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    Summary
    EudraCT Number:2022-000956-12
    Sponsor's Protocol Code Number:277HV101
    National Competent Authority:Portugal - INFARMED
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2023-02-02
    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 ConcernedPortugal - INFARMED
    A.2EudraCT number2022-000956-12
    A.3Full title of the trial
    A Randomized, Blinded, Placebo-Controlled, Phase 1 Single
    Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115
    Estudo de Fase 1 de dose única ascendente, aleatorizado, em ocultação,
    controlado por placebo, em voluntários adultos saudáveis do sexo
    masculino e um estudo aberto de dose múltipla ascendente em
    participantes pediátricos com AME previamente tratados com
    onasemnogene abeparvovec (Zolgensma™) para avaliar a segurança,
    tolerabilidade e farmacocinética de BIIB115
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BIIB115 in Healthy Volunteers and Pediatric SMA Patients Previously Treated with Zolgensma
    A.4.1Sponsor's protocol code number277HV101
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05575011
    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 SponsorBiogen Idec Research Limited
    B.1.3.4CountryUnited Kingdom
    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 supportBiogen Idec Research Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiogen Idec Research Limited
    B.5.2Functional name of contact pointSponsor
    B.5.3 Address:
    B.5.3.1Street AddressInnovation House 70 Norden Road
    B.5.3.2Town/ cityMaidenhead, Berkshire
    B.5.3.3Post codeSL6 4AY
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailClinicalTrials@biogen.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 nameBIIB115
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNBIIB115
    D.3.9.2Current sponsor codeBIIB115
    D.3.9.3Other descriptive nameBIIB115
    D.3.9.4EV Substance CodeSUB274544
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Spinal muscular atrophy
    E.1.1.1Medical condition in easily understood language
    SMA
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10041582
    E.1.2Term Spinal muscular atrophy
    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
    To evaluate the safety and tolerability of multiple ascending doses of BIIB115 administered via IT bolus injection participants with SMA who previously received onasemnogene abeparvovec
    E.2.2Secondary objectives of the trial
    To evaluate the PK of multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric SMA participants who previously received onasemnogene abeparvovec
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age 0.5 to 12 years old, inclusive, at the time of informed consent
    - Weight ≥7 kg at the time of informed consent
    - Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1)gene deletion or mutation or compound heterozygous mutation)- Survival motor neuron 2 (SMN2) copy number ≥1
    - Must have received IV onasemnogene abeparvovec per the approved label or per guidelines including the steroid regimen and monitoring specified therein
    - Treatment with onasemnogene abeparvovec ≥180 days prior to first BIIB115 dose
    - Potential for improvement due to suboptimal clinical status secondary to SMA, as determined by the Investigator
    E.4Principal exclusion criteria
    - Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoing during Screening
    - Interval of <180 days between onasemnogene abeparvovec therapy and first BIIB115 dose
    - Ongoing steroid treatment following onasemnogene abeparvovec at time of screening
    - History of drug induced liver injury or liver failure per Hy's law definition
    - History of thrombotic micrangiopathy
    - Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receiving onasemnogene abeparvovec. Treatment with nusinersen <12 months from the first dose of BIIB115.
    - Any reason, anatomical or otherwise (including abnormal hematology/coagulation),that presents increase of risk of complication from the LP procedures, CSF circulation, or safety assessments, including a history of hydrocephalus or implanted shunt for CSF drainage
    - Permanent ventilation, defined as tracheostomy or ≥16 hours ventilation/day continuously for ≥21 days in the absence of an acute reversible event

    NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
    E.5 End points
    E.5.1Primary end point(s)
    Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to Day 720
    E.5.2Secondary end point(s)
    1. Concentration of BIIB115 in Cerebral Spinal Fluid (CSF)
    2. Concentration of BIIB115 in Serum
    3. Terminal Elimination Half-Life (t½) of BIIB115 in Serum
    4. Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum
    5. Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum
    6. Maximum Observed Concentration (Cmax) of BIIB115 in Serum
    7. Time to Reach Maximum Observed Concentration (Tmax) of BIIB115 in Serum
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Days 1 and 360
    2. Day 1 to Day 720
    3. Day 1 to Day 720
    4. Day 1 to Day 720
    5. Day 1 to Day 720
    6. Day 1 to Day 720
    7. Day 1 to Day 720
    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 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
    Tolerability
    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) No
    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
    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 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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Australia
    Canada
    Israel
    France
    Poland
    Netherlands
    Spain
    Germany
    Italy
    Belgium
    Portugal
    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 years4
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days3
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days3
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 8
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 14
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric Participants aged 0.5 to 12
    years, inclusive
    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 18
    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)
    Participants with SMA in Part B who complete this study and do not discontinue study treatment may be offered the option to enter an open-label extension study (under a separate protocol), provided they meet all eligibility criteria for that study. Initiation of an open-label extension study is also contingent on emerging safety, tolerability, PK, and PD data, as well as approval by ethics committees and the appropriate regulatory authorities.
    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 Decision2023-05-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-08-18
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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