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    The EU Clinical Trials Register currently displays   42869   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2014-002053-19
    Sponsor's Protocol Code Number:CLMI070X2201
    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:2014-10-27
    Trial results
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2014-002053-19
    A.3Full title of the trial
    An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial of LMI070 given my mouth to Type I SMA infant patients.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberCLMI070X2201
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNOVARTIS FARMA S.p.A.
    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 supportNovartis Pharma services AG
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNOVARTIS FARMA S.p.A.
    B.5.2Functional name of contact pointDrug Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressLargo Umberto Boccioni, 1
    B.5.3.2Town/ cityORIGGIO
    B.5.3.3Post code21040
    B.5.4Telephone number+3902965412287
    B.5.5Fax number+39029659066
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameNA
    D.3.2Product code LMI070
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeLMI070
    D.3.9.3Other descriptive nameNA
    D.3.9.4EV Substance CodeSUB166489
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3.5
    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) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    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 No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Spinal Muscular Atrophy
    E.1.1.1Medical condition in easily understood language
    motor neuron disease
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 1
    To determine the safety and tolerability of ascending weekly doses and estimate the MTD of oral/enteral LMI070 in infants with type 1 SMA.
    Part 2
    To evaluate the safety and tolerability of multiple dose weekly regimens of oral/enteral LMI070 for 12 weeks in patients with type 1 SMA.
    E.2.2Secondary objectives of the trial
    Part 1
    -To evaluate LMI070 pharmacokinetics in serum after single and repeated doses of LMI070.
    -To evaluate the effect of LMI070 on muscle by ultrasound.
    -To evaluate the effect of LMI070 on growth parameters.
    -To evaluate the effect of LMI070 on respiratory function
    -To evaluate the effect of LMI070 on infant motor development.

    In addition to the above for Part 2:
    -To evaluate the efficacy of LMI070 on motor and developmental milestones
    -To evaluate the efficacy of LMI070 on ulnar nerve compound action potential [CMAP] (optional)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Written informed consent must be obtained from the parent / guardian before any assessment is performed.
    •Type 1 SMA, diagnosed clinically (symptom onset <6 months of age) and confirmed genetically (homozygous SMN1 gene deletion or mutation) plus SMN2 copy number of 2.
    •Best supportive care in place and stable for at least 14 days before screening.
    •Age at screening between 1 and 7 months, plus or minus 2 weeks.
    •Must be able to demonstrate antigravity strength in both biceps.
    •Must have or agree to have placement of feeding tube for enteral access via nasogastric (NG), nasojejunal (NJ), percutaneous gastrostomy (PEG), or percutaneous jejunostomy (PEJ) tube.
    E.4Principal exclusion criteria
    •Use of other investigational drugs within 14 days.
    •Neurologic, neuromuscular or genetic disorders other than SMA
    •Anemia (Hgb < 8 g/dL), leukopenia (ANC < 2000/ml), or thrombocytopenia (< 100,000/mL).
    •Hepatic dysfunction (AST or ALT > 1.5 x ULN; total bilirubin > ULN).
    •Renal dysfunction (eGFR < 80 ml/min).
    •Clinically significant abnormalities in hematology or clinical chemistry parameters, as assessed by the Site Investigator, at screening that would render the subject unsuitable for inclusion
    •Hypoxemia (O2 saturation awake <92% or O2 saturation asleep <91%,without ventilation support) or requiring oral suctioning >2 per day
    •Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period.
    •Excluding SMA, any medically unstable condition including cardiomyopathy, hepatic dysfunction, kidney disorder, endocrine disorder, GI disorders, prematurity of <32 weeks gestation, metabolic disorders, severe respiratory compromise and significant brain abnormalities or injuries including hypoxic-ischemic encephalopathy.
    •Ongoing medical condition that according to the Site Investigator would interfere with the conduct and assessments of the study. Examples are medical disability other than SMA that would interfere withthe assessment of safety or would compromise the ability of the subject to undergo study procedures including be assessed by CHOP-INTEND motor scale.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint for Part 1:
    Physical Exam : Screening, D1 – D85 + EOS
    Vital signs: Screening, D1 – D85 + EOS
    ECG : Screening, D1, D8, D85 + EOS
    Safety : Screening, Baseline, D3, D8, D15, D29, D43, D57, D71, D85 + EOS
    Adverse Events: All Visits

    Primary endpoint Part 2
    Physical Exam – Screening, Baseline, D1 – D88 + EOS
    Vital signs - Screening, Baseline, D1 – D88 + EOS
    ECG - Screening, D1, D8, D88 + EOS
    Safety – Screening, Baseline, D8, d15, D2, D29, D36, D43, D50, D57, D64, D71, D88 + EOS
    Adverse Events – All Visits
    E.5.1.1Timepoint(s) of evaluation of this end point
    See Above
    E.5.2Secondary end point(s)
    Secondary Endpoint Part 1
    PK - D1 (Pre-dose + 1, 2, 4, 8 & 24 hours post-dose), D2, D3, D5, D8, D29, D43
    Ultrasound : Baseline, D85 + EOS
    Growth measurements : Screening, D1, D8-D85 + EOS
    Respiratory function: Baseline, D8-D85 +EOS
    CHOP-INTEND : Baseline, D36, D85 + EOS

    Secondary Endpoint Part 2
    PK : D1 (Pre-dose + 1, 2, 4, 8 & 24 hours post-dose), D2, D3, D5, D8, D29, D43, D57 (Pre-dose + 2, 4, 8 & 24 hours post-dose), D59, D62
    Ultrasound : Baseline, D88 + EOS
    Growth measurements : Screening, D1, D8-D57, D64, D71, D88 + EOS
    Respiratory function - Baseline, D8-D57, D64, D71, D88 + EOS
    CHOP-INTEND – Baseline, D15, D36, D57, D88 + EOS
    CMAP – Baseline, D88 + EOS
    E.5.2.1Timepoint(s) of evaluation of this end point
    See above
    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 Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. 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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other No
    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 No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    European Union
    United States
    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
    When the last subject completes their End of Study visit, and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator, or in the event of an early study termination decision, the date of that decision.
    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 months0
    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 months0
    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.1.1Number of subjects for this age range: 22
    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) Yes
    F. of subjects for this age range: 22
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    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. of subjects incapable of giving consent
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 22
    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)
    Patients will initially be dosed for 13 weeks, however dosing may be extended if recommended by the independent DMC.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-01-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-04-01
    P. End of Trial
    P.End of Trial StatusCompleted
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