Strona Badania kliniczne Nct

Summary
EudraCT Number:2021-004849-20
Sponsor's Protocol Code Number:283PD201
National Competent Authority:Italy - Italian Medicines Agency
Clinical Trial Type:EEA CTA
Trial Status:Ongoing
Date on which this record was first entered in the EudraCT database:2022-09-14
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
A. Protocol Information
A.1Member State ConcernedItaly - Italian Medicines Agency
A.2EudraCT number2021-004849-20
A.3Full title of the trial
A Phase 2b, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of BIIB122 in Participants with Parkinson’s Disease
Studio di fase 2b, multicentrico, randomizzato, in doppio cieco, controllato con placebo per determinare l’efficacia e la sicurezza di BIIB122 in partecipanti con malattia di Parkinson
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
A Study to Assess the Safety of BIIB122 Tablets and if it can Slow the Worsening of Early-Stage Parkinson’s Disease in Participants Between the Ages of 30 and 80
Studio per valutare la sicurezza delle compresse di BIIB122 e se può rallentare il peggioramento della malattia di Parkinson allo stadio iniziale in partecipanti di età compresa tra i 30 e gli 80 anni.
A.3.2Name or abbreviated title of the trial where available
.
.
A.4.1Sponsor's protocol code number283PD201
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 pointN/A
B.5.3 Address:
B.5.3.1Street AddressInnovation House, 70 Norden Road
B.5.3.2Town/ cityMaidenhead
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 nameBIIB122
D.3.2Product code [DNL151, DN0001575, DN1575]
D.3.4Pharmaceutical form Tablet
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.3.9.1CAS number 2170179-24-3
D.3.9.2Current sponsor codeBIIB122
D.3.9.3Other descriptive nameDNL151, DN0001575, DN1575
D.3.9.4EV Substance CodeSUB221129
D.3.10 Strength
D.3.10.1Concentration unit mg milligram(s)
D.3.10.2Concentration typeequal
D.3.10.3Concentration number75
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 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 placeboTablet
D.8.4Route of administration of the placeboOral use
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Parkinson's Disease
malattia di Parkinson
E.1.1.1Medical condition in easily understood language
Parkinson's Disease
malattia di Parkinson
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 10061536
E.1.2Term Parkinson's disease
E.1.2System Organ Class 10029205 - Nervous system disorders
E.1.3Condition being studied is a rare disease No
E.2 Objective of the trial
E.2.1Main objective of the trial
To evaluate the efficacy of BIIB122 225 mg compared with placebo.
Valutare l’efficacia di 225 mg di BIIB122 rispetto al placebo
E.2.2Secondary objectives of the trial
To evaluate the efficacy, safety and tolerability and of BIIB122 225 mg compared with placebo
valutare l'efficacia, la sicurezza e la tollerabilità di 225 mg di BIIB122 rispetto al placebo
E.2.3Trial contains a sub-study Yes
E.2.3.1Full title, date and version of each sub-study and their related objectives

Other types of substudies
Specify title, date and version of each substudy with relative objectives: Two biomarker sub-studies, a biofluid sub-study and an imaging substudy,
may be conducted to evaluate exploratory biofluid and imaging
biomarkers in a subset of participants.
- objective of biofluid sub-study is to evaluate change in central and
peripheral biomarkers over time. Collection will include repeat LPs for
CSF collection and blood sampling for PBMC. All biofluid sub-study
participants are planned to participate in CSF portion of the sub-study. A
subset of participants are planned to participate in the PBMC portion of
the sub-study. Samples will be evaluated for measures of LRRK2
pathway, lysosomal function, and exploratory measures relevant to PD.
-objective of imaging sub-study is to quantify changes in nigral-striatal
dopaminergic integrity over time using PET and MRI. The sub-study will
be conducted with participants who are willing and able to travel to
central imaging facilities from participating study sites. Participating
study sites will be based on PET radiotracer availability and site
geography, at the discretion of the Sponsor. PET imaging will use 18FAV133
radioligand that binds the vesicular monoamine transporter
(VMAT-2). MRI may include, but not be limited to, structural,
neuromelanin-sensitive, and susceptibility-weighted MRI sequences (to
be detailed in the Imaging/MRI Manual) to allow PET quantification and
evaluation of exploratory MRI biomarkers.

