Clinical Trials Register

Summary
EudraCT Number:	2010-023744-33
Sponsor's Protocol Code Number:	FOR-DMD
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2012-12-28
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2010-023744-33

A.3

Full title of the trial

Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen.

Distrofia muscolare di Duchenne: studio in doppio cieco randomizzato per trovare il regime ottimale di steroidi.

A.3.1

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

Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen.

Distrofia muscolare di Duchenne: studio per trovare il regime ottimale di terapia con gli steroidi (cortisone)

A.3.2

Name or abbreviated title of the trial where available

FOR-DMD

A.4.1

Sponsor's protocol code number

FOR-DMD

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

ISRCTN46102316

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01603407

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	AZIENDA OSPEDALIERA DI PADOVA
B.1.3.4	Country	Italy
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	University of Rochester
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	Azienda Ospedaliera di Padova
B.5.2	Functional name of contact point	Clinica Neurologica I
B.5.3	Address:
B.5.3.1	Street Address	Via Giustiniani, 1
B.5.3.2	Town/ city	Padova
B.5.3.3	Post code	35128
B.5.3.4	Country	Italy
B.5.4	Telephone number	049-8212622; 049-8211943
B.5.5	Fax number	049-8211770
B.5.6	E-mail	elena.pegoraro@unipd.it

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	DELTACORTENE*20CPR 5MG
D.2.1.1.2	Name of the Marketing Authorisation holder	BRUNO FARMACEUTICI SpA
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.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.4	EV Substance Code	SUB10020MIG
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	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	Yes
D.3.11.13.1	Other medicinal product type	di natura chimica
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	DELTACORTENE*20CPR 5MG
D.2.1.1.2	Name of the Marketing Authorisation holder	BRUNO FARMACEUTICI SpA
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.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.4	EV Substance Code	SUB10020MIG
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	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	Yes
D.3.11.13.1	Other medicinal product type	di natura chimica
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	DEFLAN*10CPR 6MG
D.2.1.1.2	Name of the Marketing Authorisation holder	LAB.GUIDOTTI SpA
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.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	DEFLAZACORT
D.3.9.1	CAS number	14484-47-0
D.3.9.4	EV Substance Code	SUB06943MIG
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	6
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	Yes
D.3.11.13.1	Other medicinal product type	di natura chimica

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

Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids.

Pazienti di sesso maschile affetti da Distrofia muscolare di Duchenne, di età compresa fra 4 e 8 anni, in grado di alzarsi da terra autonomamente, non precedentemente trattati con steroidi.

E.1.1.1

Medical condition in easily understood language

Duchenne muscular Dystrophy children between 4 and 8 years of age, able to get up from the floor without help, not previously treated with steroids.

Bambini affetti da Distrofia muscolare di Duchenne, età compresa fra 4 e 8 anni, in grado di alzarsi da terra da soli, che non hanno fatto uso in passato di terapia steroidea(cortisone)

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	14.1
E.1.2	Level	PT
E.1.2	Classification code	10013801
E.1.2	Term	Duchenne muscular dystrophy
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 compare 3 commonly employed corticosteroid regimens in terms of efficacy as valuated with functional measures and patient/parent satisfaction.

Confrontare 3 regimi di corticosteroidi usati relativamente all'efficacia valutata attraverso la misura di valutazioni funzionali e la soddisfazione dei soggetti/genitori riguardo al trattamento.

E.2.2

Secondary objectives of the trial

1. tolerabulity; 2. advese events and safety; 3. secondary functional outcomes; 4. quality of life; 5. cardiac function.

1- tollerabilità; 2. eventi avversi e sicurezza; 3. outcome secondari funzionali; 4. qualità di vita; 5. funzione cardiaca

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Subjects with a confirmed diagnosis of DMD (defined as evidence of clinical signs of DMD and a confirmed mutation in the dystrophy gene (deletion, duplication or point mutation), age of at least 4 years and less than 8 years. ability to risde from the floor independently, ability and willingness of parents or legal tutor to give consent and to respect the scheduled appointments, ability to obtain consistent measures of FVC.

Soggetti con diagnosi confermata di DMD(evidenza di segni clinici compatibili con DMD e mutazione confermata nel gene della distrofia (delezione o duplicazione o mutazione puntiforme); età compresa fra 4 e 8 anni, capacità di alzarsi da solo da terra, volontà e capacità del genitore o tutore legale di dare il consenso informato; volontà e capacità di rispettare le visite programmate, nonchè la capacità di mantenere misure riproducibili di FVC.

