measurable disease (per RECIST v1.1). 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.
4. Documentation of known/likely actionable known or likely FGFR1-3 alterations.
5. Must have objective documented progression after at least 1 prior therapy, and must have no therapy available that is likely to provide clinical benefit. Participants who are intolerant of or decline the approved therapy are eligible only if they have no therapy available that is likely to provide clinical benefit.
6. ECOG performance status of 0 to 2.
7. Baseline archival tumor specimen (if less than 24 months from date of screening) or willingness to undergo a pretreatment tumor biopsy to obtain the specimen. Must be a tumor block or approximately 15 unstained slides from biopsy or resection of primary tumor or metastasis.
8. Willingness to avoid pregnancy or fathering children based on the criteria below.
a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children from screening through 90 days (a spermatogenesis cycle) after the last dose of study drug and must refrain from donating sperm during this period. Permitted methods in preventing pregnancy should be communicated to the participants and their understanding confirmed.
b. Female participants who are WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test before the first dose on Day 1 and must agree to take appropriate precautions to avoid pregnancy from screening through 30 days (1 menstrual cycle) after the last dose of study drug and must refrain from donating oocytes during this period. Permitted methods in preventing pregnancy should be communicated to the participants and their understanding confirmed.
c. A female participant not considered to be of childbearing potential is eligible.

1. 18 años de edad o mayores, inclusive en el momento de la firma del FCI.
2. CPNM avanzado o metastásico confirmado histológica o citológicamente (estadio IIIB/C o IV según el Manual de Estadificación del Cáncer de la AJCC, 8.ª edición [Rami-Porta et al 2017]). Son aptos tanto el CPNM escamoso como no escamoso.
3. Enfermedad medible radiográficamente (según los criterios RECIST v1.1). Las lesiones tumorales localizadas en una zona previamente irradiada o en una zona sometida a otro tratamiento locorregional se consideran medibles si la progresión se ha demostrado claramente en la lesión.
4. Documentación de alteraciones conocidas o probables del FGFR1-3 (consulte el Apéndice B).
5. Debe presentar progresión documentada objetiva después de al menos 1 tratamiento previo y no debe disponer de ningún tratamiento que pueda aportar un beneficio clínico. Los participantes que sean intolerantes o que rechacen el tratamiento aprobado son aptos para participar solo si no disponen de un tratamiento que pueda aportar un beneficio clínico.
6. Estado general según ECOG de 0 a 2.
7. Muestra tumoral de archivo inicial (si se obtuvo menos de 24 meses a partir de la fecha de selección) o voluntad de someterse a una biopsia tumoral previa al tratamiento para obtener la muestra. Debe ser un bloque tumoral o aproximadamente 15 portaobjetos sin tinción de biopsia o resección del tumor primario o metástasis.
8. Voluntad de prevenir embarazos o engendrar hijos en función de los criterios que aparecen a continuación.
a. Los participantes de sexo masculino con potencial reproductivo deben aceptar tomar las precauciones adecuadas para evitar engendrar hijos desde la selección hasta 90 días (un ciclo de espermatogénesis) después de la última dosis del fármaco del estudio y deben abstenerse de donar esperma durante este periodo. Los métodos permitidos para prevenir embarazos (consulte el Apéndice A) se deben comunicar a los participantes y se debe confirmar su comprensión.
b. Las participantes de sexo femenino en edad fértil deben tener una prueba del embarazo en suero negativa en la selección y una prueba del embarazo en orina negativa antes de la primera dosis el día 1 y deben aceptar tomar las precauciones adecuadas para prevenir embarazos desde la selección hasta 30 días (1 ciclo menstrual) después de la última dosis del fármaco del estudio, y deben abstenerse de donar ovocitos durante este periodo. Los métodos permitidos para prevenir embarazos (consulte el Apéndice A) se deben comunicar a los participantes y se debe confirmar su comprensión.
c. Una participante de sexo femenino no considerada en edad fértil según se define en el Apéndice A es apta para participar.

