Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01

Parkinson Disease Clinical Trial

Official title:

Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01, a Human Embryonic Stem Cell-Derived Midbrain Dopaminergic Neuronal Cell Therapy

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05897957
• Other study ID #: BRT-DA01-501
• Status: Enrolling by invitation
• Start date: May 16, 2023
• Est. completion date: September 2027

Study information

• Verified date: April 2024
• Source: BlueRock Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a multicenter study to evaluate the safety and clinical outcomes of BRT-DA01 in subjects with PD who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101.No investigational therapy will be administered in this study.

Description:

All subjects who received BRT-DA01 transplantation in Study MSK-DA01-101 (ie, parent study) will be asked to enroll in Study BRT-DA01-501. Subjects enrolled in this study are to be monitored for safety and clinical outcomes through 5 years post-BRT-DA01 transplantation. For individual participating subjects, the Baseline visit for the BRT-DA01-501 study will be defined as the last observation recorded in the parent study.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 12
• Est. completion date: September 2027
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years to 78 Years

Eligibility

Inclusion Criteria:

• Subject received BRT-DA01 in Study MSK-DA01-101 (ie, parent study) and will have completed or discontinued the parent study
• Subject must be willing and able to provide written informed consent for the BRT-DA0-501 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures

Exclusion Criteria:

• No exclusion criteria

Related Conditions & MeSH terms

• Parkinson Disease

Locations

Country	Name	City	State
Canada	Toronto Western Hospital	Toronto	Ontario
United States	Weill Cornell Medical College	New York	New York
United States	University of California, Irvine	Orange	California

Sponsors (1)

Lead Sponsor	Collaborator
BlueRock Therapeutics

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Adverse Events	AEs related to the cellular drug product	Baseline through 5 years post-transplantation
Other	Change in modified Schwab and England Activities of Daily Living (mSEADL) scale	Change in mSEADL	Baseline through 5 years post-transplantation
Other	Change in brain atrophy using MRI	Change in brain atrophy using MRI	Baseline through 5 years post-transplantation
Other	Subjects using alternative advanced treatment options in Parkin's Disease	Number of subjects using alternative advanced treatment options in PD (eg, CSAI, LCIG, DBS, focused ultrasound ablation)	Baseline through 5 years post-transplantation
Primary	Total SAEs and total number of subjects with at least 1 SAE	Total SAEs and total number of subjects with at least 1 SAE related to cellular drug product	Baseline through 5 years post-transplantation
Secondary	Change in striatal 18F-DOPA uptake using positron emission tomography (PET)	Change in striatal 18F-DOPA uptake using positron emission tomography (PET)	Baseline through 5 years post-transplantation
Secondary	Change in Hauser PD diary measurements	Change in Hauser PD diary measurements	Baseline through 5 years post-transplantation
Secondary	Change in the International Parkinson and Movement Disorder Society (MDS)-Unified Parkinson's Disease Rating Scale (UPDRS)	Change in the MDS-UPDRS	Baseline through 5 years post-transplantation
Secondary	Change in levodopa equivalent daily dose (LEDD)	Change in LEDD	Baseline through 5 years post-transplantation
Secondary	Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index	Change in PDQ-39	Baseline through 5 years post-transplantation
Secondary	Change in the Unified Dyskinesia Rating Scale (UDysRS)	Change in UDysRS	Baseline through 5 years post-transplantation