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En undersøgelse af TAK-341 i behandling af multipel systematrofi

6. maj 2026 opdateret af: Takeda

Et randomiseret, dobbeltblindt, placebokontrolleret, fase 2-studie til evaluering af effektivitet, sikkerhed, tolerabilitet, farmakokinetik og farmakodynamik af intravenøs TAK-341 hos forsøgspersoner med multipel systematrofi

Hovedformålet er at se, hvordan TAK-341 virker efter 52 uger hos deltagere med multipel systematrofi målt ved Unified Multiple System Atrophy Rating Scale Part I (UMSARS).

Undersøgelsen vil inkludere cirka 138 patienter. Deltagerne vil modtage i alt 13 intravenøse infusioner hver 4. uge cirka, disse kan være enten af ​​TAK-341 eller placebo, efter hver infusion vil nogle blodprøver blive taget og andre vurderinger afsluttet.

Dette forsøg vil blive udført i Nordamerika, Europa og Asien.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

Lægemidlet, der testes i denne undersøgelse, hedder TAK-341. Studiet vil evaluere effektivitet, sikkerhed, tolerabilitet, farmakokinetik (PK) og farmakodynamik af intravenøs (IV) TAK-341 hos deltagere med multipel systematrofi (MSA).

Undersøgelsen vil omfatte cirka 138 deltagere. Undersøgelsen omfatter en screeningsperiode på op til 42 dage (6 uger), en 52 ugers dobbeltblind behandlingsperiode og et opfølgende sikkerhedsbesøg. Deltagerne vil blive tilfældigt tildelt (tilfældigt, som at vende en mønt) til en af ​​behandlingsskemaerne - som forbliver uoplyst for deltageren, plejepersonalet og efterforskeren under undersøgelsen:

  • Tidlig PK-kohorte: TAK-341 Dosis 1
  • Tidlig PK-kohorte: Placebo
  • Hovedkohorte: TAK-341 Dosis 2
  • Hovedkohorte: Placebo

Ændringen fra baseline i UMSARS vil blive målt i uge 52 efter dosis.

Dette multicenterforsøg vil blive gennemført over hele verden. Behandlingsvarigheden i denne undersøgelse vil være 52 uger. Deltagerne vil foretage et opfølgende besøg på stedet efter cirka 90 dage efter den sidste dosis af undersøgelsesbehandlingen. Deltagere med tidlig opsigelse vil ikke foretage et opfølgende sikkerhedsbesøg.

