Clinical Trials Register

Summary
EudraCT Number:	2017-002541-29
Sponsor's Protocol Code Number:	PVO-1A-301
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Restarted
Date on which this record was first entered in the EudraCT database:	2021-01-26
Trial results	View 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

2017-002541-29

A.3

Full title of the trial

A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)

Studio di fase 3 sull¿efficacia e sicurezza di palovarotene orale per il trattamento della Fibrodisplasia Ossificante Progressiva (FOP)

A.3.1

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

A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)

Sperimentazione clinica per valutare l¿efficacia e la sicurezza di palovarotene orale come trattamento per la fibrodisplasia ossificante progressiva (FOP)

A.3.2

Name or abbreviated title of the trial where available

MOVE Trial

Sperimentazione MOVE

A.4.1

Sponsor's protocol code number

PVO-1A-301

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

ISRCTN00000000

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT00000000

A.5.3

WHO Universal Trial Reference Number (UTRN)

U0000-0000-0000

A.5.4

Other Identifiers

Name:

00000000000000000000

Number:

00000000000000000000

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/121/2018

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	CLEMENTIA PHARMACEUTICALS INC.
B.1.3.4	Country	Canada
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	Clementia Pharmaceuticals Inc
B.4.2	Country	Canada
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Clementia Pharmaceuticals Inc.
B.5.2	Functional name of contact point	Clinical Trials Information
B.5.3	Address:
B.5.3.1	Street Address	1000, De La Gauchetière, Suite 1200
B.5.3.2	Town/ city	Montreal, Quebec
B.5.3.3	Post code	H3Z2Y5
B.5.3.4	Country	Canada
B.5.4	Telephone number	0015142283630
B.5.5	Fax number	0018889660135
B.5.6	E-mail	clinicaltrials@clementiapharma.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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE
D.3.2	Product code	not assigned
D.3.4	Pharmaceutical form	Capsule, hard
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	Palovarotene
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not assigned
D.3.9.3	Other descriptive name	PALOVAROTENE
D.3.9.4	EV Substance Code	SUB75998
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	1
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE 1.5 mg
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not assigned
D.3.9.3	Other descriptive name	Palovarotene
D.3.9.4	EV Substance Code	SUB75998
D.3.10	Strength
D.3.10.1	Concentration unit	Bq/µg becquerel(s)/microgram
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1500
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE-2 mg
D.3.2	Product code	[not assigned]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not assigned
D.3.9.3	Other descriptive name	Palovarotene
D.3.9.4	EV Substance Code	SUB75998
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	2
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: 4
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	palovarotene
D.3.9.4	EV Substance Code	SUB75998
D.3.10	Strength
D.3.10.1	Concentration unit	Bq/µg becquerel(s)/microgram
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25000
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: 5
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE-3 mg
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	Palovarotene
D.3.9.4	EV Substance Code	SUB75998
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	3
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: 6
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE-4 mg
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	PALOVAROTENE
D.3.9.4	EV Substance Code	SUB75998
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: 7
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE-5mg
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	PALOVAROTENE
D.3.9.4	EV Substance Code	SUB75998
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	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	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: 8
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	No
D.2.1.1.1	Trade name	not applicable
D.2.1.1.2	Name of the Marketing Authorisation holder	not applicable
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1368
D.3 Description of the IMP
D.3.1	Product name	PALOVAROTENE-10 mg
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Capsule, hard
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	PALOVAROTENE
D.3.9.1	CAS number	410528-02-8
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	Palovarotene
D.3.9.4	EV Substance Code	SUB75998
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	10
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: 9
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	EMLA - 2.5% + 2.5% CREMA TUBO 30 G
D.2.1.1.2	Name of the Marketing Authorisation holder	ASTRAZENECA S.P.A.
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	EMLA
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Cream
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Cutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	LIDOCAINA
D.3.9.1	CAS number	137-58-6
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	Lidocaine
D.3.9.4	EV Substance Code	SUB178322
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	25
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PRILOCAINA
D.3.9.1	CAS number	721-50-6
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	Prilocaina
D.3.9.4	EV Substance Code	SUB10041MIG
D.3.10	Strength
D.3.10.1	Concentration unit	g/ml gram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
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: 10
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	IDROCORTISONE ACETATO DYNACREN - 1% CREMA 1 TUBO DA 30 G
D.2.1.1.2	Name of the Marketing Authorisation holder	DYNACREN LABORATORIO FARMACEUTICO DEL DOTT.A.FRANCIONI E DI M.GEROSA S.R.L.
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	IDROCORTISONE ACETATO DYNACREN 1% CREMA.
D.3.2	Product code	[not applicable]
D.3.4	Pharmaceutical form	Cream
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Cutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	IDROCORTISONE ACETATO
D.3.9.1	CAS number	50-03-3
D.3.9.2	Current sponsor code	not applicable
D.3.9.3	Other descriptive name	IDROCORTISONE ACETATO
D.3.9.4	EV Substance Code	SUB02562MIG
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1
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

Fibrodysplasia Ossificans Progressiva (FOP)

Fibrodisplasia ossificante progressiva (FOP)

E.1.1.1

Medical condition in easily understood language

FOP is a rare, severely disabling disease characterized by heterotopic ossification in muscles, tendons, and ligaments often associated with painful and recurrent episodes of soft tissue swelling.

