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

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

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05897957

Enrollment

Registered

2023-06-12

Start date

2023-05-16

Completion date

2027-09-30

Last updated

2024-04-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Parkinson Disease

Keywords

Parkinson Disease, Parkinsonian Disorders, Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Synucleinopathies, Neurodegenerative Diseases

Brief 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.

Detailed 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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

50 Years to 78 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Total SAEs and total number of subjects with at least 1 SAE	Baseline through 5 years post-transplantation	Total SAEs and total number of subjects with at least 1 SAE related to cellular drug product

Secondary

Measure	Time frame	Description
Change in Hauser PD diary measurements	Baseline through 5 years post-transplantation	Change in Hauser PD diary measurements
Change in the International Parkinson and Movement Disorder Society (MDS)-Unified Parkinson's Disease Rating Scale (UPDRS)	Baseline through 5 years post-transplantation	Change in the MDS-UPDRS
Change in levodopa equivalent daily dose (LEDD)	Baseline through 5 years post-transplantation	Change in LEDD
Change in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index	Baseline through 5 years post-transplantation	Change in PDQ-39
Change in the Unified Dyskinesia Rating Scale (UDysRS)	Baseline through 5 years post-transplantation	Change in UDysRS
Change in striatal 18F-DOPA uptake using positron emission tomography (PET)	Baseline through 5 years post-transplantation	Change in striatal 18F-DOPA uptake using positron emission tomography (PET)

Other

Measure	Time frame	Description
Change in brain atrophy using MRI	Baseline through 5 years post-transplantation	Change in brain atrophy using MRI
Subjects using alternative advanced treatment options in Parkin's Disease	Baseline through 5 years post-transplantation	Number of subjects using alternative advanced treatment options in PD (eg, CSAI, LCIG, DBS, focused ultrasound ablation)
Change in modified Schwab and England Activities of Daily Living (mSEADL) scale	Baseline through 5 years post-transplantation	Change in mSEADL
Adverse Events	Baseline through 5 years post-transplantation	AEs related to the cellular drug product

Countries

Canada, United States

Outcome results