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    Summary
    EudraCT Number:2021-004276-33
    Sponsor's Protocol Code Number:ATA-001-FKRP
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-10-01
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2021-004276-33
    A.3Full title of the trial
    A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophy
    A.4.1Sponsor's protocol code numberATA-001-FKRP
    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 SponsorAtamyo Therapeutics
    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 supportAtamyo Therapeutics
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAtamyo Therapeutics
    B.5.2Functional name of contact pointSophie Olivier
    B.5.3 Address:
    B.5.3.1Street Address1bis, rue de l’Internationale
    B.5.3.2Town/ cityEvry-Courcouronnes
    B.5.3.3Post code91000
    B.5.3.4CountryFrance
    B.5.6E-mails.olivier@atamyo.com
    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/3/21/2566
    D.3 Description of the IMP
    D.3.1Product namerAAV9-hFKRPco_miR-208a
    D.3.2Product code GNT0006
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot yet available
    D.3.9.2Current sponsor codeGNT0006
    D.3.9.3Other descriptive nameAdeno-associated virus serotype 9 carrying the human Fukutin-Related protein and target sequence of the miR-208a
    D.3.9.4EV Substance CodeSUB235392
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number1.7E+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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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 placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    FKRP-related limb-girdle muscular dystrophy (LGMD R9)
    E.1.1.1Medical condition in easily understood language
    FKRP-related limb-girdle muscular dystrophy (LGMD R9)
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10028356
    E.1.2Term Muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Stage 1 : To assess the safety and tolerability of intravenous administration of GNT0006 in ambulant patients with LGMDR9 at two different dosage levels and to select the recommended dose for stage 2
    Stage 2 : To demonstrate efficacy of intravenous administration of GNT0006 in ambulant patients with LGMDR9 one year post IMP administration
    E.2.2Secondary objectives of the trial
    Stage 1 : To collect preliminary efficacy data
    Stage 2 : To assess the safety and tolerability of GNT0006 in ambulant patients with LGMDR9
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Ambulant male or female patients at least 16 years old
    2. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
    3. Able to:
    • Perform the 10-meter walk test (10MWT) within 30 sec with unilateral help, such as cane, or bilateral help, such as elbow crutches or orthotic devices below the knees
    • Rise from a standard-height chair with or without arm support
    4. Diaphragmatic muscle impairment defined as forced vital capacity (FVC) between 40 and 80% (inclusive) of the expected value
    5. Effective contraception
    • If sexually active male patients, commitment to use barrier contraception such as condom from the day of IMP administration and up to 12 month-visit after IMP administration. In addition, the female partner of childbearing potential is encouraged to use effective contraception up to the 12-month visit after IMP administration.
    • If female patients of childbearing potential, commitment to use highly effective methods of contraception from signature of informed consent to participate to this trial and up to 12 month-visit after IMP administration with negative blood pregnancy test at screening visit
    6. Signed written informed consent before any study related procedure is performed
    7. Patient medical status sufficiently stable and ability of patient and parents/legal guardian, in the opinion of the Investigator, to adhere to the study visit schedule and other protocol requirements.
    E.4Principal exclusion criteria
    1. Detectable serum neutralizing antibodies against AAV9
    2. Known hypersensitivity to IMP excipients, to eculizumab, murine
    proteins, or any excipients in eculizumab formulation
    3. Cardiomyopathy based on physical and cardiological examination and echocardiography with Left Ventricular Ejection Fraction (LVEF) below 50%
    4. Any respiratory assistance, including non-invasive daytime or nocturnal ventilation
    5. Inability to cooperate with muscle testing or to perform respiratory function tests
    6. Presence or history of concomitant muscular or other medical condition that might interfere with LGMDR9 evolution or that would confound scientific rigor or interpretation of results, e.g., current infectious episode (pulmonary, ENT,...), abnormal laboratory test if clinically significant
    7. Acute illness within 4 weeks of the anticipated IMP administration which may interfere with study assessments
    8. Recent immunosuppressive treatment within 3 months prior to
    screening
    9. Current or history of significant heart, lung, hepato-biliary or renal disease or impairment that jeopardize the safety of the subject according to the investigator
    10. Current participation in a clinical trial of another investigational medicinal product
    11. Previous participation in gene and cell therapy trials
    12. Any condition that would contraindicate treatment with immunosuppressant therapy
    13. Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
    14. Any vaccination 1 month prior to the planned IMP administration
    15. Serology consistent with HIV exposure or active hepatitis B or C infection
    16. Grade 2 or higher lab abnormalities for LFT, bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT, according to current version of CTCAE.
    E.5 End points
    E.5.1Primary end point(s)
    The change from baseline in FVC% in sitting position (expressed as percentage of the expected value based on age, gender, weight, and height-adjusted norms) at one year post-IMP administration in the treated group as compared to the placebo group (Stage 2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    One year post-IMP administration in Stage 2
    E.5.2Secondary end point(s)
    - Muscular functional tests
    - Imaging assessments
    - Respiratory assessments
    - Muscular Biopsy
    - Patient reported outcome and quality-of-life assessment
    - Biomarkers
    E.5.2.1Timepoint(s) of evaluation of this end point
    12-month
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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.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
    open-label dose escalation (Stage 1) then double-blind placebo controlled, randomized (Stage 2)
    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.8.2.4Number of treatment arms in the trial2
    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 EEA7
    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
    Canada
    United States
    France
    Sweden
    Germany
    Denmark
    Norway
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 years7
    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: 5
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 32
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 32
    F.4.2.2In the whole clinical trial 39
    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)
    After a patient has ceased his/her participation in the study, his/her medical doctor/ study investigator will offer the most appropriate treatment currently available at their discretion
    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 Decision2021-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-11
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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