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    Summary
    EudraCT Number:2019-000601-77
    Sponsor's Protocol Code Number:5051-201
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2019-06-26
    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 ConcernedIreland - HPRA
    A.2EudraCT number2019-000601-77
    A.3Full title of the trial
    A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with
    Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping
    Treatment
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients

    A.4.1Sponsor's protocol code number5051-201
    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 SponsorSarepta Therapeutics, Inc.
    B.1.3.4CountryUnited States
    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 supportSarepta Therapeutics, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation Syneos Health
    B.5.2Functional name of contact pointproject management
    B.5.3 Address:
    B.5.3.1Street Address4 The Fleming Building
    B.5.3.2Town/ city Edinburgh Technopole
    B.5.3.3Post codeEH26 0BE
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44131 448 1303
    B.5.6E-mailcarolyn.mills@syneoshealth.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSRP-5051
    D.3.2Product code SRP-5051
    D.3.4Pharmaceutical form Lyophilisate 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 INNSRP-5051
    D.3.9.1CAS number 2101569-97-3
    D.3.9.2Current sponsor codeSRP-5051
    D.3.9.3Other descriptive namepeptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
    D.3.9.4EV Substance CodeSUB197949
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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.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 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
    Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy is a rare, fatal degenerative neuromuscular disease with an X-linked recessive inheritance caused by mutations in the dystrophin gene
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10052655
    E.1.2Term Duchenne muscular dystrophy gene carrier
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: Multiple-Ascending-Dose (MAD) for Dose Determination
    To evaluate the safety and tolerability of multiple ascending doses of SRP-5051 (4.0, 10.0, 16.0, and 20.0 mg/kg), administered intravenously (IV) every 4 weeks, and determine the maximum tolerated dose (MTD)

    Part B – Dose Expansion
    • To evaluate exon-skipping and dystrophin protein production levels (derived from muscle) after additional treatment with SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    E.2.2Secondary objectives of the trial
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    To determine the pharmacokinetics (PK) of each dose level of the aforementioned multiple ascending doses of SRP-5051, administered IV every 4 weeks
    Part B – Dose Expansion
    • To evaluate the ongoing safety and tolerability of additional treatment with SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    • To evaluate the clinical effect of additional treatment with SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    • To determine the PK of SRP-5051, administered IV every 4 weeks at the MTD determined in Part A, in patients who have completed Part A
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A patient must meet all of the following criteria to be eligible to participate in this study:
    1. Is male.
    2. Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
    3. Is 7 to 21 years of age, inclusive.
    4. Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.
    5. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a highly effective form of contraceptive during this timeframe.
    6. Is willing to provide informed consent or informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
    E.4Principal exclusion criteria
    A patient who meets any of the following criteria will be excluded from this study:
    1. Has a LVEF < 40.0% based on an ECHO performed within 12 weeks prior to Screening or at the Screening visit.
    2. Has a QT interval corrected with Fridericia’s formula ≥ 450 msec on the Screening ECG.
    3. Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
    4. Requires anti-arrhythmic and/or diuretic therapy for heart failure.
    5. Has a FVC < 40.0% of predicted value (according to the American Thoracic Society/European Respiratory Society criteria) within 12 weeks prior to Screening or at Screening.
    6. Has a current infection, or history of an infection within 12 weeks prior to Screening requiring treatment with an antibiotic.
    7. Has a known kidney disease or had an acute kidney injury within 24 weeks prior to Screening.
    8. Use of any herbal medication/supplement containing aristolochic acid.
    9. Treatment with any exon 51-skipping therapy within 24 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.
    10. Participation in an interventional clinical trial within 12 weeks prior to Screening.
    11. Use of any aminoglycoside antibiotic or statin within 12 weeks prior to Screening.
    12. Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
    13. Major surgery within 12 weeks prior to Screening, or planned surgery or procedures that would interfere with the conduct of the study.
    14. Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy.
    15. Any other condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the trial.
    16. Inability to comply with the study protocol.
    17. Is an employee of the Investigator or study center, with direct involvement in the proposed study or other studies under the direction of that Investigator or study center, as well as family members of the employees or the Investigator.
    18. Any patient who is taking medications that increase the risk of bleeding, in the Investigator's opinion (eg, anticoagulants, antiplatelet agents, novel oral anticoagulants, selective norepinephrine reuptake inhibitors, etc.)
    19. Platelet count < 150 × 103/μL.
    E.5 End points
    E.5.1Primary end point(s)
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    • Incidence of AEs
    • Clinically significant laboratory abnormalities (eg, hematology, chemistry, coagulation, urinalysis)

    Part B – Dose Expansion
    • Change from Baseline biopsy in exon-skipping levels (as measured by PCR) in muscle.
    • Change from Baseline biopsy in dystrophin protein production levels (as measured by Western blot and/or other relevant methods) in muscle.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination

    All AEs will be reported from the time of providing signed informed consent/assent through the last follow-up visit.
    Clinically significant laboratory abnormalities will be assessed from Baseline at week 12
    All the routine clinical laboratory tests will be performed during clinic visits at the time points specified in the Schedule of Events for Part A as stated in the Table 1 of the protocol

    Part B – Dose Expansion
    -biopsy in exon-skipping levels (as measured by PCR) in muscle collected from the Part B, Week 24 biopsy.
    -dystrophin protein production levels from the Part B, Week 24 biopsy.
    E.5.2Secondary end point(s)
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    • PK parameters

    Part B – Dose Expansion
    • Incidence of AEs
    • Clinically significant laboratory abnormalities (eg, hematology, chemistry, coagulation, urinalysis)
    • Change from Baseline assessment in the following, conducted at Part B, Week 24:
    o Forced vital capacity (FVC) (% predicted) calculation
    o North Star Ambulatory Assessment (NSAA) (in ambulatory patients)
    o Performance Upper Limb (PUL)
    o Brooke Upper Extremity Scale score (“Brooke Score”)
    • PK parameters at the MTD determined in Part A
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A – Multiple-Ascending-Dose (MAD) for Dose Determination
    • PK parameters at each dose level
    Part B – Dose Expansion
    •All AEs will be reported from Baseline through the last follow-up visit.
    •Clinically significant laboratory abnormalities will be conducted in part B Week 24
    • Change from baseline assessment in functional test conducted at part B, capture all timepoints
    • PK parameters at MTD
    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 Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Multiple-Ascending-Dose Study for Dose Determination, then Dose Expansion
    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 No
    E.8.1.2Open Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Multiple-Ascending-Dose Study for Dose Determination, then Dose Expansion
    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.8.2.4Number of treatment arms in the trial4
    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 EEA14
    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
    Belgium
    Canada
    Germany
    Ireland
    Italy
    Netherlands
    Spain
    United Kingdom
    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
    LPLV
    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 months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months1
    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: 24
    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.1.1.5.1Number of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 4
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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.3.3.6.1Details of subjects incapable of giving consent
    This study will be conducted in both ambulatory and nonambulatory patients, aged 7 to 21 years, inclusive, with DMD amenable to exon 51-skipping treatment.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 24
    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)
    none
    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 Decision2019-08-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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