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Summary
EudraCT Number:2004-004158-23
Sponsor's Protocol Code Number:3303
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:2007-08-09
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 number2004-004158-23
A.3Full title of the trial
DOUBLE-BLIND PLACEBO-CONTROLLED TRIAL ON THE USE OF ACETYL-L-CARNITINE FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
A.3.2Name or abbreviated title of the trial where available
ND
A.4.1Sponsor's protocol code number3303
A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberND
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 SponsorIST. DI RICERCHE FARMACOLOG. M. NEGRI
B.1.3.4CountryItaly
B.3.1 and B.3.2Status of the sponsorNon-Commercial
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 Yes
D.2.1.1.1Trade name ZIBREN*20BUST 500MG
D.2.1.1.2Name of the Marketing Authorisation holderSIGMA-TAU
D.2.1.2Country which granted the Marketing AuthorisationItaly
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.4Pharmaceutical form Oral powder
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 INNAcetylcarnitine
D.3.10 Strength
D.3.10.1Concentration unit mg milligram(s)
D.3.10.2Concentration typeequal
D.3.10.3Concentration number500
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 Information not present in EudraCT
D.3.11.8Extractive medicinal product Information not present in EudraCT
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 Yes
D.3.11.13.1Other medicinal product typeL-(acetyloxy)-trimethylamino- butyric acid
D.8 Information on Placebo
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Amyotrophic lateral sclerosis therapy
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 9.1
E.1.2Level LLT
E.1.2Classification code 10002026
E.1.2Term Amyotrophic lateral sclerosis
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
Assessment of the impact of LAC at the dose of 3g/day by oral route for 12 consecutive months on the disability of ALS, in a clinically significant and measurable way
E.2.2Secondary objectives of the trial
1)Assessment of the impact of the drug on mortality (all causes); 2)Evaluation of the tolerability and safety of the drug at the given treatment regimen.
E.2.3Trial contains a sub-study Information not present in EudraCT
E.3Principal inclusion criteria
Inclusion criteria To be accepted, a case must satisfy all of the following: a. Diagnosis of definite, probable (laboratory-supported) or probable ALS (Sect. 5); b. Age between 40 and 70 years; c. Disease duration 6 < months < 24 months; d. Mild to moderate disability, documented by satisfactory bulbar and spinal function (minimal score of 3 on ALS Functional Rating Scale [ALS-FRS-R] 34 for swallowing, cutting food and handling utensils, and walking), satisfactory respiratory function (forced vital capacity (FVC) > 80% of predicted); e. Documented progression of the disease in the last 3 months; f. Patients able to understand, to comply with the requirements of the entire study, and to give an informed written consent.
E.4Principal exclusion criteria
Exclusion criteria The presence of at least one of the following leads to exclusion of the patient: a. Familial ALS; b. Antecedent polio infection; c. Motor neuron disease other than ALS (progressive bulbar palsy; progressive muscular atrophy; primary lateral sclerosis); d. Involvement of other neurological systems (sensory, extra pyramidal, oculomotor, cerebellar, autonomic). e. Antecedent or current exposure to metals (lead, aluminium, mercury, manganese, magnesium, selenium); f. Diabetes, severe clinical conditions, like cardiovascular disorders, arterial hypertension, kidney and liver disorders, dysthyroidism; g. Neoplasms or other diseases reducing life expectancy; h. Severe mental deterioration; i. Poor compliance with previous treatments; l. Neuroradiologic findings documenting lesions which might be responsible of the clinical findings; m. Experimental treatments in the preceding 3 months; n. Women who are pregnant or breast-feeding and/or of childbearing potential for the duration of the study; o. Patient and/or caring physician unwilling to take and/or prescribe riluzole.
E.5 End points
E.5.1Primary end point(s)
The primary end-point is the proportion of cases becoming non-self supporting during the 12-month follow-up period.
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 No
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic Information not present in EudraCT
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others Information not present in EudraCT
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 No
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.8.5The trial involves multiple Member States No
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 Information not present in EudraCT
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
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years1
E.8.9.1In the Member State concerned months9
E.8.9.1In the Member State concerned days
E.8.9.2In all countries concerned by the trial years2
E.8.9.2In all countries concerned by the trial months0
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 No
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) No
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) Yes
F.1.3Elderly (>=65 years) Yes
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 Information not present in EudraCT
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
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 No
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state80
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 Decision2005-04-15
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2005-04-15
P. End of Trial
P.End of Trial StatusCompleted

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