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 the first dose of pemigatinib. Participants must have recovered (≤ Grade 1 as per CTCAE v5.0 or at pretreatment baseline) from AEs from previously administered therapies (excluding alopecia).
3. Concurrent anticancer therapy (eg, chemotherapy, immunotherapy, biologic therapy, hormonal therapy, or investigational therapy).
4. Candidate for potentially curative surgery.
5. Current evidence of clinically significant corneal (including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis) or retinal disorder (including but not limited to macular/retinal degeneration, diabetic retinopathy, and retinal detachment) as confirmed by ophthalmologic examination.
6. Radiation therapy administered for the treatment of cancer lesions within 2 weeks before enrollment/first dose of study drug. Participants must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. Evidence of fibrosis within a radiation field from prior radiotherapy is permitted with medical monitor approval. A 1-week washout is permitted for palliative radiation to non-CNS disease.
7. Untreated brain or CNS metastases or brain or CNS metastases that have progressed (eg, evidence of new or enlarging brain metastasis or new neurological symptoms attributable to brain or CNS metastases). Participants who have previously treated and clinically stable brain or CNS metastases are eligible if there is no evidence of progression for at least 4 weeks after CNS-directed treatment, as ascertained by clinical examination and brain imaging (MRI or CT scan) during the screening period, and if they are on a stable or decreasing dose of corticosteroids for at least 1 week.
8. Known additional malignancy that is progressing or requires active 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.
9. Participants with laboratory values at screening defined in Table 5.
Rest in the protocol

1. Administración previa de un inhibidor selectivo del FGFR.
2. Haber recibido medicamentos antineoplásicos o fármacos en investigación para cualquier indicación o por cualquier motivo en el plazo de 28 días antes de la primera dosis de pemigatinib. Los participantes deben haberse recuperado (≤ grado 1 según los CTCAE v5.0 o al valor inicial antes del tratamiento) de los AA causados por los tratamientos administrados previamente (excepto la alopecia).
3. Tratamiento antineoplásico concomitante (p. ej., quimioterapia, inmunoterapia, tratamiento biológico, tratamiento hormonal o tratamiento en investigación).
4. Candidato para cirugía potencialmente curativa.
5. Pruebas actuales de trastorno clínicamente significativo de la córnea (incluidos, entre otros, queratopatía bullosa o en banda, abrasión de la córnea, inflamación o ulceración y queratoconjuntivitis) o de la retina (incluidos, entre otros, degeneración macular o retiniana, retinopatía diabética y desprendimiento de la retina) confirmado mediante exploración oftalmológica.
6. Radioterapia administrada para el tratamiento de las lesiones cancerosas en el plazo de 2 semanas antes de la inscripción o de la primera dosis del fármaco del estudio. Los participantes deben haberse recuperado de todas
las toxicidades relacionadas con la radiación, no requerir corticoesteroides y no haber presentado neumonitis por radiación. Se permiten indicios de fibrosis en un campo de radiación de la radioterapia previa con la aprobación del monitor médico. Se permite reposo farmacológico de 1 semana en el caso de radioterapia paliativa para enfermedad que no sea del SNC.
7. Metástasis cerebrales o en el SNC no tratadas o metástasis cerebrales o en el SNC que hayan progresado (p. ej., indicios de metástasis cerebral nueva o que haya aumentado de tamaño o síntomas neurológicos nuevos atribuibles a metástasis cerebrales o en el SNC). Los participantes que hayan recibido tratamiento previo y presenten metástasis cerebrales o en el SNC clínicamente estables son aptos para participar si no hay indicios de progresión durante al menos 4 semanas después del tratamiento dirigido al SNC, según lo determine
la exploración clínica y la obtención de imágenes del cerebro (RM o TAC) durante el periodo de selección y si reciben una dosis estable o decreciente de corticoesteroides durante al menos 1 semana.
8. Neoplasia maligna adicional conocida, que esté progresando o necesite tratamiento activo. Son excepciones el carcinoma basocelular de la piel, el carcinoma de células escamosas de la piel o el cáncer de cuello uterino localizado que se haya sometido a tratamiento potencialmente curativo.
9. Participantes con los valores analíticos en la selección definidos en la tabla 5.
10. Antecedentes de trastorno de la hemostasia del calcio y el fosfato o desequilibrio mineral sistémico con calcificación ectópica de tejidos blandos (excepción: calcificaciones frecuentemente observadas en tejidos blandos como piel, tendón renal o vasos sanguíneos debidas a lesiones, enfermedad o envejecimiento en ausencia de desequilibrio mineral sistémico).
11. Afecciones o trastornos gastrointestinales que pueden aumentar el pH gástrico o del intestino delgado que podrían interferir en la absorción, el metabolismo o la excreción de pemigatinib.
12. Incapacidad para tragar y para retener medicamentos por vía oral.
13. Enfermedad cardíaca clínicamente significativa o no controlada, incluida angina inestable, infarto agudo de miocardio en los 6 meses anteriores al día 1 de la administración del fármaco del estudio, insuficiencia cardíaca congestiva de clase III o IV según la New York Heart Association y arritmia no controlada (se permiten participantes con marcapasos o con fibrilación auricular y frecuencia cardíaca bien controlada).
14. Antecedentes de un ECG anormal que, en opinión del investigador, sea clínicamente significativo. Se excluye un intervalo QTcF >480 milisegundos en la selección. En el caso de los participantes con un retraso de la conducción intraventricular (intervalo QRS >120 ms), se puede utilizar el intervalo JTc en vez del intervalo QTcF con la aprobación del monitor médico. El JTc deberá ser ≤340 milisegundos si se utiliza el JTc en vez del QTcF.
15. Enfermedad infecciosa crónica o actual activa que requiera tratamiento con antibióticos sistémicos, antifúngicos o antivirales en las 2 semanas previas a la inscripción (se permiten participantes con infecciones crónicas asintomáticas que reciban tratamiento profiláctico).
16. Indicios de infección activa por el VHB o el VHC (definido como participantes con transaminasas elevadas o cirrosis). Se permiten participantes con infección crónica por el VHB/VHC sin cirrosis y sin Se excluirá la infección por el VIH conocida con un recuento de linfocitos T CD4+ <350 células/μl. Los participantes con recuentos de linfocitos T CD4+ ≥350 células/μl generalmente deberían ser aptos para participar.
Resto en el protocolo

