A Clinical Study to Evaluate the Safety, and Tolerability of BBM-P002 in the Treatment of Parkinson's Disease

Официальное название

A Single-arm, Open-label, Single-dose Clinical Study to Evaluate the Safety and Tolerability of BBM-P002 for Stereotactic Bilateral Putamen Injection in the Treatment of Idiopathic Parkinson's Disease

Обзор

The purpose of the study is to evaluate the safety and tolerability of BBM-P002 for stereotactic injection to treat participants with idiopathic Parkinson's Disease (PD) during the dose-limiting toxicity (DLT) observation period.

Подробное описание

This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-P002 within 52 weeks after into the bilateral putamen in Parkinson's disease, as well as the long-term safety and efficacy of BBM-P002 for up to 5 years post injection.

BBM-P002 is an AAV-based gene therapy that delivers a therapeutic gene cassette into putamen for the treatment of Parkinson's Disease.

Вмешательства

• Генная терапия Injecting BBM-P002 into the bilateral putamen
  BBM-P002 is an AAV-based gene therapy that delivers a therapeutic gene cassette into putamen for the treatment of Parkinson's Disease

Первичные конечные точки

• Incidence of dose limiting toxicity (DLT) events [Срок оценки: Within 4 weeks]
• Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [Срок оценки: Within 4 weeks]

Вторичные конечные точки (9)

• Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [Срок оценки: Within 5 years]
• Changes in the titers of neutralizing antibodies and binding antibodies in the recombinant adenovirus capsid of blood [Срок оценки: Within 5 years]
• Changes in the results of the cranial MRI scan [Срок оценки: Within 5 years]
• Changes from baseline in MDS - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) I/II/III/IV scores [Срок оценки: Within 5 years]
• Changes from baseline in the Patient Global Impression of Improvement (PGI-I) scores [Срок оценки: Within 5 years]
• Changes from baseline in the Clinical Global Impression of Improvement (CGI-I) scores [Срок оценки: Within 5 years]
• Changes in the activity levels of gene expression products under18F-dopa PET scanning from baseline [striatum - occipital lobe average radioactivity count ratio] [Срок оценки: Within 5 years]
• Changes from baseline in daily levodopa dose or equivalent dose of levodopa [Срок оценки: Within 5 years]
• Changes from baseline in the percentage at on time. [Срок оценки: Within 5 years]

Критерии участия

Критерии включения

• Participants with a diagnosis of Parkinson's disease for ≥ 5 years.
• The Hoehn-Yahr staging meets the requirements during the OFF periods.
• The MDS-UPDRS Part III score met the requirements during the OFF periods. the levodopa challenge test was positive
• During the screening period, participants were required to take the same stable dose of dopamine-releasing drugs (such as levodopa) regularly
• The participants agreed to postpone the neurosurgical procedures related to the indication treatment during the main study phase
• The participants agreed not to participate in any other therapeutic intervention studies during the trial period
• The participants agreed not to receive the vaccine during the main study phase
• From the screening period until at least 52 weeks after dosing, use a reliable contraceptive method
• The participants had good compliance and were able to undergo regular follow-ups. During the follow-up period, they were able to accurately complete the diary cards. Family members, guardians or caregivers could assist the subjects in filling out the diary cards
• Voluntarilyvoluntarily participated in the study and signed the informed consent form

Критерии исключения

• Atypical or secondary Parkinsonism
• Have contraindications for surgery or have previously undergone brain surgery
• The participants has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease
• Those with severe cognitive impairments
• Those with severe depression or severe anxiety
• Abnormal liver function
• Abnormal coagulation function
• Abnormalities in infectious disease screening
• Currently undergoing antiviral treatment for hepatitis
• Suffering from unstable or severe diseases in the cardiovascular, respiratory, digestive, nervous, hematological, immune and other systems
• Suffering from malignant tumors or having a history of tumors
• Previous history of severe allergies
• Exclude those who have participated in other clinical trials within the past three months
• Had received gene therapy during the screening period
• Select those who have received stem cell treatment within the past 12 months
• Exclude those who have used other investigational drugs within the past 4 weeks
• During the screening period, if the individual had received live vaccines within the previous 2 months, or had a history of vaccination within the last 30 days, they would be excluded.
• Having a history of alcohol dependence or drug addiction
• Pregnant or lactating female participants
• The situations that were determined by the investigator to be unsuitable for inclusion in the study

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine — Шанхай

Идентификаторы

NCT: NCT07195825 · BBM003-CLN001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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