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

158

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Danmark
      • Aarhus N, Danmark, 8200
        • Aarhus Universitetshospital
    • Capital
      • København NV, Capital, Danmark, 2400
        • Bispebjerg Hospital
  • Det Forenede Kongerige
    • Hampshire
      • Southampton, Hampshire, Det Forenede Kongerige, SO16 6YD
        • Southampton General Hospital
  • Forenede Stater
    • Michigan
      • Farmington Hills, Michigan, Forenede Stater, 48025
        • Quest Research Institute - Alcanza - HyperCore
    • Minnesota
      • Rochester, Minnesota, Forenede Stater, 55905-0001
        • Mayo Clinic
    • New York
      • New York, New York, Forenede Stater, 10016-6402
        • NYU Langone Health
    • North Carolina
      • Durham, North Carolina, Forenede Stater, 27705-4410
        • Duke University School of Medicine
    • Ohio
      • Cleveland, Ohio, Forenede Stater, 44195
        • The Cleveland Clinic Foundation
    • Texas
      • Dallas, Texas, Forenede Stater, 75390-7208
        • University of Texas Southwestern Medical Center
    • Washington
      • Spokane, Washington, Forenede Stater, 99202-1342
        • Inland Northwest Research
  • Frankrig
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, Frankrig, 13385
        • Hopitaux de La Timone
  • Italien
      • Salerno, Italien, 84131
        • Azienda Ospedaliera Universitaria OO.RR. San Giovanni di Dio Ruggi dAragona
    • Lazio
      • Rome, Lazio, Italien, 00163
        • IRCCS San Raffaele Roma
    • Lombardy
      • Milan, Lombardy, Italien, 20122
        • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
      • Milan, Lombardy, Italien, 20133
        • Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
      • Rozzano, Lombardy, Italien, 20089
        • Istituto Clinico Humanitas
    • Veneto
      • Padova, Veneto, Italien, 35126
        • Azienda Ospedale Universita Padova
  • Japan
    • Chiba
      • Chuo-ku, Chiba, Japan, 260-8677
        • Chiba University Hospital
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 060-8638
        • Hokkaido University Hospital
    • Kyoto
      • Kyoto, Kyoto, Japan, 606-8507
        • Kyoto University Hospital
    • Tokyo
      • Bunkyo-Ku, Tokyo, Japan, 113-0033
        • The University of Tokyo Hospital
      • Bunkyo-Ku, Tokyo, Japan, 113-8519
        • Medical Hospital of Tokyo Medical and Dental University
      • Kodaira-Shi, Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
  • Portugal
    • Lisbon District
      • Loures, Lisbon District, Portugal, 2674-514
        • Campus Neurológico Sénior
    • Porto District
      • Senhora da Hora, Porto District, Portugal, 4464-513
        • Hospital Pedro Hispano
  • Spanien
      • Barcelona, Spanien, 08035
        • Hospital Universitario Vall d'Hebron
      • Barcelona, Spanien, 08041
        • Hospital de La Santa Creu i Sant Pau
      • Madrid, Spanien, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spanien, 28006
        • Hospital Universitario de La Princesa
      • Valencia, Spanien, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia
    • Vizcaya
      • Barakaldo, Vizcaya, Spanien, 48903
        • Hospital Universitario Cruces
  • Tyskland
      • Berlin, Tyskland, 10117
        • Charité - Universitätsmedizin Berlin
    • Bavaria
      • München, Bavaria, Tyskland, 81377
        • Klinikum Groshadern, LMU
    • Hesse
      • Kassel, Hesse, Tyskland, 34128
        • Paracelsus-Elena-Klinik Kassel
    • Lower Saxony
      • Hanover, Lower Saxony, Tyskland, 30625
        • Medizinische Hochschule Hannover
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Tyskland, 44791
        • Universitaetsklinikum der Ruhr-Universitaet Bochum (UKRUB) - St. Josef-Hospital
      • Bonn, North Rhine-Westphalia, Tyskland, 53127
        • Deutsches Zentrum fur Neurodegenerative Erkrankung
      • Münster, North Rhine-Westphalia, Tyskland, 48149
        • Universitätsklinikum Münster
    • Saxony
      • Dresden, Saxony, Tyskland, 01307
        • Universitatsklinikum Carl Gustav Carus an der TU Dresden
      • Leipzig, Saxony, Tyskland, 04103
        • Universitatsklinikum Leipzig
  • Østrig
    • Styria
      • Graz, Styria, Østrig, 8036
        • Medizinische Universität Graz

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

40 år og ældre (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

Diagnostisk:

  1. Deltageren har en diagnose af mulig eller sandsynlig MSA ved hjælp af de modificerede Gilman et al, 2008 diagnostiske kriterier.
  2. Deltagerens indtræden af ​​første MSA-symptomer forekom ≤4 år før screening, som vurderet af investigator.
  3. Evidens for MSA-specifikke symptomer og mangler målt ved UMSARS-skalaen.

Ekskluderingskriterier:

Medicinsk historie:

1. Deltageren har enhver kontraindikation til undersøgelsesprocedurer.

Diagnostiske vurderinger:

  1. Tilstedeværelse af forvirrende diagnose og/eller tilstande, der kan påvirke deltagerens sikkerhed under undersøgelsen ifølge investigatorens vurdering.
  2. Deltagerens deltagelse i en tidligere undersøgelse af en sygdomsmodificerende terapi (med bevist modtagelse af aktiv behandling) vil kompromittere fortolkningen af ​​dataene fra denne undersøgelse, pr. konsultation med medicinsk monitor eller udpeget.

Andet:

1. Deltageren har deltaget i et andet studie, der undersøger aktiv eller passiv immunisering mod α-synuclein (αSYN) for progressiv sygdom (PD) eller MSA, eller har haft immunglobulin G-behandling inden for 6 måneder før screening.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Tredobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Placebo komparator: Placebo
Participants received TAK-341 matching placebo intravenous (IV) infusions, once every 4 weeks (Q4W) for 52 weeks.
Medicin: Placebo
TAK-341-matching placebo IV infusion
Eksperimentel: TAK-341
Participants received TAK-341, IV Q4W for 52 weeks. An early set of participants initially received 2400 milligrams (mg) to determine pharmacokinetic (PK) parameters. Following the early participants, a dose of 2000 mg was given until the data from the PK participants became available. All participants then received 2400 mg until the end of treatment at 52 weeks.
Medicin: TAK-341
TAK-341 IV infusion
Andre navne:
  • MEDI1341

