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    Summary
    EudraCT Number:2024-000202-16
    Sponsor's Protocol Code Number:MLB-01-003
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2024-05-31
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2024-000202-16
    A.3Full title of the trial
    AN OPEN LABEL PHASE 2 STUDY OF BBP 418 IN PATIENTS WITH LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2I (MLB 01 003)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    AN OPEN LABEL PHASE 2 STUDY OF BBP 418 IN PATIENTS WITH LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2I (MLB 01 003)
    A.4.1Sponsor's protocol code numberMLB-01-003
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04800874
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/387/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorML Bio Solutions
    B.1.3.4CountryUnited States
    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 supportML Bio Solutions
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationML Bio Solutions
    B.5.2Functional name of contact pointDoug Sproule
    B.5.3 Address:
    B.5.3.1Street Address1800 Owens Street Suite C-1200
    B.5.3.2Town/ citySan Francisco
    B.5.3.3Post code94158
    B.5.3.4CountryUnited States
    B.5.6E-mailclinops@mlbiosolutions.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/3/20/2337
    D.3 Description of the IMP
    D.3.1Product nameBBP-418
    D.3.4Pharmaceutical form Oral powder
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    Limb Girdle Muscular Dystrophy 2I/R9
    E.1.1.1Medical condition in easily understood language
    Limb Girdle Muscular Dystrophy 2I/R9
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    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
    • Explore the safety and tolerability of ascending dose levels of BBP-418 in patients with Limb Girdle Muscular Dystrophy (LGMD)2I.
    E.2.2Secondary objectives of the trial
    • Explore the dose response of BBP-418 with regard to the glycosylation of αDG.
    • Explore the feasibility and usefulness of selected clinical efficacy and pharmacodynamic (PD) assessments in patients with LGMD2I receiving ascending doses of BBP-418.
    • Explore the pharmacokinetics (PK) of BBP-418 in patients with LGMD2I receiving ascending doses of BBP-418.

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be a male or female patient, ages 12 to 55 years of age (inclusive)
    2. Have a body weight >30 kg
    3. Have a genetically confirmed diagnosis of LGMD2I and be clinically affected (defined as demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
    4. Able to complete the 10MWT in ≤12 seconds unaided (“moderate disease”) or are with “severe disease”/non-ambulatory as defined by being unable to complete the 10MWT unaided in >12 seconds.
    E.4Principal exclusion criteria
    1. Evidence of clinically significant concomitant disease, including:
    a. Any history of a gastrointestinal condition, including surgeries, which may affect absorption after oral administration
    b. Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I
    c. Any condition other than LGMD2I requiring therapy with prescription medicine (medication for common and mild concomitant conditions may be permitted after consultation with the PI)
    d. Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the Investigator’s opinion, is likely to unfavourably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
    E.5.1.1Timepoint(s) of evaluation of this end point
    15 months
    E.5.2Secondary end point(s)
    • Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax)
    • Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG)
    • Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior
    • Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC)
    E.5.2.1Timepoint(s) of evaluation of this end point
    15 months
    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 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) 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 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.8.2.4Number of treatment arms in the trial1
    E.8.3 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee No
    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
    Enrollment completed. Study will remain ongoing until an open label extension study is initiated, the product receives regulatory approval or the program is discontinued
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years4
    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: 6
    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: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 14
    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)
    Enrollment into an open label
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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