Altre tipologie di sottostudi
specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Potranno essere condotti due sottostudi sui biomarcatori, un sottostudio sui biofluidi e un sottostudio di diagnostica per immagini, per valutare i biomarcatori esplorativi dei biofluidi e della diagnostica per immagini in un sottogruppo di partecipanti.
- L’obiettivo del sottostudio sui biofluidi è valutare la variazione nei biomarcatori centrali e periferici nel tempo. Il prelievo includerà la ripetizione delle PL per il prelievo di LCS e il prelievo di sangue per le cellule mononucleate da sangue periferico (PBMC). Si prevede che tutti i partecipanti al sottostudio sui biofluidi partecipino alla porzione di LCS del sottostudio. Si prevede che un sottogruppo di partecipanti partecipino alla porzione di PBMC del sottostudio. I campioni saranno valutati per le misure della via della chinasi ripetuta 2 ricca di leucine (LRRK2), la funzione lisosomiale e le misure esplorative rilevanti per la malattia di Parkinson (PD).
- L’obiettivo del sottostudio di diagnostica per immagini è quantificare le variazioni nell’integrità dopaminergica nigro-striatale nel tempo utilizzando la tomografia ad emissione di positroni (PET) e la RM. Il sottostudio sarà condotto con i partecipanti che sono disposti e in grado di recarsi presso le strutture di diagnostica per immagini centrali dai centri dello studio partecipanti. I centri dello studio partecipanti si baseranno sulla disponibilità di radiotraccianti PET e sull’area geografica del centro, a discrezione dello sponsor. La PET utilizzerà il radioligando 18FAV133 che si lega al trasportatore vescicolare delle monoamine 2 (VMAT-2). La RM può includere, a titolo esemplificativo ma non esaustivo, sequenze di RM strutturali, sensibili alla neuromelanina e pesate in suscettibilità (da indicare in dettaglio nel Manuale di diagnostica per immagini/RM), per consentire la quantificazione della PET e la valutazione di biomarcatori RM esplorativi.
E.3Principal inclusion criteria
Key Inclusion Criteria:
- Clinical diagnosis of PD meeting the Movement Disorder Society Clinical Diagnostic Criteria within 2 years of the screening visit, inclusive, and at least 30 years of age at the time of diagnosis
- Modified Hoehn and Yahr scale, stages 1 to 2 (in OFF state), inclusive
- MDS-UPDRS Parts II and III (in OFF state) combined score less than or equal to (=)40 at screening
- Screening genetic test results verifying the absence of a pathogenic leucine-rich repeat kinase 2 (LRRK2) variant (i.e., G2019S, N1437H, R1441G, R1441C, R1441H, Y1699C, or I2020T). Participants with additional LRRK2 variants may be excluded if data emerge to convincingly support an association of the variants with LRRK2-PD pathogenicity. Confirmation of this eligibility requirement may come from an accredited genetic test that includes all exclusionary LRRK2 genetic variants.

NOTE: Other protocol defined Inclusion criteria may apply. See Protocol section 6.1
Principali criteri di inclusione:
- Diagnosi clinica di PD che soddisfi i criteri di diagnostica clinica della Società per i disturbi del movimento entro 2 anni dalla visita di screening, incluso, e avente almeno 30 anni di età al momento della diagnosi.
- Stadio 1 e 2 della scala di Hoehn e Yahr modificata (in stato OFF), incluso.
- Punteggio combinato delle Parti II e III della Scala di valutazione della malattia di Parkinson unificata della Società per i disturbi del movimento (MDS-UPDRS) (in stato OFF) inferiore o uguale a (=) 40 allo screening.
- Risultati delle analisi genetiche di screening che accertano l’assenza di una variante patogena della chinasi ripetuta 2 ricca di leucine (LRRK2) (ovvero, G2019S, N1437H, R1441G, R1441C, R1441H, Y1699C o I2020T). I partecipanti con varianti aggiuntive di LRRK2 possono essere esclusi se emergono dei dati per supportare in modo convincente un’associazione delle varianti con la patogenicità di LRRK2-PD. Potrebbe arrivare la conferma di questo requisito di idoneità da un test genetico accreditato che includa tutte le varianti genetiche di LRRK2 di esclusione.
NOTA: possono essere applicati altri criteri di inclusione definiti dal protocollo. Vedere la sezione 6.1 del protocollo
E.4Principal exclusion criteria
Key Exclusion Criteria:
- Clinically significant neurological disorder other than PD, including but not limited to stroke, dementia, or seizure, within 5 years of screening visit, in the opinion of the Investigator
- Clinical evidence of atypical parkinsonism (e.g., multiple-system atrophy or progressive supranuclear palsy) or evidence of drug-induced parkinsonism.
- Montreal Cognitive Assessment (MoCA) score <24 at the screening visit