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline in Modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I Total Score at Week 52
Tidsramme: Baseline, Week 52
UMSARS Part I (historical review) is a 12-item scale that was adapted from the Unified Parkinson's Disease Rating Scale (UPDRS) and is used to assess activities related to motor disability and autonomic dysfunction. In this study, the UMSARS was modified to exclude the sexual function item. Thus, total 11 items were assessed. Each item was initially scored on a scale from 0 (normal) to 4 (severe); ratings of normal (0) and mild (1) were then combined and recorded as 0, making minimum score 0 and maximum score 3. The investigator rated the average functional situation for the past 2 weeks according to findings from the participant and caregiver interview and indicated the score that best fit with the participant's status. The total score is a sum of scores from all domains and range from 0 to 33. Higher scores indicate worse impairment.
Baseline, Week 52

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline in 11-item UMSARS at Week 52
Tidsramme: Baseline, Week 52
The 11- item UMSARS includes 11 items from Part I and II to assess both motor and autonomic disability. UMSARS Part I (historical review) is used to assess activities related to motor disability and autonomic dysfunction. UMSARS Part II (motor examination) is used to measure the functional impairment and specific parkinsonian or cerebellar features. Each item was scored on a scale from 0 (normal) to 4 (severe); total score ranges from 0 to 44, higher scores indicated worse impairment.
Baseline, Week 52
Change From Baseline in the UMSARS Total Score (UMSARS Part I + Part II) at Week 52
Tidsramme: Baseline, Week 52
UMSARS total scale consists of all items from UMSARS Parts I and II. UMSARS Part I (historical review): 12-item scale used to assess activities related to motor disability and autonomic dysfunction. Each item is scored from 0 (normal) to 4 (severe). UMSARS Part II (motor examination): 14-item scale used to measure the functional impairment (for example speech, rapid alternating movements of the hands, finger taps, leg agility) of selected complex movements, and specific parkinsonian (tremor at rest) or cerebellar (ocular motor dysfunction, heel-shin test) features. The worst affected limb was assessed, and each item was scored from 0 (normal) to 4 (severe). UMSARS Part I and Part II total score is the sum of UMSARS Part I and Part II and ranges from 0 to 104. A higher score indicates worse impairment.
Baseline, Week 52
Change From Baseline in UMSARS Part I 11-Item Score at Week 52
Tidsramme: Baseline, Week 52
UMSARS Part I (historical review) is a 12-item scale that was adapted from the UPDRS. In this study, the UMSARS was modified to exclude the sexual function item. The UMSARS is used to assess activities related to motor disability and autonomic dysfunction. Each item was scored on a scale from 0 (normal) to 4 (severe). UMSARS Part I 11-item total score is the total score of UMSARS Part I, excluding the sexual function item, and without collapse of ratings of scale items. The UMSARS Part I 11-item total score ranges from 0 to 44, and higher scores indicate worse impairment.
Baseline, Week 52
Change From Baseline in UMSARS Part II at Week 52
Tidsramme: Baseline, Week 52
UMSARS Part II (motor examination): 14-item scale used to measure the functional impairment (e.g., speech, rapid alternating movements of the hands, finger taps, leg agility) of selected complex movements, and specific parkinsonian (tremor at rest) or cerebellar (ocular motor dysfunction, heel-shin test) features. the worst affected limb was assessed, and each item was scored from 0 (normal) to 4 (severe). The UMSARS Part II total score ranges from 0 to 56, and higher scores indicate worse impairment.
Baseline, Week 52
Change From Baseline on Clinical Global Impression-Severity (CGI-S) Score
Tidsramme: Baseline, Week 24 and Week 52
The CGI-S is used to assess the clinician's impression of the participant's clinical condition. The clinician rates the current severity of the participant's illness on a 7-point scale ranging from 1 (normal, not at all ill) to 7 (most extremely ill). The rating was based on observed and reported symptoms, behaviour, and function and reflected the severity level at the time of the assessment. A higher score indicates worse impairment.
Baseline, Week 24 and Week 52
Change From Baseline in Scales for Outcomes in Parkinson's Disease - Autonomic Dysfunction (SCOPA-AUT) Total Score
Tidsramme: Baseline, Week 24 and Week 52
The SCOPA-AUT is a participant-reported outcome that assesses autonomic function. Autonomic function is a critical symptom domain for MSA. The scale was completed by participants and consisted of 25 items assessing the following domains: gastrointestinal (7 items), urinary (6 items), cardiovascular (3 items), thermoregulatory (4 items), pupillomotor (1 item), and sexual (2 items for men and 2 items for women). The score for each item ranged from 0 (never experiencing the symptom) to 3 (often experiencing the symptom). The total composite score including all domains was reported. The score range was 0 (no symptoms) to 69 (highest burden of symptoms). A higher score indicates worse impairment.
Baseline, Week 24 and Week 52
Overall Survival (OS) at Week 52
Tidsramme: At Week 52
OS was estimated with Kaplan-Meier survival estimates, along with 95% confidence interval. A cox proportional hazards model was fitted to model the survival probability with treatment as the predictor. The participants with missing value were censored. The probabilities of survival at Week 52 for participants were estimated and reported.
At Week 52
Change From Baseline in Cerebrospinal Fluid (CSF) Free Alpha-Synuclein (αSYN)
Tidsramme: Baseline, Week 52
α-Synucleinopathies are diseases characterized by abnormal accumulation of aggregated αSYN. In participants with MSA, αSYN is seen to accumulate primarily in oligodendrocytes, forming glial cytoplasmic inclusions. A negative change from Baseline indicates an improvement.
Baseline, Week 52
Maximum Observed Steady State Serum Concentration (Cmax) for TAK-341
Tidsramme: On Day 57- immediately before end of infusion (EOI) (60 minutes), 6 hours and 24 hours.
Cmax is the maximum observed serum concentration for TAK-341.
On Day 57- immediately before end of infusion (EOI) (60 minutes), 6 hours and 24 hours.
Time to Maximum Steady State Concentration (Tmax) for TAK-341
Tidsramme: On Day 57- immediately before EOI (60 minutes), 6 hours and 24 hours.
Tmax is the time of first occurrence of maximum observed concentration for TAK-341.
On Day 57- immediately before EOI (60 minutes), 6 hours and 24 hours.
Area Under the Serum Concentration Time Curve (AUCτ) at Steady State for TAK-341
Tidsramme: On Day 57- immediately before EOI (60 minutes), 6 hours and 24 hours.
AUCτ is the area under the serum concentration-time curve during a dosing interval.
On Day 57- immediately before EOI (60 minutes), 6 hours and 24 hours.
CSF Concentration of TAK-341
Tidsramme: On Day 1, Day 85 and Day 365
Lumbar puncture was performed for CSF on Day 1, Day 85 and Day 365, predose.
On Day 1, Day 85 and Day 365
Number of Participants With at Least One Adverse Event (AE)
Tidsramme: Up to Week 61
An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (e.g., a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as an adverse event that occurs after administration of the first dose of study treatment and up through 90 days after the last dose of study treatment.
Up to Week 61
Number of Participants With Anti-Drug Antibodies
Tidsramme: Up to Week 61
Up to Week 61

