Phase 2 Completed N=44 Treatment

Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease

Parkinson's Disease

Source: ClinicalTrials.gov NCT02462603 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

May 2022

Primary outcomePrimary: Number of Participants With Drug-Related Serious Adverse Events (SAEs) — 0 Participants

Summary

Open-label study with 30-day run-in phase and adaptive design component to include more participants if deemed appropriate by investigators.

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Drug-Related Serious Adverse Events (SAEs)	—	—
SECONDARY Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3	5.0; -0.1; 5.8; 0.1; 22.5; -0.6	—
SECONDARY Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3	15.7; -0.6	—
SECONDARY Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3	7.06; 0.19; 11.56; 0.21; 10.84; 0.10	—
SECONDARY Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3	1.2; 0; 1.1; 0; 1.2; 0.1	—
SECONDARY Montreal Cognitive Assessment (MoCA) Score	28.5; 23.4	—
SECONDARY Beck Depression Inventory (BDI) Score	3.6; 13.1	—
SECONDARY Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3	2.4; 0.1	—
SECONDARY Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3	8.637; -0.347	—
SECONDARY Maximum Observed Plasma Concentration (Cmax) of PTC589	3718.3; 2903.5	—
SECONDARY Level of Disease-Related Biomarker (Glutathione) in Plasma	1.54	—
SECONDARY Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)	0.10	—
SECONDARY Level of Disease-Related Biomarker (Glutathione) in Urine	0.0000061	—

Eligibility Criteria

Inclusion Criteria

Hoehn and Yahr stage ≤3.0
Ambulatory with or without assistance
Sexually active fertile participants and their partners must agree to use medically accepted methods of contraception (such as, hormonal methods, including oral, subcutaneous, and intrauterine; barrier methods, including male condom, female condom, or diaphragm with spermicidal gel) during the course of the study and for 3 months after the last dose of study treatment.
Willingness and ability to comply with study procedures
If on medications for Parkinson's disease drugs, then medication regimen must be stable for 60 days prior to enrollment
Abstention from use of other investigative or non-approved drugs for the duration of the trial

For Idiopathic Participants

A diagnosis of idiopathic Parkinson's disease confirmed by the presence of bradykinesia plus one or both of the following symptoms: rigidity or resting tremor; and with an abnormal DaTscan consistent with a dopaminergic deficit
Age 40 to 75 years
Within 5 years of diagnosis of Parkinson's disease

For Genetic Subtype Participants

A confirmed diagnosis of Parkinson's disease plus a genetic diagnosis consistent with Parkinson's disease, specifically PTEN-induced kinase 1 (PINK1), parkin, Leucine-rich repeat kinase 2 (LRRK2) or other mitochondrial genetic subtype
Age 21 to 75 years

Exclusion Criteria

Allergy to PTC-589 or other components of the PTC-589 tablet formulation
Use of antioxidant supplements, specifically vitamins E and C beyond the recommended daily allowance
Other Parkinsonian disorders
Montreal Cognitive Assessment (MoCA) score of 3 times upper limit of normal
Renal insufficiency as defined by creatinine >1.5 times normal
End stage cardiac failure
Participation within past 3 months and for duration of study in a trial of a device, drug, or other therapy for Parkinson's disease

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02462603). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.