A Prospective Exploratory Study of the Safety and Preliminary Efficacy of the α-Syn H21 Monoclonal Antibody in Patients With Multiple System Atrophy
17. srpna 2026 aktualizováno: Ruijin Hospital
Multiple system atrophy (MSA) is a progressive neurodegenerative disorder characterized by autonomic dysfunction, parkinsonism, and cerebellar ataxia. Abnormal aggregation of alpha-synuclein is believed to play an important role in disease progression. The α-Syn H21 monoclonal antibody is designed to selectively bind pathological alpha-synuclein aggregates and may reduce their spread and related neuroinflammation.
This single-center, prospective, exploratory study will evaluate the safety, tolerability, and preliminary efficacy of the α-Syn H21 monoclonal antibody in patients with MSA. Participants will receive intravenous infusions of H21 every 4 weeks for 3 doses and will be followed for 12 weeks. Clinical symptoms, laboratory tests, imaging findings, and adverse events will be assessed to determine whether H21 may provide clinical benefit and support future larger studies.
Přehled studie
Postavení
Zatím nenabíráme
Podmínky
Intervence / Léčba
Typ studie
Intervenční
Zápis (Odhadovaný)
3
Fáze
- Fáze 1
Kontakty a umístění
Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.
Studijní kontakt
- Jméno: Jun Liu, MD, PhD
- Telefonní číslo: 021-86-64370045
- E-mail: jly0520@hotmail.com
Studijní záloha kontaktů
- Jméno: Yan SHEN, MD, PhD
- Telefonní číslo: +86-021-64370045
- E-mail: shenyanzmins@sina.com
Studijní místa
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Shanghai Municipality
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Shanghai, Shanghai Municipality, Čína, 200025
- Department of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, Shanghai 200025
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Kontakt:
- Jun LIU, MD, PhD
- Telefonní číslo: 86-021-64370045
- E-mail: jly0520@hotmail.com
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Kritéria účasti
Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Ne
Popis
Inclusion Criteria:
- Age 45 to 75 years, male or female
- Diagnosis of Multiple System Atrophy meeting current clinical diagnostic criteria for probable or possible MSA
- Disease duration of 2 years or less from onset of motor or autonomic symptoms
- Clinically stable disease without significant fluctuation or acute worsening within 4 weeks before enrollment
- Able to comply with study procedures and follow-up assessments
- Mini-Mental State Examination (MMSE) score not consistent with significant dementia
- Willing and able to provide written informed consent
Exclusion Criteria:
- History or presence of other neurological disorders that may interfere with study assessments, including Parkinson's disease, progressive supranuclear palsy, corticobasal degeneration, or stroke
- Severe cognitive impairment or psychiatric disorder, including clinically significant depression or anxiety
- Severe cardiac, hepatic, renal, or other major systemic disease
- History of severe hypersensitivity to monoclonal antibody therapies Pregnant or breastfeeding women
- Women or men unwilling to use effective contraception during the study
- Participation in another clinical trial or receipt of investigational treatment within 3 months before enrollment
- Any other condition that, in the investigator's judgment, would make participation inappropriate
Studijní plán
Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: N/A
- Intervenční model: Přiřazení jedné skupiny
- Maskování: Žádné (otevřený štítek)
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
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Experimentální: H21 Treatment
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The α-Syn H21 monoclonal antibody is a humanized monoclonal antibody designed to selectively bind pathological alpha-synuclein aggregates.
Participants will receive the study drug by intravenous infusion once every 4 weeks for a total of 3 doses during the 12-week study period.
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Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Incidence of treatment-emergent adverse events
Časové okno: From first dose through Week 12
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Incidence, severity, and relationship of adverse events (AEs) and serious adverse events (SAEs) to study treatment.
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From first dose through Week 12
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Change from Baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) Total Score (Parts I-IV)
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Change from baseline in the total score and subscale scores of the Unified Multiple System Atrophy Rating Scale (UMSARS Parts I-IV).
The UMSARS is a clinician-administered scale used to assess disease severity in patients with multiple system atrophy.
Total scores are derived from Parts I-IV, with higher scores indicating greater disease severity and worse clinical status.
The total score ranges from 0 to 249, with higher scores indicating more severe impairment.
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Baseline, Week 4, Week 8, and Week 12
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Change from baseline in DAT-PET/MRI measures of striatal dopamine transporter uptake and brain structural changes
Časové okno: Baseline and Week 12
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Change from baseline in DAT-PET/MRI imaging parameters, including brain metabolic and structural changes.
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Baseline and Week 12
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Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Change from Baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Change from baseline in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS Parts I-IV) total and subscale scores.
The MDS-UPDRS is a clinician-administered scale used to assess the severity and progression of Parkinsonian symptoms.
The total score ranges from 0 to 260, with higher scores indicating greater disease severity and worse motor and non-motor impairment.
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Baseline, Week 4, Week 8, and Week 12
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Change in Patient Global Impression of Improvement (PGI-I) Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
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The Patient Global Impression of Improvement (PGI-I) is a patient-reported measure assessing overall perceived improvement following treatment.
Scores range from 1 (very much improved) to 7 (very much worse), with lower scores indicating better perceived improvement.
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Baseline, Week 4, Week 8, and Week 12
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Change from Baseline in Mini-Mental State Examination (MMSE) Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
|
Change from baseline in the Mini-Mental State Examination (MMSE) score.
The MMSE is a widely used clinician-administered screening tool for global cognitive function, assessing domains including orientation, attention, memory, language, and visuospatial abilities.