E.5 End points

E.5.1

Primary end point(s)

ORR in Cohort A, defined as the proportion of participants who achieve a CR or PR based on RECIST v1.1. Response will be determined by an ICR review.

TRO en la cohorte A, definida como la proporción de participantes que logran una RC o RP según los criterios RECIST v1.1. La respuesta la determinará la RCI.

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 drug or until the start of new anticancer therapy.

Las evaluaciones se realizaran de acuerdo al horario de actividades informado en el protocolo.
Los acontecimientos adversos se controlarán desde el momento en que el participante firma el ICF hasta al menos 30 días después de la última dosis del fármaco del estudio o hasta el inicio de una nueva terapia contra el cáncer.

E.5.2

Secondary end point(s)

• ORR in Cohort B, defined as the proportion of participants who achieve a CR or PR based on RECIST v1.1. Response will be determined by an ICR review.
• PFS in Cohort A, defined as the time from the first dose of study drug until PD (according to RECIST v1.1 as assessed by an ICR review) or death, whichever is first.
• DOR in Cohort A, defined as the time from the date of the first CR or PR until the date of the first PD (according to RECIST v1.1 as assessed by an ICR review) or death, whichever is first.
• OS in Cohort A, defined as the time from the first dose of study drug to death of any cause.
• Safety and tolerability, as assessed by the occurrence of TEAEs and treatment-related TEAEs according to NCI CTCAE v5.0, physical examination changes, vital sign changes, laboratory evaluations, and ECGs.

* TRO en la cohorte B, definida como la proporción de participantes que logran una RC o RP según los criterios RECIST v1.1. La respuesta la determinará la RCI.
• SSP en la cohorte A, definida como el tiempo desde la primera dosis del fármaco del estudio hasta la PE (según los criterios RECIST v1.1 evaluados mediante RCI) o la muerte, lo que ocurra primero.
• DR en la cohorte A, definida como el tiempo desde la fecha de la primera RC o RP hasta la fecha de la primera PE (según los criterios RECIST v1.1 evaluados mediante RCI) o la muerte, lo que ocurra primero.
• SG en la cohorte A, definida como el tiempo desde la primera dosis del fármaco del estudio hasta la muerte por cualquier causa.
Seguridad y tolerabilidad, evaluadas por la aparición de AAAT y AAAT relacionados con el tratamiento según los Criterios Comunes de Terminología para Eventos Adversos (CTCAE, por sus siglas en inglés) del Instituto Nacional del Cáncer estadounidense (NCI) versión 5.0, cambios en la exploración física, cambios en las constantes vitales, evaluaciones analíticas y ECG.

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

Yes

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

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.8.3

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

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

United States

France

Germany

Italy

Spain

E.8.7

Trial has a data monitoring committee

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

UVUP

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 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:

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

125

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 finalizar este estudio, para los participantes que continúan recibiendo el fármaco del estudio y se benefician del tratamiento con pemigatinib, el estudio de transferencia, INCB 54828-801, está disponible.

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

2022-05-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-05-13

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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