FOP rara malattia invalidante caratterizzata da ossificazione eterotopica a livello di muscoli, tendini e legamenti, associata a dolorosi episodi ricorrenti di gonfiore a carico dei tes molli.

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10068715
E.1.2	Term	Fibrodysplasia ossificans progressiva
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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 evaluate the efficacy of palovarotene in decreasing heterotopic ossification (HO) in adult and pediatric subjects with FOP as assessed by low-dose, whole body computed tomography (WBCT), excluding head, as compared to untreated subjects from Clementia¿s FOP natural history study over 24 months.
¿ To evaluate the safety of palovarotene in adult and pediatric subjects with FOP.

¿ Valutare l¿efficacia di palovarotene nel ridurre l¿ossificazione eterotopica (HO) in soggetti adulti e pediatrici con FOP valutata mediante tomografia computerizzata dell¿intero corpo (WBCT) a basse dosi, esclusa la testa, rispetto ai soggetti non trattati di uno studio di storia naturale della FOP sponsorizzato da Clementia nell’arco di 24 mesi.
¿ Valutare la sicurezza di palovarotene in soggetti adulti e pediatrici con FOP.

E.2.2

Secondary objectives of the trial

- To evaluate the effect of palovarotene on flare-up rate and proportion of subjects reporting at least one flare-up.
- To evaluate the effect of palovarotene on range of motion (ROM) as assessed by the Cumulative Analogue Joint Involvement Scale for FOP (CAJIS).
- To evaluate the effect of palovarotene on physical function using age appropriate forms of the FOP Physical Function Questionnaire (FOP PFQ).
- To evaluate the effect of palovarotene on physical and mental health using age-appropriate forms of the Patient Reported Outcomes Measurement Information System (PROMIS) Global Health Scale.
- To evaluate the pharmacokinetics of palovarotene.
Secondary Objective (Part B)
- To continue to provide palovarotene to adult and pediatric subjects with FOP and to monitor longer-term safety.

- Valutare l¿effetto di palovarotene sul tasso di riacutizzazioni e sulla percentuale di soggetti che riportano almeno una riacutizzazione.
- Valutare l¿effetto di palovarotene sul raggio di movimento (ROM) valutato mediante la Scala analogica cumulativa del coinvolgimento articolare (CAJIS) per la FOP.
- Valutare l¿effetto di palovarotene sulla funzione fisica utilizzando moduli del Questionario sulla funzione fisica nella FOP (FOP-PFQ) adeguati all¿et¿.
- Valutare l¿effetto di palovarotene sulla salute fisica e mentale utilizzando moduli della Scala sulla salute globale del Sistema informativo di misurazione degli esiti riferiti dal paziente (PROMIS) adeguati all¿et¿.
- Valutare la farmacocinetica di palovarotene.
Obiettivo secondario (Parte B)
• Continuare a fornire palovarotene a soggetti adulti e pediatrici con FOP e a monitorare la sicurezza a lungo termine.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Subjects must meet all of the following criteria to be eligible for enrollment: 1. Written, signed, and dated informed subject/parent consent; and for subjects who are minors, age-appropriate assent (performed according to local regulations).
2. Male or female at least 4 years of age. 3. Previous participation in the NHS; or clinically diagnosed with FOP, with the R206H ACVR1 mutationor other FOP variants reported to be
associated with progressive HO (who have not previously participated in
any Clementia sponsored study); or participants in Study PVO 1A 202 or
Study PVO-1A-204 who cannot currently receive the chronic/flare-up
regimen due to country of residence or those traveling long distances to participate in the Phase 2 trial.
4. No flare-up symptoms within the past 4 weeks, including at the time of enrollment.
5. Females of child-bearing potential must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of palovarotene. Male and FOCBP subjects must agree to remain abstinent from heterosexual sex during treatment and for 1 month after treatment or, if sexually active, to use two highly effective methods of birth control during and for 1 month after treatment. Additionally, sexually active FOCBP subjects must already be using two highly effective methods of birth control 1 month before treatment is to start. Specific risk of the use of retinoids during pregnancy, and the agreement to remain abstinent or use two highly effective methods of birth control will be clearly defined in the informed consent and the subject or legally authorized representatives (eg, parents, caregivers, or legal guardians) must specifically sign this section.
6. Must be accessible for treatment and follow-up, and be able to undergo all study procedures. Subjects living at distant locations from the investigational site must be able and willing to travel to a site for the initial and all on-site follow-up visits. Subjects must be able to undergo low-dose, WBCT (excluding head)without sedation