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Takeda

Samarbejdspartnere

AstraZeneca

Efterforskere

  • Studieleder: Medical Director, Takeda

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Hjælpsomme links

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

16. november 2022

Primær færdiggørelse (Faktiske)

28. juli 2025

Studieafslutning (Faktiske)

28. juli 2025

Datoer for studieregistrering

Først indsendt

1. september 2022

Først indsendt, der opfyldte QC-kriterier

1. september 2022

Først opslået (Faktiske)

2. september 2022

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

2. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

6. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • TAK-341-2001
  • 2022-000336-28 (EudraCT nummer)
  • jRCT2011220029 (Registry Identifier: jRCT)
  • 2023-509876-40-00 (Ctis)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

Takeda giver adgang til de afidentificerede individuelle deltagerdata (IPD) for kvalificerede undersøgelser for at hjælpe kvalificerede forskere med at løse legitime videnskabelige mål (Takedas tilsagn om datadeling er tilgængelig på https://clinicaltrials.takeda.com/takedas-commitment?commitment= 5). Disse IPD'er vil blive leveret i et sikkert forskningsmiljø efter godkendelse af en anmodning om datadeling og under betingelserne i en datadelingsaftale.

IPD-delingsadgangskriterier

IPD fra kvalificerede undersøgelser vil blive delt med kvalificerede forskere i henhold til kriterierne og processen beskrevet på https://vivli.org/ourmember/takeda/ For godkendte anmodninger vil forskerne få adgang til anonymiserede data (for at respektere patientens privatliv i overensstemmelse med gældende love og regler) og med oplysninger, der er nødvendige for at opfylde forskningsmålene i henhold til vilkårene i en datadelingsaftale.

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ja

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Abonner