Clinical Trials Register

Summary
EudraCT Number:	2020-006161-11
Sponsor's Protocol Code Number:	RC31/20/0421
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:	2021-04-21
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

France - ANSM

A.2

EudraCT number

2020-006161-11

A.3

Full title of the trial

LONG -TERM INTERVENTIONAL FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL AND COMPARISON WITH AN UNTREATED COHORT OF CHILDREN WITH PRADER-WILLI SYNDROME

Étude interventionnelle de suivi à long terme jusqu’à l’âge de 4 ans des enfants avec un syndrome de Prader-Willi inclus dans l’étude clinique OTBB3 et comparaison avec une cohorte non traitée d’enfants avec un syndrome de Prader-Willi

A.3.1

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

FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL

A.3.2

Name or abbreviated title of the trial where available

OTBB3 Follow-Up

A.4.1

Sponsor's protocol code number

RC31/20/0421

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	Toulouse University Hospital
B.1.3.4	Country	France
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	OT4B
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Centre de référence Prader-Willi
B.5.2	Functional name of contact point	TAUBER
B.5.3	Address:
B.5.3.1	Street Address	330 avenue de Grande-Bretagne
B.5.3.2	Town/ city	Toulouse
B.5.3.3	Post code	31059
B.5.3.4	Country	France
B.5.4	Telephone number	+330534558551
B.5.5	Fax number	+330534558558
B.5.6	E-mail	tauber.mt@chu-toulouse.fr

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	Yes
D.2.5.1	Orphan drug designation number	EU/3/141302
D.3 Description of the IMP
D.3.1	Product name	oxytocin
D.3.4	Pharmaceutical form	Nasal spray, solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Nasal use
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Nasal spray
D.8.4	Route of administration of the placebo	Nasal use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Prader-willi syndrome

Syndrome de Prader-Willi

E.1.1.1

Medical condition in easily understood language

Prader-Willi Syndrome

Syndrome de Prader-Willi

E.1.1.2

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

MedDRA Classification

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 confirm the long term safety profile including the associated comorbidities in all children with PWS who have been treated in the OTBB3 study.

confirmer le profil de sécurité d’emploi à long terme, notamment les principales comorbidités associées, chez tous les enfants présentant un SPW ayant été traités au cours de l’étude OTBB3.

E.2.2

Secondary objectives of the trial

• To complete the safety assessment by describing the development of children and severity of the disease in all children included in the OTBB3 study;
• To compare the long-term safety and efficacy of early OT treatment in children included in OTBB3 with an untreated cohort of French children with PWS. This comparison will be performed on the subset of patients who have been treated in France in the OTBB3 study in a comparable context.

Compléter l’évaluation de la sécurité d’emploi en décrivant le développement des enfants et la sévérité de la maladie chez tous les enfants inclus dans l’étude OTBB3 ;
Comparer la sécurité d’emploi et l’efficacité à long terme d’un traitement précoce par l’ocytocine chez les enfants inclus dans l’étude OTBB3, avec une cohorte non traitée d’enfants français présentant un SPW. Cette comparaison sera réalisée sur le sous-ensemble de patients ayant été traités en France dans l’étude OTBB3 dans un contexte comparable.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Male or female child with a genetically confirmed diagnosis of PWS
The parents (or legal representative) must have signed the consent form;
Treated cohort: the child participated in the OTBB3 study
Untreated cohort: the child has never received OT, is aged 30±6 months at inclusion and is followed in France.

1. Enfant de sexe masculin ou féminin présentant un diagnostic génétiquement confirmé de SPW
2. Les parents (ou le représentant légal) doivent avoir signé le formulaire de consentement ;
3. Cohorte traitée : l’enfant a participé à l’étude OTBB3 ;
4. Cohorte non traitée : l’enfant n’a jamais reçu d’ocytocine, est âgé de 30±6 mois à l’inclusion et est suivi en France.

E.4

Principal exclusion criteria

1. Administrative problems:
a. Inability for the parents (or legal representative) to understand/fulfil study requirements;
b. No coverage by a social security regime;
2. Refusal of parents (or legal representative) to sign the consent form;

1. Problèmes administratifs :
a. Incapacité des parents (ou du représentant légal) à comprendre/respecter les exigences de l’étude ;
b. Absence de couverture par un régime de sécurité sociale ;
2. Refus des parents (ou du représentant légal) de signer le formulaire de consentement ;

E.5 End points

E.5.1

Primary end point(s)

• The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
• The occurrence of the following main comorbidities:
o Digestive disorders
o Scoliosis;
o Sleep disorders;
- Obstructive and central sleep apnoea
- Narcolepsy;
- Excessive daytime sleepiness
o Endocrine disorders
o Metabolic disorders
o Other comorbidities assessed by a checklist.
• The occurrence of medications, surgery and rehabilitations

• Nombre et pourcentage de patients présentant des événements indésirables (EI) et des événements indésirables graves (EIG) ;
• Survenue des principales comorbidités suivantes :
o Troubles digestifs
o Scoliose ;
o Troubles du sommeil ;
- Apnée obstructive et apnée centrale du sommeil
- Narcolepsie ;
- Somnolence diurne excessive
o Troubles endocriniens
o Troubles métaboliques
o Autres comorbidités évalués par une liste de contrôle.
• Prise de médicaments, chirurgie et rééducation

E.5.1.1

Timepoint(s) of evaluation of this end point

every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)

chaque année en V1, V2 et V3 entre 36 et 48 mois (42 +/- 6 mois)

E.5.2

Secondary end point(s)

• The number and percentage of patients with AEs and SAEs;
• The occurrence of the following main comorbidities:
o Digestive disorders
o Scoliosis;
o Sleep disorders
o Endocrine disorders
o Metabolic disorders
o Other comorbidities
• The occurrence of medications, surgery and rehabilitations
• The severity of the comorbidities
• Auxology, anthropometry and psychomotor development
• Severity of the disease in terms of:
o Miller nutritional phases;
o Eating behaviour
o Adaptive behaviour
o Behavioural and psychiatric disorders
• Caregiver burden

• Nombre et pourcentage de patients présentant des EI et des EIG ;
• Survenue des principales comorbidités suivantes :
o Troubles digestifs
o Scoliose ;
o Troubles du sommeil
o Troubles endocriniens
o Troubles métaboliques
o Autres comorbidités
• Prise de médicaments, chirurgie et rééducation
• Sévérité des comorbidités
• Auxologie, anthropométrie et développement psychomoteur
• Sévérité de la maladie en utilisant les critères suivants :
o Phases nutritionnelles de Miller ;
o Comportement alimentaire
o Comportement adaptatif
o Troubles comportementaux et psychiatriques
• Charge pesant sur les aidants

E.5.2.1

Timepoint(s) of evaluation of this end point

every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)

chaque année en V1, V2 et V3 entre 36 et 48 mois (42 +/- 6 mois)

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

Yes

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

Yes

E.8.2.3.1

Comparator description

children untreated by oxytocin

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

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

Dernière visite du dernier patient

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

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)

F.1.2

Adults (18-64 years)

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

F.3.3.1

Women of childbearing potential not using contraception