1. Consenso informato del soggetto/genitore scritto, firmato e datato e, per i minori, assenso adeguato all’età (rilasciato secondo le normative locali). 2. Soggetti di sesso maschile o femminile di almeno 4 anni di età.
3. Precedente partecipazione all’NHS o Diagnosi clinica di FOP, con la mutazione R206H ACVR1 o diagnosi clinica di FOP, con la mutazione R206H ACVR1 o altre varianti di FOP segnalate come associate a HO progressiva (che non hanno partecipato in precedenza ad alcuno studio sponsorizzato da Clementia); o partecipanti allo studio PVO-1A-202 o allo studio PVO-1A-204 che attualmente non possono ricevere il regime di trattamento cronico/basato sulle riacutizzazioni a causa del Paese di residenza o coloro i quali percorrono distanze considerevoli per partecipare alla Sperimentazione di Fase 2. 4. Nessun sintomo di riacutizzazione nelle ultime 4 settimane, compreso il momento dell’arruolamento.
5. Le donne in età fertile devono risultare negative al test di gravidanza sulle urine o sul sangue (con sensibilità di almeno 50 mUI/ml) prima della somministrazione di palovarotene. Le donne e i soggetti FOCBP devono acconsentire a praticare l’astinenza da rapporti eterosessuali durante il trattamento e per 1 mese dopo il trattamento oppure, se sessualmente attive, ad utilizzare due metodi contraccettivi altamente efficaci durante il trattamento e per 1 mese dopo lo stesso. Inoltre, i soggetti FOCBP sessualmente attivi devono già utilizzare due metodi contraccettivi altamente efficaci 1 mese prima dell’inizio del trattamento. Il rischio specifico dell’uso di retinoidi durante la gravidanza e il consenso a praticare l’astinenza o all’uso di due metodi contraccettivi altamente efficaci saranno definiti in maniera chiara nel consenso informato e il soggetto o rappresentante legale (es. genitori, la persona che se ne prenderà cura o tutori legali) deve firmare specificatamente questa sezione.
6. Il soggetto deve essere disponibile a ricevere il trattamento e il follow-up e in grado di sottoporsi a tutte le procedure dello studio. I soggetti che vivono in luoghi distanti dal centro sperimentale devono essere in grado di e disposti a viaggiare verso il centro per le visite iniziali e tutte le visite di follow-up presso il centro. I soggetti devono essere in grado di sottoporsi alla WBCT a basse dosi (esclusa la testa).senza sedazione

E.4

Principal exclusion criteria

1. Weight <10 kg. 2. If currently using vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, or herbal preparations, fish oil, and unable or unwilling to discontinue use of these products during palovarotene treatment.
3. Exposure to synthetic oral retinoids other than palovarotene within 4 weeks prior to screening.
4. Concurrent treatment with tetracycline or any tetracycline derivatives due to the potential increased risk of pseudotumor cerebri.
5. History of allergy or hypersensitivity to retinoids, gelatin or lactose (note that lactose intolerance is not exclusionary).
6. Concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity; or kinase inhibitors such as imatinib (see Section 5.2.1).
7. Amylase or lipase >2x above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
8. Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5x ULN.
9. Fasting triglycerides >400 mg/dL with or without therapy.
10. Female subjects who are breastfeeding.
11. Subjects with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.
12. Subjects experiencing suicidal ideation (type 4 or 5) or any suicidal behavior within the past month as defined by the Columbia-Suicide Severity Rating Scale (C-SSRS).
13. Simultaneous participation in another investigational clinical research study (other than palovarotene studies) within 4 weeks prior to Screening; or within five half-lives of the agent, whichever is longer.
14. Any reason that, in the opinion of the Investigator, would lead to the inability of the subject and/or family to comply with the protocol

