A Study on the Safety and Tolerability of RGL-193 in Patients With Advanced Parkinson's Disease

NCT06195124 | Recruiting Early Phase 1

• 1 other identifier: RGL-193-IIT
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

A safety study in patients with Parkinson's disease.

Conditions

Advanced Parkinson's Disease

Outcome Measures

Primary Outcomes (6)

• the number of patients with adverse events

  26 weeks after administration

• the number of patients with clinically significant change from baseline in vital signs value

  26 weeks after administration

• the number of patients with clinically significant change in physical examination

  26 weeks after administration

• the number of patients with clinically significant change from baseline in laboratory examination

  26 weeks after administration

• the number of patients with clinically significant change from baseline in 12-ECG values

  26 weeks after administration

• the number of patients with clinically significant change in brain MRI

  26 weeks after administration

Study Arms (2)

RGL-193（low-dose）Treatment group

EXPERIMENTAL

Each side of the putamen received 150 μL of RGL-193, with a unilateral dose of 1.5×10\^11 VG and a bilateral dose of 3.0×10\^11 VG.

Drug: RGL-193（low-dose）

RGL-193（high-dose）Treatment group

EXPERIMENTAL

Each side of the putamen received 200 μL of RGL-193, with a unilateral dose of 5.0×10\^11 VG and a bilateral dose of 1.0×10\^12 VG.

Drug: RGL-193（high-dose）

Interventions

RGL-193（low-dose） DRUG

Each side of the putamen received 150 μL of RGL-193, with a unilateral dose of 1.5×10\^11 VG and a bilateral dose of 3.0×10\^11 VG.

RGL-193（low-dose）Treatment group

RGL-193（high-dose） DRUG

Each side of the putamen received 200 μL of RGL-193, with a unilateral dose of 5.0×10\^11 VG and a bilateral dose of 1.0×10\^12 VG.

RGL-193（high-dose）Treatment group

Eligibility Criteria

• Age: 45 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The patients who voluntarily participate in the study, fully understand the content, process, and possible adverse events of the trial, can complete the study according to the requirements of the study protocol, and sign the informed consent form.
• The patients with clinically diagnosed Parkinson's disease (as per PD diagnostic criteria, China, 2016) and the course of disease for at least 5 years at the time of screening.
• The patient must be 45-70 years old (inclusive) on the day signing the informed consent form.
• The patients who are receiving Levodopa treatment with clear response to Levodopa treatment.
• The patients showing stable clinical symptoms within 1 month before baseline, with drug dosage remain the same.
• The patients with the modified Hoehn \& Yahr stage ≥ 3.
• The patients with the score of MDS-UPDRS part III ≥ 30 in the off period.
• There must be fluctuation of motor symptoms, which is defined as at least cumulatively 3-h off time in the awake time every day (confirmed by PD diary for 3 consecutive days).
• The results of 18F-DOPA-PET examination shall be consistent with PD phenotype.
• The total score of HAMD (24-item) in the screening period shall be ≤ 20 points.
• The patients' hematological indicators and vital signs at baseline must be within the normal range, unless the values beyond the normal range have no clinical significance for whether the subjects are suitable to receive treatment with the investigational product as considered by the investigator.
• The body weight at screening and baseline shall be 45-100 kg (inclusive), and the body mass index (BMI) shall be 18-32 kg/m2 (inclusive).
• The subjects (including partners) shall have no fertility planning and be willing to take effective contraceptive measures during the study period (2 weeks before drug administration for female subjects) and within 6 months after administration.
• The subjects shall have stable caregivers.

You may not qualify if:

• The subjects with secondary or atypical Parkinson's syndrome caused by drugs, metabolic disorders, or other reasons.
• The subjects who have known or suspected allergies or serious adverse reactions to the ingredients of the study preparation, or those have an allergic constitution;
• For the population with an education background of junior high school or above, the MMSE score during the screening period shall be \< 23; for the population with an education background below junior high school, the MMSE score shall be \< 20 during the screening period.
• The subjects with alcoholism, acute alcohol intoxication, or drug abuse or drug dependence;
• The subjects with a previous history of PD-related brain surgery (deep brain stimulation, thalamus destruction, etc.).
• The subjects who have previously received any biological agents (including marketed drugs and clinical trial drugs) for PD treatment.
• The subjects who have a history of deep venous thrombosis of lower limbs.
• The subjects suffering from massive blood loss (more than 500 mL) due to surgery or trauma within 3 months before screening.
• Antiplatelet and anticoagulant drugs were used within 3 months before drug administration (except aspirin (≤325mg per day));
• The subjects with coagulation disorder, which is defined as PT \> 1.2 times the upper limit of normal; a re-examination is accepted in case of a single abnormality for confirmation.
• The subjects with obvious abnormalities in brain MRI examination, including but not limited to: more than 10 microhemorrhage foci; angiogenic edema; serious white matter demyelination (Fazekas score ≥ 3); brain contusion; encephalomalacia; aneurysm; vascular malformation, and space-occupying lesions.
• The subjects with other major medical or nervous system diseases that may pose unacceptable risks of surgery or anesthesia, or those with any surgical contraindications.
• The subjects unable to cooperate with the surgery or radiologic examination (DOPA PET or MRI scanning).
• The subjects with systolic blood pressure \> 160 mmHg and diastolic blood pressure \> 100 mmHg in supine position in the screening period or baseline period; a re-examination is accepted in case of a single abnormality for confirmation.
• In the screening period or baseline period, the continuous resting heart rate is \< 50 bpm or \> 100 bpm by repeated testing twice within 30 min.

Sponsors & Collaborators

• Second Affiliated Hospital of Soochow University: lead
• Shanghai Regenelead Therapies Co., Ltd.: collaborator

Study Sites (1)

Department of Neurology, Second Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215004, China

RECRUITING

Study Officials

• Chun-Feng Liu, MD

  Second Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chun-Feng Liu, MD

CONTACT

+8613606210609; liuchunfeng@suda.edu.cn

Xiao-Yu Cheng, MD

CONTACT

+8651267783307; chengxiaoyu2007@126.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician

Study Record Dates

• First Submitted: October 14, 2023
• First Posted: January 8, 2024
• Study Start: December 1, 2023
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: March 15, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)