An Observational Study of Patients With Moderate Parkinson's Disease (TRANSCEND 1)

The main purpose of this study is to follow and observe a group of people living with Parkinson's disease to see how study participation affects their signs and symptoms in the months after starting in the study. While taking part in this study participants will take their usual medication as prescribed. However, the study doctor may recommend adjustments to their medication to provide a better treatment of their Parkinson's disease. Participation will last from 3 up to 24 months. During visits to the clinic, the study doctor or study nurse will evaluate signs and symptoms of Parkinson's disease using several different assessments. At a minimum of 2 visits participants will be asked to undergo 'off'-assessments.

Study Overview

• Status: Enrolling by invitation
• Conditions: Parkinson Disease
• Intervention / Treatment: Other: No treatment given

• Study Type: Observational
• Enrollment (Estimated): 96

Contacts and Locations

Study Locations

• Japan
  • Shinjuku-ku, Tokyo, Japan, 160-8582
    • Keio University Hospital
• Sweden
  • Lund, Sweden, 222 42
    • Neurologimottagningen Lund
• United Kingdom
  • Cambridge, United Kingdom, CB2 0PY
    • Clinical Neuroscience
• United States
  • New York
    • New York, New York, United States, 10016
      • NYU Langone Health
    • New York, New York, United States, 10021
      • NY Presbyt Hosp-W Cornell Med

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years to 68 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with Parkinson's disease will be included. The participants will not receive any investigational treatment in this study.

Description

Inclusion Criteria:

• Male or female, aged 50-68 years (both inclusive) at the time of signing informed consent.
• Diagnosed with Parkinson's disease using the Movement Disorder Society (MDS) criteria (fulfilling the definition of clinically probable Parkinson's disease).
• Moderate Parkinson's disease, e.g. as defined by i) modified Hoehn and Yahr stage 2-3 in OFF state and ii) MDS-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III total score greater than or equal to (≥) 30 in OFF state, both as judged by the investigator.
• Patient has symptoms that are not adequately controlled by existing oral anti-Parkinson's disease medications, i.e., having both ON and OFF periods, as judged by the investigator.
• Parkinson's disease duration greater than (>) 5 years and lesser than or equal to (≤) 12 years from diagnosis at the time of signing informed consent.
• Potential candidate for a future cell therapy transplantation, as judged by the investigator.
• Life expectancy >7 years as judged by the investigator.

Exclusion Criteria:

• Any atypical or secondary Parkinson's disease, or non-Parkinson's disease-suspected cause(s) of, or contributors to, parkinsonism, as judged by the investigator.
• Significant drug-induced dyskinesias as judged by the investigator, e.g., as defined by a score of > 2 in the Abnormal Involuntary Movement Scale (AIMS), in any body part in the ON state.
• Tremor-dominant Parkinson's disease, as judged by the investigator.
• Cognitive impairment predictive of dementia (including Parkinson's disease dementia) or other cognitive dysfunction as judged by the investigator, e.g., as defined by Montreal Cognitive Assessment (MoCA) score ≤ 24, or evidence of major neurocognitive disorder according to the Diagnostic and Statistical Manual of Mental Disorders V (DSM-V) criteria.
• Major medical or psychiatric disorder (e.g., depression (Montgomery-Asberg Depression Rating Scale (MADRS) >20) or psychosis), or other disease making the patient unsuited for participation in the study, as judged by the investigator.
• Treatment for dystonia or spasticity within 3 months of screening (Botox for a focal dystonia is allowed).
• Substantial neurological symptoms not accounted for by Parkinson's disease, including active seizure disorder, as judged by the investigator.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Participants with Parkinson's disease; Participants will not receive any investigational treatment in this study. The participants will be treated with anti-Parkinson's disease medication according to local standard of care.	Other: No treatment given; Participants will not receive any investigational treatment in this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III (motor score) in OFF state	Measured as score. MDS-UPDRS consists of 4 parts - has 65 items/questions in total. Part III assesses the motor function of the patient at the time of the visit conducted in both the OFF and ON state. Each item will receive a score ranging from 0 to 4, where 0 represents the absence of impairment and 4 represents the highest degree of impairment.	From baseline (week 0) to end of study (13 to 104 weeks from baseline)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in MDS-UPDRS Part III (motor score) in OFF state	Measured as score. MDS-UPDRS consists of 4 parts - has 65 items/questions in total. Part III assesses the motor function of the patient at the time of the visit conducted in both the OFF and ON state. Each item will receive a score ranging from 0 to 4, where 0 represents the absence of impairment and 4 represents the highest degree of impairment.	From last medication adjustment (between week 0 and week 104) to end of study (13 to 104 weeks from baseline)
Is the patient interested in being considered for participation in a cell therapy transplantation study (yes/no)	Measured as count of participants	At end of study (13 to 104 weeks from baseline)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Transparency dept. 2834, Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): April 5, 2023
• Primary Completion (Estimated): October 22, 2025
• Study Completion (Estimated): October 22, 2025

Study Registration Dates

• First Submitted: March 9, 2023
• First Submitted That Met QC Criteria: March 9, 2023
• First Posted (Actual): March 22, 2023

Study Record Updates

• Last Update Posted (Estimated): January 18, 2024
• Last Update Submitted That Met QC Criteria: January 17, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: NN9001-4704; U1111-1254-1741 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com