Clinical Trials Register

Summary
EudraCT Number:	2014-003920-32
Sponsor's Protocol Code Number:	C13-37
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2019-11-12
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

France - ANSM

A.2

EudraCT number

2014-003920-32

A.3

Full title of the trial

Study phase II open label study using triheptanoin in patients with a deficiency of glmucose transporter type 1 (GLUT-1)

Etude de phase II en ouvert utilisant la triheptanoïne chez des patients souffrant d'un déficit en transporteur de glucose de type 1 (GLUT-1)

A.3.1

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

see beelow

Evaluer l’efficacité de l'huile de triheptanoïne chez des patients atteints d'un déficit du transporteur du glucose cérébral GLUT1 (GLUT1-DS)

A.3.2

Name or abbreviated title of the trial where available

GLUTHEP

A.4.1

Sponsor's protocol code number

C13-37

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	INSERM
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	ultragenyx Pharmaceutical 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	INSERM
B.5.2	Functional name of contact point	Sonia GUEGUEN
B.5.3	Address:
B.5.3.1	Street Address	8 rue de la Croix Jarry
B.5.3.2	Town/ city	Paris
B.5.3.3	Post code	75013
B.5.3.4	Country	France
B.5.4	Telephone number	33144236047
B.5.5	Fax number	33144236710
B.5.6	E-mail	rqrc.siege@inserm.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Triheptanoin
D.3.2	Product code	UX007
D.3.4	Pharmaceutical form	Oral solution
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	Not available
D.3.9.1	CAS number	620-67-7
D.3.9.2	Current sponsor code	UX007
D.3.9.3	Other descriptive name	TRIHEPTANOIN
D.3.9.4	EV Substance Code	SUB129584
D.3.10	Strength
D.3.10.1	Concentration unit	% percent
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
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

N/A

Déficit en transporteur de glucose de type 1

E.1.1.1

Medical condition in easily understood language

N/A

Glut1 assure le transport de glucose de la barrière hémato-encéphalique. Le déficit de ce transporteur a pour conséquence une affection neuro-métabolique rare

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	HLGT
E.1.2	Classification code	10039911
E.1.2	Term	Seizures (incl subtypes)
E.1.2	System Organ Class	10029205 - Nervous system 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

N/A

Evaluer l'efficacité de l'huile de triheptanoïne chez des patients atteints d'un déficit du transporteur du glucosecérébral GLUT1 (GLUT1-DS) avec ou sans régime cétogène, présentant des symptômes résiduels de leur maladie.

E.2.2

Secondary objectives of the trial

N/A

- confirmer la tolérance du régime à base de triheptanoïne chez des patients GLUT1DS
- évaluer les effets de la triheptanoïne sur la clinique globale, l'autonomie, la qualité de vie et les fonction motrices des patients;
- evaluer les effets de la triheptanoine sur le métabolisme énergétique du cerveau, uniquement chez des patients âgés de plus de 15 ans, en utilisant la spectroscopie 31P-RMN. lors de l'activation du cortex occipital par stimulation visuelle, cette technique permet de mesurer les variations des composés riches en phosphates tels que l'ATP ou la phosphocréatine.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

N/A

- homme ou femme, garçon ou fille
- age égal ou supérieur à 3 ans à la signature du consentement
- patient dont la maladie GLUT1-DS a été confirmée: mutation dans le gène SLC2A1
- patient présentant des manifestations paroxystiques lors des 3 premiers mois avant le début de l'étude
- patient sans régime alimentaire spécifique ou qui suit un régime cétogène ou assimilé (Atkins)
- patient ayant signé le consentement éclairé si patient majeur non protégé ou majeur sous curatelle
- signature du consentement éclairé par les titulaires de l'autorité parentale ou le représentant légal si patient mineur ou patient majeur sous tutelle
- couverture par un régime de sécurité sociale

E.4

Principal exclusion criteria

N/A

Age < 3 ans
- trouble psychiatrique majeur concomitant
- trouble neurologique concomitant significatif
- patient avec une affection intercurrent empêchant sa participation à l'étude, ex VIH, diabète
- Incapacité à recevoir une information éclairée sur le protocole
- Impossibilité de participer à la totalité de l'étude
- non couverture par un régime de sécurité sociale
- absence de signature de consentement ou refus du patient et/ou du titulaire de l'autorité parentale ou du représentant légal
- Femme enceinte parturiente, allaitante
- Personne privée de liberté par décision judiciaire ou administrative
- Personne hospitalisée sans consentement et ne faisant pas l'objet d'une mesure de protection légale
- Personne majeure hors d'état d'exprimer son consentement
- Personne sous sauvegarde de justice
- personne soumise à une période d'exclusion pour une autre recherche
- Refus d'être informé(e) d'une anomalie décelée à l'IRM

E.5 End points

E.5.1

Primary end point(s)

N/A

le critère d'évaluation principal reposera sur le nombre d'évènement paroxystiques, en particulier accès de mouvements anormaux, reportés sur les carnets patients.

E.5.1.1

Timepoint(s) of evaluation of this end point

N/A

semaine 69

E.5.2

Secondary end point(s)

N/A

- Pour les patients initialement sous régime cétogène, les évènements paroxystiques reportés lors de la phase 2 seront également étudiés. on analysera ainsi la variation des évènements paroxystiques entre les phases 1 (régime cétogène) et 2 (traitement par la triheptanoïne ajoutée au régime cétogène)
- pour les patients initialement sans régime, les évènements paroxystiques reportés lors de la phase 3 seront également étudiés. On analysera ainsi les variations des évènements paroxystiques entre les phases 2 (traitement par la triheptanoine) et 3 (absence de régime)

E.5.2.1

Timepoint(s) of evaluation of this end point

semaine 69

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

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

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

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

LPLV

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)

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)

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

N/A

sujets pédiatriques

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

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)

N/A

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

2013-11-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2013-10-30

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials