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    Summary
    EudraCT Number:2019-002827-14
    Sponsor's Protocol Code Number:TreatSPG11
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-05-24
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-002827-14
    A.3Full title of the trial
    Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11
    Studio sperimentale farmacologico di fase 2 per valutare la sicurezza della somministrazione di miglustat in soggetti con paraparesi spastica di tipo 11 (TreatSPG11)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phamacological study to test safety of miglustat in patients with SPG11
    Studio sperimentale per valutare la sicurezza della somministrazione di miglustat in soggetti con paraparesi spastica di tipo 11
    A.3.2Name or abbreviated title of the trial where available
    TreatSPG11
    TreatSPG11
    A.4.1Sponsor's protocol code numberTreatSPG11
    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 SponsorIRCCS Fondazione Stella Maris
    B.1.3.4CountryItaly
    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 supportIRCCS Fondazione Stella Maris
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportTom Wahlig Stiftung Foundation
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS Fondazione Stella Maris
    B.5.2Functional name of contact pointSegreteria Scientifica
    B.5.3 Address:
    B.5.3.1Street Addressviale del Tirreno, 331
    B.5.3.2Town/ cityPisa
    B.5.3.3Post code56128
    B.5.3.4CountryItaly
    B.5.4Telephone number050886233
    B.5.6E-maildirscient@fsm.unipi.it
    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 Yes
    D.2.1.1.1Trade name MIGLUSTAT GEN.ORPH - 100 MG - CAPSULA, RIGIDA - USO ORALE - BLISTER (OPA/ALLU/PVC) - 84 CAPSULE
    D.2.1.1.2Name of the Marketing Authorisation holderGEN.ORPH S.A.S.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameMiglustat Gen.Orph 100 mg, capsule rigide
    D.3.2Product code [Miglustat Gen.Orph 100 mg, capsule rigide]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMIGLUSTAT
    D.3.9.2Current sponsor codeOPR000000419
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    D.3.11.13.1Other medicinal product typeAltri prodotti per il tubo digerente e il metabolismo, codice ATC: A16AX06
    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
    Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy of the corpus callosum and retinal degeneration. The disease progresses with degeneration of motor and cognitive functions up to total dependence on caregivers. At the moment there are no therapies capable of modifying its natural history and progression.
    Paraparesi spastica di tipo 11 (SPG11) è la forma di paraparesi spastica autosomica recessiva più frequente dovuta a mutazioni della proteina spatacsina. E' caratterizzata dall’ associazione di neuropatia periferica, parkinsonismo, atassia, deficit cognitivo, e degenerazione retinica. La malattia progredisce con degenerazione delle funzioni motorie e cognitive fino alla totale dipendenza dai caregivers. Al momento non vi sono terapie capaci di modificarne la storia naturale e la progressione.
    E.1.1.1Medical condition in easily understood language
    SPG11 is a rare, uncurable neurodegenerative disease characterized by weak and stiff legs, ataxia, dementia with progressive loss of autonomy.
    La SPG11 è una malattia genetica del cervello e dei nervi caratterizzata da debolezza e rigidità degli arti, parkinsonismo, disequilibrio, e perdita di autonomia ed è senza cura.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10019903
    E.1.2Term Hereditary spastic paraplegia
    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
    Evaluate the tolerability and safety of miglustat administration in a group of 10 subjects with moderate form of SPG11
    Valutare la tollerabilità e la sicurezza dell’assunzione di miglustat in un gruppo di 10 soggetti affetti da forma moderata di SPG11
    E.2.2Secondary objectives of the trial
    Quantize the glycosphingolipids and GM2 gangliosides in patient serum over the course of the study.
    Evaluate changes in disease progression and severity (± 20% changes in clinical SPRS scores) during treatment with miglustat.
    Quantizzare i glicosfingolipidi e i gangliosidi GM2 nel siero dei pazienti nell’arco dello studio.
    Valutare le modifiche di progressione e di severità di malattia (variazioni di ± 20% nei punteggi della scala clinica SPRS) durante il trattamento con miglustat.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Diagnosis of SPG11 confirmed by genetic analysis positive for biallelic mutations in the SPG11 gene.
    Age > 13 years.
    Moderate degree of disease severity (SPRS disease scale > 10 and < 35).
    Signature of informed consent for enrollment in the study.
    Acceptance of the use of contraceptive methods (for subjects of childbearing age).
    Diagnosi di SPG11 confermata tramite analisi genetica positiva per mutazioni bialleliche nel gene SPG11.
    Età >13 anni.
    Grado di malattia moderato (scala di malattia SPRS >10 e <35).
