Clinical Trials Register

Summary
EudraCT Number:	2021-000138-33
Sponsor's Protocol Code Number:	R4461-PLD-20100
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-10-29
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

France - ANSM

A.2

EudraCT number

2021-000138-33

A.3

Full title of the trial

A Randomized Double-Blind Placebo-Controlled Study of the LEPR Agonist Antibody REGN4461 for the Treatment of Metabolic Abnormalities in Patients with Familial Partial Lipodystrophy

Étude randomisée, en double aveugle, contrôlée contre placebo, de l’anticorps REGN4461 agoniste du récepteur à la leptine, dans le traitement des anomalies métaboliques chez des patients atteints de lipodystrophie partielle familiale

A.3.1

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

A study to examine the effects of the LEPR Agonist Antibody REGN4461 in patients with Familial Partial Lipodystrophy (FPLD)

Étude qui évalue les effets de l’anticorps REGN4461 agoniste du récepteur à la leptine chez des patients atteints de lipodystrophie partielle familiale (LDPF)

A.3.2

Name or abbreviated title of the trial where available

LEAP

A.4.1

Sponsor's protocol code number

R4461-PLD-20100

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	Regeneron Pharmaceuticals, Inc.
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	Regeneron Pharmaceuticals, Inc.
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	Regeneron Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Clinical Trial Information
B.5.3	Address:
B.5.3.1	Street Address	777 Old Saw Mill River Road
B.5.3.2	Town/ city	Tarrytown
B.5.3.3	Post code	10591
B.5.3.4	Country	United States
B.5.6	E-mail	clinicaltrials@regeneron.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.2.5	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	REGN4461
D.3.2	Product code	REGN4461
D.3.4	Pharmaceutical form	Lyophilisate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	REGN4461
D.3.9.1	CAS number	2305770-44-7
D.3.9.2	Current sponsor code	REGN4461
D.3.9.3	Other descriptive name	REGN4461
D.3.9.4	EV Substance Code	SUB191249
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	265
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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.2.5	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	REGN4461
D.3.2	Product code	REGN4461
D.3.4	Pharmaceutical form	Lyophilisate for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	REGN4461
D.3.9.1	CAS number	2305770-44-7
D.3.9.2	Current sponsor code	REGN4461
D.3.9.3	Other descriptive name	REGN4461
D.3.9.4	EV Substance Code	SUB191249
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	265
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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.2.5	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	REGN4461
D.3.2	Product code	REGN4461
D.3.4	Pharmaceutical form	Solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	REGN4461
D.3.9.3	Other descriptive name	REGN4461
D.3.9.4	EV Substance Code	SUB191249
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	175
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Lyophilisate for solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Lyophilisate for solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use
D.8 Placebo: 3
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection/infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Familial Partial Lipodystrophy

Lipodystrophie partielle familiale

E.1.1.1

Medical condition in easily understood language

Familial Partial Lipodystrophy

lipodystrophie partielle familiale

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

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	10053857
E.1.2	Term	Partial lipodystrophy
E.1.2	System Organ Class	10040785 - Skin and subcutaneous tissue 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

Two cohorts are being studied based on leptin levels. Cohort A is composed of patients with baseline leptin <8.0 ng/mL and Cohort B is composed of patients with baseline leptin 8.0 to ≤20.0 ng/mL

The primary objectives will be evaluated for patients in Cohort A only:
• To evaluate the effect of REGN4461 on fasting triglycerides (TG) in patients with elevated baseline fasting TG
• To evaluate the effect of REGN4461 on hyperglycemia in patients with elevated baseline HbA1c

Deux cohortes seront évalués en fonction des taux de leptine. La cohorte A est composée de patients avec un taux initial de leptine < 8,0 ng/ml
et la cohorte B est composée de patients avec un taux initial de leptine compris entre 8,0 et ≤ 20,0 ng/ml
Les objectifs principaux ne seront évalués que chez les patients de la cohorte A :
• Évaluer l'effet de REGN4461 sur le taux de TG à jeun chez les patients présentant un taux initial élevé de TG à jeun
• Évaluer l'effet de REGN4461 sur l’hyperglycémie chez les patients présentant un taux initial élevé HbA1c