Scores range from 0 to 30, with higher scores indicating better cognitive function and lower scores indicating greater cognitive impairment.
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Baseline, Week 4, Week 8, and Week 12
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Change from Baseline in Hamilton Depression Rating Scale (HAMD) Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Change from baseline in the Hamilton Depression Rating Scale (HAMD) score.
The HAMD is a clinician-administered scale used to assess the severity of depressive symptoms.
Scores range from 0 to 52 (17-item version), with higher scores indicating more severe depressive symptoms and lower scores indicating less severe depression.
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Baseline, Week 4, Week 8, and Week 12
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Change from Baseline in Hamilton Anxiety Rating Scale (HAMA) Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Change from baseline in the Hamilton Anxiety Rating Scale (HAMA) score.
The HAMA is a clinician-administered scale used to assess the severity of anxiety symptoms.
Scores range from 0 to 56, with higher scores indicating more severe anxiety symptoms and lower scores indicating less severe anxiety.
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Baseline, Week 4, Week 8, and Week 12
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Change from Baseline in Parkinson's Disease Sleep Scale-2 (PDSS-2) Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Change from baseline in the Parkinson's Disease Sleep Scale-2 (PDSS-2) score.
The PDSS-2 is a patient-reported scale used to assess the severity of nocturnal disturbances and sleep-related symptoms in patients with Parkinson's disease.
Scores range from 0 to 60, with higher scores indicating more severe sleep disturbances and poorer sleep quality.
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Baseline, Week 4, Week 8, and Week 12
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Change from Baseline in Parkinson's Disease Questionnaire-39 (PDQ-39) Score
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Change from baseline in the Parkinson's Disease Questionnaire-39 (PDQ-39) score.
The PDQ-39 is a patient-reported questionnaire used to assess health-related quality of life in patients with Parkinson's disease across eight domains, including mobility, activities of daily living, emotional well-being, and social support.
Scores range from 0 to 156, with higher scores indicating poorer health-related quality of life and greater disease impact.
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Baseline, Week 4, Week 8, and Week 12
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Number of Participants With Clinically Significant Abnormal Laboratory Values
Časové okno: Baseline, Week 4, Week 8, and Week 12
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The number of participants with clinically significant abnormal laboratory values following H21 administration, including abnormalities in hematology, urinalysis, liver function, and renal function tests, as assessed by the investigator.
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Baseline, Week 4, Week 8, and Week 12
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Change from baseline in electrocardiogram parameters including heart rhythm, PR interval, QRS duration, and QTc interval
Časové okno: Baseline, Week 4, Week 8, and Week 12
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Standard 12-lead electrocardiograms will be performed at scheduled visits to assess changes from baseline in cardiac conduction and rhythm parameters, including heart rhythm, PR interval, QRS duration, and corrected QT (QTc) interval, following H21 administration.
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Baseline, Week 4, Week 8, and Week 12
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Change in Blood Pressure After Infusion
Časové okno: Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
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Change in blood pressure measured before each H21 infusion and at 10 minutes and 30 minutes after infusion to evaluate short-term hemodynamic changes associated with study drug administration.
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Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
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Change in Heart Rate After Infusion
Časové okno: Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
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Change in heart rate measured before each H21 infusion and at 10 minutes and 30 minutes after infusion to evaluate short-term cardiac responses associated with study drug administration.
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Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
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Change in Respiratory Rate After Infusion
Časové okno: Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
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Change in respiratory rate measured before each H21 infusion and at 10 minutes and 30 minutes after infusion to evaluate short-term respiratory changes associated with study drug administration.
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Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
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Serum Concentration of H21
Časové okno: During treatment through Week 12
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Serum concentrations of H21 will be measured at prespecified time points following administration to characterize the concentration-time profile of H21.
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During treatment through Week 12
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Maximum Observed Serum Concentration (Cmax) of H21
Časové okno: During treatment through Week 12
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The maximum observed serum concentration (Cmax) of H21 following administration.
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During treatment through Week 12
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Time to Maximum Observed Serum Concentration (Tmax) of H21
Časové okno: During treatment through Week 12
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The time to reach the maximum observed serum concentration (Tmax) of H21 following administration.
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During treatment through Week 12
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Area Under the Serum Concentration-Time Curve (AUC) of H21
Časové okno: During treatment through Week 12
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The area under the serum concentration-time curve (AUC) of H21 following administration.
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During treatment through Week 12
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Terminal Elimination Half-life (t1/2) of H21
Časové okno: During treatment through Week 12
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The terminal elimination half-life (t1/2) of H21 following administration.
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During treatment through Week 12
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Spolupracovníci a vyšetřovatelé
Zde najdete lidi a organizace zapojené do této studie.
Sponzor
Termíny studijních záznamů
Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.
Hlavní termíny studia
Začátek studia (Odhadovaný)
15. září 2026
Primární dokončení (Odhadovaný)
31. prosince 2026
Dokončení studie (Odhadovaný)
31. prosince 2026
Termíny zápisu do studia
První předloženo
12. května 2026
První předloženo, které splnilo kritéria kontroly kvality
17. srpna 2026
První zveřejněno (Aktuální)
19. srpna 2026
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
19. srpna 2026
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
17. srpna 2026
Naposledy ověřeno
1. dubna 2026
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
Další identifikační čísla studie
- H21-MSA
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
NE
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Ne
Studuje produkt zařízení regulovaný americkým úřadem FDA
Ne
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