NOTE: Other protocol defined Exclusion criteria may apply. See Protocol section 6.2
Principali criteri di esclusione:
- Disturbo neurologico clinicamente significativo diverso dalla PD, compresi, a titolo esemplificativo ma non esaustivo, ictus, demenza o crisi convulsive, entro 5 anni dalla visita di screening, a giudizio dello sperimentatore.
- Evidenza clinica di parkinsonismo atipico (ad es., atrofia multisistemica o paralisi sopranucleare progressiva) o evidenza di parkinsonismo farmaco-indotto.
- Punteggio della Valutazione cognitiva di Montreal (MoCA) <24 alla visita di screening.
NOTA: possono essere applicati altri criteri di esclusione definiti dal protocollo. Vedere la sezione 6.2 del protocollo.
E.5 End points
E.5.1Primary end point(s)
Time to confirmed worsening in Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III combined score over the treatment period.
Tempo al peggioramento confermato del punteggio combinato delle Parti II e III della Scala unificata di valutazione della malattia di Parkinson unificata della Società per i disturbi del movimento (MDS-UPDRS) durante il periodo di trattamento.
E.5.1.1Timepoint(s) of evaluation of this end point
Minimum 48 weeks, maximum 144 weeks.
Minimo 48 settimane, massimo 144 settimane
E.5.2Secondary end point(s)
1. Incidence of AEs and SAEs during the treatment period
2. Time to confirmed worsening in MDS-UPDRS Part II score over the treatment period
3. Change in MDS-UPDRS Parts II and III combined score
4. Time to confirmed worsening in Schwab and England Activities of Daily Living Scale (SEADL) over the treatment period
5. Change in MDS-UPDRS Parts I, II, and III combined score
1. Incidenza degli EA e degli eventi avversi seri (SAE) durante il periodo di trattamento.
2. Tempo al peggioramento confermato del punteggio della Parte II dell’MDS-UPDRS durante il periodo di trattamento.
3. Variazione nel punteggio combinato delle Parti II e III dell’MDS-UPDRS.
4. Tempo al peggioramento confermato della Scala delle attività della vita quotidiana di Schwab e England (SEADL) durante il periodo di trattamento.
5. Variazione nel punteggio combinato delle Parti I, II e III dell’MDS-UPDRS.
E.5.2.1Timepoint(s) of evaluation of this end point
1. Minimum 48 weeks, maximum 144 weeks
2. Up to Week 144
3. From Baseline to Week 48
4. Up to Week 144
5. From Baseline to Week 48
1. Minimo 48 settimane, massimo144 settimane
2. fino alla settimana 144
3. Dal Baseline alla settimana 48
4. fino alla settimana 144
5. Dal Baseline alla settimana 48
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 No
E.6.10Pharmacogenetic Yes
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 Yes
E.8.1.1Randomised Yes
E.8.1.2Open No
E.8.1.3Single blind No
E.8.1.4Double blind Yes
E.8.1.5Parallel group Yes
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 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 concerned10
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA57
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 Information not present in EudraCT
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
Canada
China
Israel
Japan
United States
Austria
France
Poland
Netherlands
Spain
Germany
Italy
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
LVLS
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years3
E.8.9.1In the Member State concerned months4
E.8.9.1In the Member State concerned days20
E.8.9.2In all countries concerned by the trial years3
E.8.9.2In all countries concerned by the trial months4
E.8.9.2In all countries concerned by the trial days20
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 No
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) No
F.1.2Adults (18-64 years) Yes
F.1.2.1Number of subjects for this age range: 416
F.1.3Elderly (>=65 years) Yes
F.1.3.1Number of subjects for this age range: 224
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 state40
F.4.2 For a multinational trial
F.4.2.1In the EEA 325
F.4.2.2In the whole clinical trial 640
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
nessuno
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 Decision2022-11-21
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2022-12-19
P. End of Trial
P.End of Trial StatusOngoing

Sponsorzy i współpracownicy

Warunki medyczne

Interwencje lekowe

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