E.2.2

Secondary objectives of the trial

The following secondary objectives of the study will be evaluated for Cohort B and for the combined set of Cohorts A plus B:
• To evaluate the effect of REGN4461 on fasting TG levels in patients with hypertriglyceridemia
• To evaluate the effect of REGN4461 on glycemic control in patients with hyperglycemia
The following secondary objectives of the study will be evaluated for Cohorts A and B separately, and for the combined set of Cohorts A plus B:
• To evaluate the effect of REGN4461 on liver fat in patients with hepatic steatosis
• To evaluate the effect of REGN4461 on hunger
• To evaluate safety and tolerability of REGN4461
• To characterize the concentration profile of REGN4461 over time
• To assess immunogenicity to REGN4461

Les objectifs secondaires suivants de l'étude seront évalués dans la cohorte B et dans les cohortes A et B combinées :
• Évaluer l'effet de REGN4461 sur le taux de TG à jeun chez les patients atteints d'hypertriglycéridémie
• Évaluer l'effet de REGN4461 sur le contrôle glycémique chez les patients présentant une hyperglycémie
Les objectifs secondaires suivants de l'étude seront évalués dans les cohortes A et B séparément et dans les cohortes A et B combinées :
• Évaluer l'effet de REGN4461 sur la graisse hépatique chez les patients atteints de stéatose hépatique
• Évaluer l'effet de REGN4461 sur la faim
• Évaluer l’innocuité et la tolérance de REGN4461
• Caractériser le profil de concentrations de REGN4461 dans le temps
• Évaluer l'immunogénicité de REGN4461

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Future Biomedical Research (Optional)
Patients who agree to participate in the future biomedical research (FBR) sub-study will be required to consent to this optional sub-study before samples are banked for FBR. Additional samples will be collected for FBR.

Pharmacogenomic Analysis (Optional)
Patients who agree to participate in the genomics sub-study will be required to consent to this optional sub-study before collection of the samples. Whole blood samples for DNA extraction should be collected on day 1/baseline but can be collected at a later study visit.
DNA samples will be collected for pharmacogenomics analyses to understand the genetic determinants of efficacy and safety associated with the treatments in this study and the molecular basis of PLD and related diseases.

E.3

Principal inclusion criteria

1. Male or female, ≥18 years of age at the screening visit.
2. Clinical diagnosis of familial partial lipodystrophy as defined in the protocol
3. Fasting leptin level <=20ng/ml, as determined during the screening period
4. Presence of significant metabolic abnormalities related to glucose and triglycerides (TGs) as defined in the protocol
5. Stable body weight within the 3 months prior to screening (no gain or loss of >5% current weight)
6. Stable diet during the past 3 months defined as no major change in macronutrient composition (eg, starting or stopping diets such as Atkins, Paleo, Vegetarianism, Veganism).
7. No clinically meaningful change in medication regimen in the 3 months prior to screening as defined in the protocol

NOTE: Other protocol defined inclusion criteria apply

1. Homme ou femme âgé(e) de 18 ans ou plus lors de la visite de sélection.
2. Diagnostic clinique de lipodystrophie partielle familiale comme défini dans le protocole
3. Taux de leptine à jeun ≤ 20,0 ng/ml, comme déterminé durant la période d'inclusion
4. Présence d'anomalies métaboliques significatives relatives au taux d’HbA1c et au taux de TG, comme défini dans le protocole
5. Poids corporel stable au cours des 3 mois précédant la sélection (pas de prise ou de perte > 5 % par rapport au poids actuel)
6. Régime alimentaire stable au cours des 3 derniers mois défini comme aucun changement majeur dans la composition des macronutriments (par exemple, commencer ou arrêter des régimes tels que Adkins, Paléo, végétarien, végan).
7. Aucun changement significatif sur le plan clinique du schéma thérapeutique antidiabétique au cours des 3 mois précédant la sélection comme défini dans le protocole.

