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    Summary
    EudraCT Number:2022-000193-25
    Sponsor's Protocol Code Number:BHV2000-301
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-09-29
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2022-000193-25
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)
    Randomizowane, podwójnie zaślepione, kontrolowane placebo badanie oceniające skuteczność i bezpieczeństwo Taldefgrobepu Alfa u pacjentów z rdzeniowym zanikiem mięśni leczonych ambulatoryjnie i nieambulatoryjnie, z kontynuacyjnym badaniem prowadzonym metodą otwartej próby (RESILIENT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to test Taldefgropeb Alfa in patients with Spinal Muscular Atrophy.
    Badanie testujące Taldefgropeb Alfa u pacjentów z rdzeniowym zanikiem mięśni.
    A.3.2Name or abbreviated title of the trial where available
    RESILIENT
    RESILIENT
    A.4.1Sponsor's protocol code numberBHV2000-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05337553
    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 SponsorBiohaven Pharmaceuticals, 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 supportBiohaven Pharmaceuticals, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiohaven Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointJackie Marin
    B.5.3 Address:
    B.5.3.1Street Address215 Church St
    B.5.3.2Town/ cityNew Haven
    B.5.3.3Post codeCT 06510
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018607827628
    B.5.6E-mailjackie.marin@biohavenpharma.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 nametaldefgrobep alfa
    D.3.2Product code BHV-2000
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTaldefgrobep alfa
    D.3.9.2Current sponsor codeBHV-2000
    D.3.9.4EV Substance CodeSUB192191
    D.3.10 Strength
    D.3.10.1Concentration unit ml millilitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number35
    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) 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.IMP: 2
    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 nametaldefgrobep alfa
    D.3.2Product code BHV-2000
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTaldefgrobep alfa
    D.3.9.2Current sponsor codeBHV-2000
    D.3.9.4EV Substance CodeSUB192191
    D.3.10 Strength
    D.3.10.1Concentration unit ml millilitre(s)
    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 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) 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection in pre-filled syringe
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Spinal Muscular Atrophy (SMA)
    Rdzeniowy zanik mięśni
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy (SMA)
    Rdzeniowy zanik mięśni
    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 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 efficacy of taldefgrobep alfa in participants who are already taking a stable dose
    of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to
    placebo, measured by change in the 32 item Motor Function Measure (MFM-32) total score
    between Baseline and Week 48
    Ocena skuteczności stosowania taldefgrobepu alfa u uczestników, którzy przyjmują już nusinersen lub risdiplam w stałej dawce lub którzy stosowali w przeszłości onasemnogen abeparwowek-xioi, w porównaniu z placebo dokonana na podstawie pomiaru zmiany całkowitego wyniku w 32-elementowej skali oceny funkcji motorycznych (ang. 32-item Motor Function Measure, MFM-32) pomiędzy wizytą wyjściową a wizytą w tygodniu 48.
    E.2.2Secondary objectives of the trial
    •To compare the efficacy of taldefgrobep alfa to placebo using the Revised Upper Limb Module (RULM).
    • To compare the efficacy of taldefgrobep alfa to placebo using the Revised Hammersmith Scale (RHS)
    • To assess the safety and tolerability of taldefgrobep alfa as reflected byincluding change from baseline in lean body mass and bone mineral density on DXA scan at Week 48, Tanner staging for puberty monitoring, new or worsening lab abnormalities, injection
    acceptability assessments, treatment related adverse events (AEs)), serious AEs and AEs leading to discontinuation.
    • To assess pharmacokinetic (PK) parameters estimated with population PK modeling
    • Porównanie skuteczności taldefgrobepu alfa względem placebo z użyciem zrewidowanej skali do oceny funkcji kończyn górnych (Revised Upper Limb Module, RULM)
    • Porównanie skuteczności taldefgrobepu alfa względem placebo z użyciem zrewidowanej skali Hammersmith (Revised Hammersmith Scale, RHS)
    • Ocena bezpieczeństwa stosowania i tolerancji taldefgrobepu alfa odzwierciedlona poprzez
    uwzględnienie zmian w beztłuszczowej masie ciała oraz gęstości mineralnej kości uwidocznionej w badaniu DXA na wizycie w tyg. 48 w porównaniu do wizyty wyjściowej, skali Tannera oceniającej dojrzałość płciową, na podstawie nowych lub nasilających się nieprawidłowości w wynikach badań laboratoryjnych, ocenach akceptowalności wstrzyknięć badanego produktu leczniczego, zdarzeń niepożądanych związanych z leczeniem, ciężkich zdarzeń niepożądanych oraz zdarzeń niepożądanych prowadzących do przerwania leczenia
    • Ocena parametrów badań farmakokinetycznych (PK) oszacowanych za pomocą modelowania PK w populacji
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ● Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number