E.4

Principal exclusion criteria

Significant renal or hepatic insufficiency; immunosuppression or other controindications to corticosteroid therapy; history of chronic systemic fungal or viral infections; diabetes mellitus; idiopatic hypercalciuria; luck of immunity to Varicella-Zoster virus and refusal of the vaccination; evidence of symptomatic cardiomyopathy; allergy/sensitivity to the study drugs or their formulations, current or previous treatment with corticosteroids or other immunosuppressive treatments for DMD or other signs of persistent inhability to swallow the tablets, severe behavioral problems, including autism, previous and current medical condition, medical history, physical findings or laboratory abnormalities that may compromise the safety or affect the evaluation of the results of the study; weight below 13 Kg; exposure to any investigational drug, currently or in the 3 months prior to starting thw study treatment.

Rilevante insufficientza renale o epatica in anamnesi; immunosoppressione o altre controindicazioni alla terapia corticosteroidea; storia di infezioni croniche sistemiche fungine o virali; diabete mellito; ipercalciuria idiopatica; mancanza di immunità alla varicella e rifiuto di sottoporsi alla vaccinazione; evidenza di cardiomiopatia sintomatica; allergia/sensibilità ai farmaci dello studio o alle loro formulazioni; trattamento in corso o precedente con corticosteroidi o altri trattamenti immunosoppressori per la DMD o di altro tipo di indicazioni ricorrenti; incapacità di assumere le compresse; gravi problemi comportamentali, tra cui l'autismo; condizioni mediche precedenti o in corso, anamnesi, riscontri fisici o anomalie di laboratorio che potrebbero compromettere la sicurezza e compromettere la valutazione dei risultati dello studio; peso < 13 Kg; esposizione a qualsiasi farmaco sperimentale attualmente o nei 3 mesi prima di iniziare il trattamento in studio.

E.5 End points

E.5.1

Primary end point(s)

Efficacy-Functional measures: time to rise from the floor, vital capacity (FVC), parent/child satisfaction about treatment by TSQM Questionnaire.

Efficacia-Valutazioni funzionali: tempo nell'alzarsi da terra, capacità vitale forzata (FVC), soddisfazione dei soggetti/genitori riguardo al trattamento mediante Questionario TSQM

E.5.1.1

Timepoint(s) of evaluation of this end point

At 0, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months.

ai mesi 0, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60.

E.5.2

Secondary end point(s)

1. tolerability; 2. adverse effects and safety; cardiac function, behavioral issues, bone fractures, cataracts, cushingoid appearance, gastro-intestinal symptoms, glycosuria, hypertansion, immunosuppression, growth reduction (in height), skin alterations, excessive weight gain. 3. secondary functional outcomes: time to run or walk 10 m, 6 min walk test, North Star Ambulatory Assessment score, lean mass (evaluation with DXA), disease ''milestones'' (loss of ambulation time, loss of ability to get up from supine or from a chair, ecc) 4. Quality of life: Peds-QoL genitore/bambino.

1. tollerabilità; 2. Eventi avversi e sicurezza: cambiamenti nel comportamento, fratture ossee, cataratte, aspetto Cushingoide, sintomi gastro-intestinali, glicosuria, ipertensione, immunosoppressione, rallentamento della crescita (in altezza) , alterazioni a carico della pelle, aumento di peso eccessivo. 3. Outcome funzionali secondari: cammino temporizzato a 10 metri, cammino temporizzato a 6 minuti, punteggio totale dlela Scala North Star Ambulatory Assessment, valutazione della massa magra tramite scansione DXA e outcome per le ''pietre miliari'' della storia di malattia (quali il tempo per la perdita della deambiulazione, della capacità di alzarsi da sdraiato, di alzarsi dalla sedia, ecc. 4. Qualità della vita: Questionario Peds-Qol genitore e bambino.

E.5.2.1

Timepoint(s) of evaluation of this end point

At 0, 3, 6, 12, 18, 24, 30, 36, 42, 48. 54, 60 months.

ai mesi 0, 3, 6, 12, 18, 24, 30, 36, 42, 48. 54, 60

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

Yes

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

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

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)

Yes

E.8.2.2

Placebo

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

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

United States

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

30 days after last patients, last visit.

30 giorni dopo l'ultima visita dell'ultimo paziente

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

F.1.1

Number of subjects for this age range:

300

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:

300

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

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

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

Children of age between 4 and 8 years.

Minori di età compresa fra 4 e 8 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

173

F.4.2.2

In the whole clinical trial

300

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)

According to the standard care for DMD.

I pazienti saranno trattati secondo la terapia standard prevista per la DMD.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	NIHR Medicines for Children Research Network

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2013-02-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-11-12

P. End of Trial
P.	End of Trial Status	Ongoing

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