Kliniska prövningar Nct sida

Summary
EudraCT Number:2010-020386-24
Sponsor's Protocol Code Number:TRO19622CLEQ1275-1
National Competent Authority:Italy - Italian Medicines Agency
Clinical Trial Type:EEA CTA
Trial Status:Completed
Date on which this record was first entered in the EudraCT database:2010-09-01
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
A. Protocol Information
A.1Member State ConcernedItaly - Italian Medicines Agency
A.2EudraCT number2010-020386-24
A.3Full title of the trial
Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients
A.4.1Sponsor's protocol code numberTRO19622CLEQ1275-1
A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorTROPHOS
B.1.3.4CountryFrance
B.3.1 and B.3.2Status of the sponsorCommercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing support
B.4.2Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisation
B.5.2Functional name of contact point
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleTest
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation No
D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
D.2.5.1Orphan drug designation numberEU/3/05/264;EMEA/OD/081/04
D.3 Description of the IMP
D.3.1Product nameolesoxime
D.3.2Product code TRO19622
D.3.4Pharmaceutical form Powder and solvent for oral suspension
D.3.4.1Specific paediatric formulation Information not present in EudraCT
D.3.7Routes of administration for this IMPOral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8INN - Proposed INNolesoxime
D.3.9.1CAS number 2203-87-0
D.3.9.2Current sponsor codeTRO19622
D.3.10 Strength
D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
D.3.10.2Concentration typeequal
D.3.10.3Concentration number10
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin Yes
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
D.3.11.3.1Somatic cell therapy medicinal product No
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product Information not present in EudraCT
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another type of medicinal product No
D.8 Information on Placebo
D.8 Placebo: 1
D.8.1Is a Placebo used in this Trial?Yes
D.8.3Pharmaceutical form of the placeboPowder and solvent for oral suspension
D.8.4Route of administration of the placeboOral use
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 years
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 9.1
E.1.2Level LLT
E.1.2Classification code 10032950
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
To assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 in non ambulant patients aged 3-25 years
E.2.2Secondary objectives of the trial
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
1.Weakness and hypotonia consistent with a clinical diagnosis of spinal muscular atrophy (SMA)type II or III 2.Laboratory documentation of homozygous absence of SMNI exon 7 and/or deletion and mutation on other allele 3.MFM relative score (percentage of the maximum sum of both dimensions)≥ 15% (D1 + D2) 4.HFMS score at baseline ≥ 3 5.Non ambulant patients defined as patients with HFMS score ≤ 38 6.Must be 3 years of age or older, but younger then 26 years of age,at time of enrolment 7.Age of onset of symptoms ≤ 3 years of age 8.Signed informed consent of patient and/or parent/guardian 9.Laboratory results drawn within 31 days prior to start of study entry demonstrating no clinically significant abnormalities 10.Ability to take study treatment (tested at screening after informed consent)
E.4Principal exclusion criteria
1.Evidence of renal dysfunction,blood dysplasia,hepatic insufficiency,symptomatic pancreatitis,congenital heart defect,known hystory of metabolic acidosis,hypertension,significant central nervous system impairment,or neurodegenerative or neuromuscular disease other then SMA 2.Any clinically significant ECG abnormality 3.Any acute co-morbid condition interfering with the well-being of the subject within 7 days of enrolment including bacterial infection,viral infectious processes,food poisoning,temperature > 37.0�C,the need for acute treatment or observation due to any other reason,as judged by investigator,patient can be included after resolution of the acute event 4.Use of medications intended for the treatment of SMA including riluzole,valproic acid,hydroxyurea,sodium phenylbutyrate,butyrate derivatives,creatine,carnitine,growth hormone,anabolic steroids,probenecid,oral or parenteral use of corticosteroids at entry,agents anticipated to increase or decrease muscle strength or agents with known or presumed histone deacetylase(HDAC)inhibition,within 30 days prior to study entry.Subjects who use a nebulizer or require an inhaler to steroids will be allowed in the study;however oral use of steroids is prohibited.The oral use of salbutamol is permitted with the following restrictions:patients should have been on salbutamol for at least 6 months before inclusion in the trial,with good tolerance.The dose of salbutamol should remain constant for the duration of the trial.The use of inhaled β-agonists (for the treatment of asthma crisis per example)is allowed 5.Spinal rod or fixation for scoliosis within the past 6 months or anticipated need of it within 6 months of enrolment 6.Inability to meet study visit requirements or cooperate reliably with functional testing 7.Coexisting medical conditions that contraindicate travel,testing or study medication 8.Olesoxime is contraindicated in subjects who develop drug hypersensitivity to it or one of the formulaton excipients including hypersensitivity to sesame oil 9.Patients with hemostasis disorders 10.patients with known biliary tract obstruction 11.Current or planned pregnancy or nursing period 12.For woman:failure to use one of the following safe methods of contraception-female condoms,diaphragm or coil,each used in combination with spermicides;intra-uterine device;hormonal contraception in combination with a mechanical method of contraception 13.participation in any other investigational drug or therapy study within the previous 3 months.
E.5 End points
E.5.1Primary end point(s)
The primary outcome measure is the Motor Function Measure (MFM)D1+D2 score
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis No
E.6.3Therapy No
E.6.4Safety Yes
E.6.5Efficacy Yes
E.6.6Pharmacokinetic Yes
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) No
E.7.1.1First administration to humans No
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) Yes
E.7.3Therapeutic confirmatory (Phase III) No
E.7.4Therapeutic use (Phase IV) No
E.8 Design of the trial
E.8.1Controlled Yes
E.8.1.1Randomised Yes
E.8.1.2Open No
E.8.1.3Single blind No
E.8.1.4Double blind Yes
E.8.1.5Parallel group Yes
E.8.1.6Cross over No
E.8.1.7Other Yes
E.8.1.7.1Other trial design description
adattivo
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo Yes
E.8.2.3Other No
E.8.3 The trial involves single site in the Member State concerned No
E.8.4 The trial involves multiple sites in the Member State concerned Yes
E.8.4.1Number of sites anticipated in Member State concerned4
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA22
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA No
E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
E.8.7Trial 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
ultima visita dell`ultimo paziente
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years2
E.8.9.1In the Member State concerned months6
E.8.9.1In the Member State concerned days0
E.8.9.2In all countries concerned by the trial years2
E.8.9.2In all countries concerned by the trial months6
E.8.9.2In all countries concerned by the trial days0
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 Yes
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) Yes
F.1.1.6Adolescents (12-17 years) Yes
F.1.2Adults (18-64 years) Yes
F.1.3Elderly (>=65 years) No
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients Yes
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Yes
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally Yes
F.3.3.6.1Details of subjects incapable of giving consent
bambini e adolescenti
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state40
F.4.2 For a multinational trial
F.4.2.1In the EEA 150
F.4.2.2In the whole clinical trial 150
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)
Alla fine dello studio i pazienti avranno la possibilita di continuare la terapia con olesoxime open-label. Protocollo separato sara preparato per estensione open-label dopo la inclusione dell`ultimo paziente.
G. Investigator Networks to be involved in the Trial
N. Review by the Competent Authority or Ethics Committee in the country concerned
N.Competent Authority Decision Authorised
N.Date of Competent Authority Decision2010-09-14
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2010-07-28
P. End of Trial
P.End of Trial StatusCompleted

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