    ● Ambulant or non-ambulant

    ● Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial, including the following:
    i. a stable regimen of nusinersen for 6 months prior to Screening; and/or
    ii. a stable regimen of risdiplam, for 6 months prior to Screening; and/or
    iii. a single dose of onasemnogene abeparvovec, received at least 2 years prior to Screening.
    1. Rdzeniowy zanik mięśni potwierdzony genetycznym rozpoznaniem autosomalnego recesywnego rdzeniowego zaniku mięśni (5q-SMA), jak również liczbą kopii genu SMN2
    2. Osoba chodząca lub niechodząca.
    3. Stosowanie leczenia zmieniającego przebieg SMA (przy założeniu, że pozostanie ono niezmienne – zarówno w odniesieniu do schematu, jak i dawkowania – przez cały okres trwania badania) obejmującego:
    i. stały schemat podawania nusinersenu przez 6 miesięcy poprzedzających wizytę przesiewową,
    ii. stały schemat podawania risdiplamu przez 6 miesięcy poprzedzających wizytę przesiewową i/lub
    iii. pojedynczą dawkę onasemnogenu abeparwoweku przyjętą co najmniej 2 lata przed wizytą przesiewową.
    E.4Principal exclusion criteria
    ● Receiving or have received previous administration of anti-myostatin therapies

    ● Weight <15 kg

    ● Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)

    ● History of spinal fusion or major surgeries within 6 months prior to screening or planned during the study. Non-surgical adjustments are allowed during the study (such as MAGEC rods).

    ● Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in the MFM-32 at Week 48
    Zmiana wyniku oceny w skali MFM-32 podczas wizyty w tygodniu 48 względem wizyty wyjściowej.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Double blind phase: Baseline, visit 5, visit 6, visit 7, visit 8
    Faza podwójnie ślepej próby: wizyta wyjściowa, wizyta 5, wizyta 6, wizyta 7, wizyta 8
    E.5.2Secondary end point(s)
    - Change from baseline in the RULM at Week 48
    - Change from baseline in the RHS at Week 48
    - Frequency of unique subjects with: new or worsening lab abnormalities, treatment related
    adverse events, serious adverse events, and adverse events leading to discontinuation.
    • Zmiana wyniku oceny w skali RULM podczas wizyty w tygodniu 48 względem wizyty wyjściowej.
    • Zmiana wyniku oceny w skali RHS podczas wizyty w tygodniu 48 względem wizyty wyjściowej.
    • Odsetek indywidualnych uczestników, u których doszło do nasilenia się lub wystąpienia nowych nieprawidłowości w wynikach badań laboratoryjnych, wystąpienia zdarzeń niepożądanych związanych z leczeniem, wystąpienia ciężkich zdarzeń niepożądanych lub wystąpienia zdarzeń niepożądanych skutkujących przerwaniem udziału w badaniu.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Double blind phase:
    RULM: Baseline, visit 5, visit 6, visit 7, visit 8
    RHS: Baseline, visit 5, visit 6, visit 7, visit 8
    Faza podwójnie ślepej próby:
    RULM: wizyta wyjściowa, wizyta 5, wizyta 6, wizyta 7, wizyta 8
    RHS: wizyta wyjściowa, wizyta 5, wizyta 6, wizyta 7, wizyta 8
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 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
    Podwójnie zaślepione z kontynuacyjnym badaniem prowadzonym metodą otwartej próby
    Double-blind with Open label follow-up
    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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    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
    United Kingdom
    United States
    Belgium
    Czechia
    France
    Germany
    Italy
    Netherlands
    Spain
    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
    LVLS
    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 months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 191
    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: 135
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 56
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 34
    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
    subjects under age incapable of giving consent personally (participants age 4-18)
    uczestnicy niepełnoletni niezdolni do wyrażenia zgody (uczestnicy w wieku 4-18)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 71
    F.4.2.2In the whole clinical trial 180
    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
    Żadne
    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 Decision2022-10-05
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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