1. Peso <10 kg. 2. Uso corrente di vitamina A o beta carotene, multivitamine contenenti vitamina A o beta carotene o preparazioni erboristiche, olio di pesce e incapacità o riluttanza a interrompere l’uso di questi prodotti durante il trattamento con palovarotene.
3. Esposizione a retinoidi orali sintetici diversi da palovarotene nelle 4 settimane precedenti lo screening. 4. Trattamento concomitante con tetraciclina o derivati della tetraciclina a causa del potenziale aumento del rischio di pseudotumor cerebri.
5. Anamnesi di allergia o ipersensibilità a retinoidi, gelatina o lattosio (si noti che l’intolleranza al lattosio non determina l’esclusione).
6. Farmaci concomitanti che agiscono da forti inibitori o induttori dell’attività della subunità 3A4 del citocromo P450 (CYP450), oppure inibitori delle chinasi come imatinib (si veda la Sezione 5.2.1).
7. Amilasi o lipasi >2 volte il limite superiore della norma (ULN) o con un’anamnesi di pancreatite cronica.
8. Valori elevati di aspartato aminotransferasi (AST) o alanina aminotransferasi (ALT) >2,5 x ULN.
9. Trigliceridi a digiuno >400 mg/dl con o senza terapia.10. Soggetti di sesso femminile che allattano al seno.11. Soggetti con malattia cardiovascolare, epatica, polmonare, gastrointestinale, endocrina, metabolica, oftalmica, immunologica, psichiatrica non controllata o altra malattia significativa.
12. Soggetti che manifestano ideazione suicidaria (di tipo 4 o 5) o qualsiasi comportamento suicidario nell’ultimo mese, secondo quanto definito dalla Scala della Columbia University per la valutazione della gravità del rischio di suicidio (C-SSRS).
13. Partecipazione simultanea ad un altro studio interventivo di ricerca clinica interventivo (diverso dagli studi su palovarotene)nelle 4 settimane precedenti lo screening o nelle cinque emivite dell’agente sperimentale, a seconda di quale evento duri più a lungo.
14. Qualsiasi motivo che, nell’opinione dello sperimentatore, comporterebbe l’incapacità del soggetto e/o della famiglia di attenersi al protocollo

E.5 End points

E.5.1

Primary end point(s)

The annualized change in new HO volume as assessed by low-dose, WBCT (excluding head) compared to untreated subjects from the NHS over 24 months.

Variazione nell’anno nel volume di nuova HO valutata mediante WBCT a basse dosi (esclusa la testa) rispetto ai soggetti non trattati dell’NHS nell'arco di 24 mesi.

E.5.1.1

Timepoint(s) of evaluation of this end point

Month 6, 12, 18 and 24 months (final analysis). It will also being evaluated in supportive analysis at 36 and 48 months.

Mese 6, 12, 18 e 24 mesi (analisi finale). Sarà inoltre valutato in analisi di supporto a 36 e 48 mesi.

E.5.2

Secondary end point(s)

1. The proportion of subjects with any new HO.
2. The change from baseline in the number of body regions with new HO.
3. The proportion of subjects reporting flare-ups.
4. The flare-up rate per subject-month exposure.

1. Percentuale di soggetti con qualsiasi nuova HO.
2. Variazione rispetto al basale nel numero di regioni corporee con nuova HO.
3. Percentuale di soggetti che riportano riacutizzazioni.
4. Tasso di riacutizzazioni per soggetto-mese di esposizione.

E.5.2.1

Timepoint(s) of evaluation of this end point

Month 6, 12, 18 and 24 months (final analysis). It will also being evaluated in supportive analysis at 36 and 48 months.

Mese 6, 12, 18 e 24 mesi (analisi finale). Sarà inoltre valutato in analisi di supporto a 36 e 48 mesi.

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

E.6.7

Pharmacodynamic

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

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

Yes

E.8.1.7.1

Other trial design description

Lo studio ha un controllo esterno (studio NHS-soggetti non trattati)

The trial has en external control (NHS study - untreated subjects)

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

Yes

E.8.2.3.1

Comparator description

Lo studio ha un controllo esterno (studio NHS-soggetti non trattati)

The trial has en external control (NHS study - untreated subjects)

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

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

Argentina

Australia

Brazil

Canada

Japan

Korea, Democratic People's Republic of

Korea, Republic of

Russian Federation

South Africa

United States

France

Germany

Italy

Netherlands

Spain

Sweden

United Kingdom

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

Yes

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

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)

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

subjects of study are male of female at least 4 years old

i soggetti di studio sono di sesso maschile e femminile di almeno 4 anni

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

107

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)

risk/benefit profile based on the emerging Phase 3 data warrants it, subjects will be offered to continue treatment until commercial availability of the product in a subject's country of residence.

profilo rischi/benefici basato sui dati di fase 3 emergenti lo giustifica, i soggetti avranno la possibilit¿ di continuare il trattamento fino alla disponibilit¿ commerciale del prodotto nel proprio paese di residenza.

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

2018-03-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-02-12

P. End of Trial
P.	End of Trial Status	Restarted

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