NOTE: d'autres définitions des critères d'inclusion du protocole s'appliquent

E.4

Principal exclusion criteria

1. Treatment with metreleptin within 3 months of the screening visit
2. Patients with a diagnosis of generalized lipodystrophy
3. Patients with a diagnosis of acquired lipodystrophy
4. Pregnant or breastfeeding women

NOTE: Other protocol defined exclusion criteria apply

1. Traitement par métréleptine au cours des 3 mois précédant la visite de sélection
2. Patients présentant un diagnostic de lipodystrophie généralisée
3. Patients présentant un diagnostic de lipodystrophie acquise
4. Grossesse ou allaitement.

NOTE: d'autres définitions des critères d'exclusion du protocole s'appliquent

E.5 End points

E.5.1

Primary end point(s)

Co-primary:
• Percent change in fasting serum TG from baseline to week 12 in patients with elevated baseline fasting TG and with baseline leptin <8.0 ng/mL (Cohort A)
• Absolute change in HbA1c from baseline to week 12 in patients with elevated baseline HbA1c and with baseline leptin <8.0 ng/mL (Cohort A)

Co-principaux :
• Pourcentage de changement du taux sérique de TG à jeun entre l’inclusion et la semaine 12 chez les patients présentant un taux initial élevé de TG et un taux initial de leptine < 8,0 ng/ml (cohorte A)
• Changement absolu du taux d’HbA1c entre l’inclusion et la semaine 12 chez les patients présentant un taux initial élevé d’HbA1c et un taux initial de leptine < 8,0 ng/ml (cohorte A)

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline to week 12

Entre l’inclusion et la semaine 12

E.5.2

Secondary end point(s)

The following secondary endpoints will be analyzed for Cohort B and for the combined set of Cohorts A plus B:
• Percent change in fasting serum TG from baseline to week 12 in patients with elevated baseline fasting TG
• Absolute change in HbA1c from baseline to week 12 in patients with elevated baseline HbA1c
The following secondary endpoints will be analyzed for Cohorts A and B separately, and for the combined set of Cohorts A plus B:
• Percent change in fasting serum TG from baseline to week 12 compared to the percent change between week 12 and week 24 (study arm 1)
• Percent change in fasting serum TG from baseline to week 24 (study arm 2)
• Percent change in fasting serum TG after the first 12 weeks of exposure to REGN4461— from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1; patients must meet stability criteria)
• Change in HbA1c from baseline to week 12 compared to the change between week 12 and week 24 (study arm 1)
• Change in fasting glucose from baseline to week 12 compared to the change between week 12 and week 24 (study arm 1)
• Change in HbA1c from baseline to week 24 (study arm 2)
• Change in fasting glucose from baseline to week 24 (study arm 2)
• Change in HbA1c from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1; patients must meet stability criteria)
• Change in fasting glucose from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1)
• Percent change in liver fat measured by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF) from baseline to week 12, REGN4461 versus placebo in patients with baseline liver fat (MRI-PDFF) ≥8.5%
• Percent change in liver fat (MRI-PDFF) from baseline to week 12 compared to the percent change between week 12 and week 24 (study arm 1) in patients with baseline liver fat (MRI-PDFF) ≥8.5%
• Percent change in liver fat (MRI-PDFF) from baseline to week 24 (study arm 2) in patients with baseline liver fat (MRI-PDFF) ≥8.5%
• Percent change in liver fat (MRI-PDFF) from baseline to week 12 (study arm 2) and from week 12 to week 24 (study arm 1) in patients with baseline liver fat (MRI-PDFF) ≥8.5%
• Change on the daily lipodystrophy hunger questionnaire from baseline to week 12 and week 24
• The incidence and severity of treatment-emergent adverse events (TEAEs)
• Concentrations of REGN4461 in serum over time
• Immunogenicity of REGN4461 over time compared to placebo

