A Prospective Exploratory Study of the Safety and Preliminary Efficacy of the α-Syn H21 Monoclonal Antibody in Patients With Multiple System Atrophy
17 sierpnia 2026 zaktualizowane przez: 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.
Przegląd badań
Status
Jeszcze nie rekrutacja
Warunki
Interwencja / Leczenie
Typ studiów
Interwencyjne
Zapisy (Szacowany)
3
Faza
- Faza 1
Kontakty i lokalizacje
Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.
Kontakt w sprawie studiów
- Nazwa: Jun Liu, MD, PhD
- Numer telefonu: 021-86-64370045
- E-mail: jly0520@hotmail.com
Kopia zapasowa kontaktu do badania
- Nazwa: Yan SHEN, MD, PhD
- Numer telefonu: +86-021-64370045
- E-mail: shenyanzmins@sina.com
Lokalizacje studiów
-
-
Shanghai Municipality
-
Shanghai, Shanghai Municipality, Chiny, 200025
- Department of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, Shanghai 200025
-
Kontakt:
- Jun LIU, MD, PhD
- Numer telefonu: 86-021-64370045
- E-mail: jly0520@hotmail.com
-
-
Kryteria uczestnictwa
Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.
Kryteria kwalifikacji
Wiek uprawniający do nauki
- Dorosły
- Starszy dorosły
Akceptuje zdrowych ochotników
Nie
Opis
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
Plan studiów
Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Nie dotyczy
- Model interwencyjny: Zadanie dla jednej grupy
- Maskowanie: Brak (otwarta etykieta)
Broń i interwencje
Grupa uczestników / Arm |
Interwencja / Leczenie |
|---|---|
|
Eksperymentalny: H21 Treatment
|
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.
|
Co mierzy badanie?
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
|
Incidence of treatment-emergent adverse events
Ramy czasowe: From first dose through Week 12
|
Incidence, severity, and relationship of adverse events (AEs) and serious adverse events (SAEs) to study treatment.
|
From first dose through Week 12
|
|
Change from Baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) Total Score (Parts I-IV)
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from baseline in DAT-PET/MRI measures of striatal dopamine transporter uptake and brain structural changes
Ramy czasowe: Baseline and Week 12
|
Change from baseline in DAT-PET/MRI imaging parameters, including brain metabolic and structural changes.
|
Baseline and Week 12
|
Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
|
Change from Baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change in Patient Global Impression of Improvement (PGI-I) Score
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from Baseline in Mini-Mental State Examination (MMSE) Score
Ramy czasowe: 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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from Baseline in Hamilton Depression Rating Scale (HAMD) Score
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from Baseline in Hamilton Anxiety Rating Scale (HAMA) Score
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from Baseline in Parkinson's Disease Sleep Scale-2 (PDSS-2) Score
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from Baseline in Parkinson's Disease Questionnaire-39 (PDQ-39) Score
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Number of Participants With Clinically Significant Abnormal Laboratory Values
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change from baseline in electrocardiogram parameters including heart rhythm, PR interval, QRS duration, and QTc interval
Ramy czasowe: Baseline, Week 4, Week 8, and Week 12
|
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.
|
Baseline, Week 4, Week 8, and Week 12
|
|
Change in Blood Pressure After Infusion
Ramy czasowe: Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
|
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.
|
Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
|
|
Change in Heart Rate After Infusion
Ramy czasowe: Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
|
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.
|
Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
|
|
Change in Respiratory Rate After Infusion
Ramy czasowe: Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
|
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.
|
Pre-dose and 10 minutes and 30 minutes after each infusion through Week 12
|
|
Serum Concentration of H21
Ramy czasowe: During treatment through Week 12
|
Serum concentrations of H21 will be measured at prespecified time points following administration to characterize the concentration-time profile of H21.
|
During treatment through Week 12
|
|
Maximum Observed Serum Concentration (Cmax) of H21
Ramy czasowe: During treatment through Week 12
|
The maximum observed serum concentration (Cmax) of H21 following administration.
|
During treatment through Week 12
|
|
Time to Maximum Observed Serum Concentration (Tmax) of H21
Ramy czasowe: During treatment through Week 12
|
The time to reach the maximum observed serum concentration (Tmax) of H21 following administration.
|
During treatment through Week 12
|
|
Area Under the Serum Concentration-Time Curve (AUC) of H21
Ramy czasowe: During treatment through Week 12
|
The area under the serum concentration-time curve (AUC) of H21 following administration.
|
During treatment through Week 12
|
|
Terminal Elimination Half-life (t1/2) of H21
Ramy czasowe: During treatment through Week 12
|
The terminal elimination half-life (t1/2) of H21 following administration.
|
During treatment through Week 12
|
Współpracownicy i badacze
Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.
Sponsor
Daty zapisu na studia
Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.
Główne daty studiów
Rozpoczęcie studiów (Szacowany)
15 września 2026
Zakończenie podstawowe (Szacowany)
31 grudnia 2026
Ukończenie studiów (Szacowany)
31 grudnia 2026
Daty rejestracji na studia
Pierwszy przesłany
12 maja 2026
Pierwszy przesłany, który spełnia kryteria kontroli jakości
17 sierpnia 2026
Pierwszy wysłany (Rzeczywisty)
19 sierpnia 2026
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
19 sierpnia 2026
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
17 sierpnia 2026
Ostatnia weryfikacja
1 kwietnia 2026
Więcej informacji
Terminy związane z tym badaniem
Dodatkowe istotne warunki MeSH
Inne numery identyfikacyjne badania
- H21-MSA
Plan dla danych uczestnika indywidualnego (IPD)
Planujesz udostępniać dane poszczególnych uczestników (IPD)?
NIE
Informacje o lekach i urządzeniach, dokumenty badawcze
Bada produkt leczniczy regulowany przez amerykańską FDA
Nie
Bada produkt urządzenia regulowany przez amerykańską FDA
Nie
Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .