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    The EU Clinical Trials Register currently displays   35475   clinical trials with a EudraCT protocol, of which   5824   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2011-000362-35
    Sponsor's Protocol Code Number:AMSC-ALS-001
    National Competent Authority:Czech Republic - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-04-05
    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
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedCzech Republic - SUKL
    A.2EudraCT number2011-000362-35
    A.3Full title of the trial
    A Prospective, Non-randomized, Open Label Study to Assess the Safety and the Efficacy of Autologous Multipotent Mesenchymal Stem Cells in the Treatment of Amyotrophic Lateral Sclerosis.
    Bezpečnost a účinnost autologních mesenchymálních buněk kostní dřeně v léčbě amyotrofické laterální sklerózy. Prospektivní, nerandomizovaná, otevřená studie.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of Stem Cell Therapy of Motor Neuron Disease.
    Bezpečnost a účinnost buněčné terapie nemoci motorického neuronu (ALS).
    A.3.2Name or abbreviated title of the trial where available
    AMSC in ALS
    A.4.1Sponsor's protocol code numberAMSC-ALS-001
    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 SponsorBioinova, s.r.o.
    B.1.3.4CountryCzech Republic
    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 supportBioinova, s.r.o.
    B.4.2CountryCzech Republic
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioinova, s.r.o.
    B.5.2Functional name of contact pointHeadquarters
    B.5.3 Address:
    B.5.3.1Street AddressVídeňská 1083
    B.5.3.2Town/ cityPraha 4 - Krč
    B.5.3.3Post code142 20
    B.5.3.4CountryCzech Republic
    B.5.4Telephone number+420241 063 350
    B.5.6E-mailbazant@biomed.cas.cz
    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 nameSuspension of human autologous MSC 3P in 1,5 ml
    D.3.2Product code AMSC
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman autologous mesenchymal stem cells
    D.3.9.2Current sponsor codeAMSC
    D.3.10 Strength
    D.3.10.1Concentration unit million organisms/ml million organisms/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number7 to 13
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee 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
    Amyotrophic lateral sclerosis
    E.1.1.1Medical condition in easily understood language
    Motor Neuron disease (Lou Gehrig´s disease)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Safety: To assess an absence of complications at the site of intrathecal infusion and no new neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributed to the natural progression of the disease.
    E.2.2Secondary objectives of the trial
    Efficacy: To assess progress of the disease measured according to ALS functional rating scale (ALSFRS) and Norris scale and spirometry (FVC) after the AMSC treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To participate in this trial, patients will have to meet following criteria:
    1. established diagnosis of definite ALS according to El Escorial kriteria and data available from detailed neurologic observation (including ALSFRS, Norris scale, spirometry, brain and spinal cord MRI) for at least 6 months prior to the study entrance,
    2. riluzole naive or at stable dose for at least 2 months,
    3. patients between 18–65 years, both sexes, life expectancy more than 2 years,
    4. patients able to provide written informed consent.

    E.4Principal exclusion criteria
    Patients meeting any of the following criteria will be excluded from the participation in this study:
    1. FVC less than 70% ,
    2. in case of primary bulbar paralysis less than 15 points on Norris bulbar scale,
    3. less than 15 points on Norris spinal scale,
    4. pregnancy, breastfeeding
    5. coagulopathy,
    6. skin infection at the site of bone marrow aspiration or application of the cell product,
    7. gastrostomy,
    8. any significant medical condition that would compromise the safety of the patient (e.g. recent myocardial infarction, congestive heart failure, renal failure, liver failure, cancer, systemic infection, reccurent thromboembolic disease .....),
    9. alcohol or drug abuse,
    10. cancer,
    11. women of childbearing potential not using effective contraception (established oral contraception, intrauterine device, ligation of the uterine tube) including proven contraceptive measures taken by their sexual partners,
    12. fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube).
    E.5 End points
    E.5.1Primary end point(s)
    1) Complication at the site of intrathecal infusion
    2) New neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributable to the natural progression of the disease.
    E.5.1.1Timepoint(s) of evaluation of this end point
    ad 1: at Visits IV, V, and VI (1 day to 3 months after treatment administration)
    ad 2: at Visits IV, V, VI and IX (1 day to 12 months after treatment administration)
    E.5.2Secondary end point(s)
    ALS disease progression (assessment of functional deterioration milestones)
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 3, 6, 9, 12 and 18 months following treatment administration.
    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 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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 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 No
    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
    The end of the trial is defined by 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 years4
    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 years0
    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 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.1.2.1Number of subjects for this age range: 30
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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)
    Not different from the expected normal treatment of the studied condition (ALS disease)
    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 Decision2012-03-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-08-18
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