    Firma del consenso informato per l'arruolamento nello studio.
    Accettazione all’uso di metodi contraccettivi (per i soggetti in età fertile).
    E.4Principal exclusion criteria
    Diagnosis of other concomitant neurodegenerative disease.
    Very severe (SPRS = 35 scale) or mild / paucisymptomatic (SPRS =10) SPG11 disease.
    Outcomes of severe pre- / peri-natal suffering.
    Sensitivity or intolerance to miglustat.
    Participation in other pharmacological studies within 30 days of the first visit of this study (T0).
    Inability to take the study drug due to severe swallowing problems or the presence of percutaneous endoscopic gastrostomy access.
    Other medical conditions that may interfere with the study results or the safety of the participants.
    Intractable diarrhea in the 3 months prior to enrollment.
    Diagnosi di altra patologia neurodegenerativa concomitante.
    Malattia SPG11 molto grave (scala di SPRS = 35) oppure lieve/paucisintomatica (SPRS =10).
    Esiti di grave sofferenza pre-/peri-natale.
    Sensibilità o intolleranza al miglustat.
    Partecipazione ad altri studi farmacologici entro 30 giorni dalla prima visita del presente studio (T0).
    Incapacità ad assumere il farmaco in studio per gravi problemi di deglutizione o per la presenza di accesso di gastrostomia endoscopica percutanea (PEG).
    Altre condizioni mediche che possono interferire con i risultati dello studio o con la sicurezza dei partecipanti.
    Diarrea intrattabile nei 3 mesi precedenti l’arruolamento.
    E.5 End points
    E.5.1Primary end point(s)
    Percentage of subjects reporting serious / significant adverse reactions or changes in blood or neurophysiological parameters greater than ± 20% (or both) during the study assessed at the two follow-up visits at 12 weeks (T1) and 24 weeks (T2 ) from the start of the study with respect to the enrollment visit (T0)
    Percentuale di soggetti che durante lo studio riportano nuovi eventi avversi intesi come EA clinici gravi/significativi o variazioni significative negli indici ematici o neurofisiologici (maggiori di ± 20% valutati a T1 e T2 rispetto alla visita di arruolamento) o entrambi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From the first day of drug administration (day after the enrollment visit) to the completion visit (T2, 24 weeks from enrollment).
    Dal primo giorno di somministrazione del farmaco (giorno dopo della visita di arruolamento) fino alla visita di conclusione di partecipazione (T2, 24 settimane dall'arruolamento).
    E.5.2Secondary end point(s)
    Greater than ± 20% variations in plasma GM2 glycosphingolipid and ganglioside levels (expressed as percentages of the total pool of identified glycosphingolipids (e.g.% total profile, mean ± SD) (Fan et al., 2013) measured at T1 and T2 compared to measurement performed during the enrollment visit (T0).; Score changes greater than ± 20% in the scores of the clinical disease scales and questionnaires at T1 and T2 compared to the scores obtained at the enrollment visit (T0).
    Variazioni maggiori di ± 20% nel livello di glicosfingolipidi e gangliosidi GM2 plasmatici (espressi come percentulae del pool totale di glicosphingolipidi identificati (es. % profilo totale, media±SD) (Fan et al., 2013) misurati a T1 e T2 rispetto alla misurazione eseguita durante la visita di arruolamento (T0).; Variazioni di punteggio maggiori di ± 20% nei punteggi delle scale cliniche di malattia e dei questionari rilevati a T1 e T2 rispetto ai punteggi ottenuti alla visita di arruolamento (T0).
    E.5.2.1Timepoint(s) of evaluation of this end point
    From the enrollment visit to the completion visit (T2, 24 weeks from enrollment).; From the enrollment visit to the completion visit (T2, 24 weeks from enrollment).
    Dalla visita di arruolamento fino alla visita di conclusione di partecipazione (T2, 24 settimane dall'arruolamento).; Dalla visita di arruolamento fino alla visita di conclusione di partecipazione (T2, 24 settimane dall'arruolamento).
    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 No
    E.6.6Pharmacokinetic No
    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) 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 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 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 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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    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 years0
    E.8.9.2In all countries concerned by the trial months7
    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.1.1.1In 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: 4
    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.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 10
    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)
    At the end of the treatment, the participants will be followed at the UOC5 of the IRCCS Stella Maris Foundation as usual clinical practice.
    Al termie del trattamento i partecipanti continueranno ad essere seguiti presso la UOC5 dell’IRCCS Fondazione Stella Maris come da usuale pratica clinica.
    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 Decision2021-01-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-19
    P. End of Trial
    P.End of Trial StatusCompleted
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