Les critères d’évaluation secondaires suivants seront analysés dans la cohorte B et dans les cohortes A et B combinées :
• Pourcentage de changement du taux sérique de TG à jeun entre l’inclusion et la semaine 12 chez les patients présentant un taux initial élevé de TG à jeun
• Changement absolu du taux d’HbA1c entre l’inclusion et la semaine 12 chez les patients présentant un taux initial élevé d’HbA1c
Les critères d’évaluation secondaires suivants seront analysés dans les cohortes A et B séparément et dans les cohortes A et B combinées :
• Pourcentage de changement du taux sérique de TG à jeun entre l’inclusion et la semaine 12 par rapport au pourcentage de changement entre la semaine 12 et la semaine 24 (bras 1)
• Pourcentage de changement du taux sérique de TG à jeun entre l’inclusion et la semaine 24 (bras à l’étude 2)
• Pourcentage de changement du taux sérique de TG à jeun après les 12 premières semaines d’exposition à REGN4461 : entre l’inclusion et la semaine 12 (bras 2) et entre la semaine 12 et la semaine 24 (bras 1 ; les patients doivent répondre aux critères de stabilité)
• Changement du taux d’HbA1c entre l’inclusion et la semaine 12 par rapport au changement entre la semaine 12 et la semaine 24 (bras 1)
• Changement de la glycémie à jeun entre l’inclusion et la semaine 12 par rapport au changement entre la semaine 12 et la semaine 24 (bras 1)
• Changement du taux d’HbA1c entre l’inclusion et la semaine 24 (bras 2)
• Changement de la glycémie à jeun entre l’inclusion et la semaine 24 (bras 2)
• Changement du taux d’HbA1c entre l’inclusion et la semaine 12 (bras à 2) et entre la semaine 12 et la semaine 24 (bras 1 ; les patients doivent répondre aux critères de stabilité)
• Changement de la glycémie à jeun entre l’inclusion et la semaine 12 (bras 2) et entre la semaine 12 et la semaine 24 (bras 1)
• Pourcentage de changement de la fraction de graisse hépatique (FGDP-IRM) entre l’inclusion et la semaine 12 sous REGN4461 par rapport au placebo chez les patients présentant une fraction de graisse hépatique (FGDP-IRM) initiale ≥ 8,5 %.
• Pourcentage de changement de la fraction de graisse hépatique (FGDP-IRM) entre l’inclusion et la semaine 12 par rapport au pourcentage de changement entre la semaine 12 et la semaine 24 (bras 1) chez les patients présentant une fraction de graisse hépatique (FGDP-IRM) initiale ≥ 8,5 %.
• Pourcentage de changement de la fraction de graisse hépatique (FGDP-IRM) entre l'inclusion et la semaine 24 (bras 2) chez les patients présentant une fraction de graisse hépatique (FGDP-IRM) initiale ≥ 8,5 %.
• Pourcentage de changement de la fraction de graisse hépatique (FGDP-IRM) entre l’inclusion et la semaine 12 (bras 2) et entre la semaine 12 et la semaine 24 (bras 1) chez les patients présentant une fraction de graisse hépatique (FGDP-IRM) initiale ≥ 8,5 %.
• Changement du score du questionnaire évaluant la faim quotidienne liée à la lipodystrophie entre l'inclusion et les semaines 12 et 24
• Incidence et sévérité des évènements indésirables apparus pendant le traitement (EIAT)
• Concentrations sériques de REGN4461 en fonction du temps
• Immunogénicité de REGN4461 en fonction du temps par rapport au placebo

E.5.2.1

Timepoint(s) of evaluation of this end point

As Stated Above

Comme mentionné plus haut

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)

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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

Yes

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

Yes

E.8.4

The trial involves multiple sites in the 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

Turkey

United States

France

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

The end of study is defined as the date the last patient either completes the last study visit, withdraws from the study, or is lost to follow-up (ie, the study patient can no longer be contacted by the investigator).

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

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

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)

none

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

2022-04-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-12-07

P. End of Trial
P.	End of Trial Status	Completed

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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