Health Trials

Prevent Cognitive Decline in GBA-associated Parkinson's Disease

ConditionParkinson's Disease
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age35-80
SponsorUniversity Hospital Tuebingen

About this trial

This is a proof-of-concept trial to investigate the efficacy of prasinezumab to slow or prevent cognitive decline in people with Parkinson's disease carrying a severe mutation in the GBA (glucocerebrosidase) gene. The duration of the intervention per patient will be 104 weeks with monthly infusions. The investigators plan to enroll 120 participants (60 participants per treatment arm). This study will be conducted across Europe in the following countries: France, Germany, Italy, Luxembourg, Spain, Sweden, UK.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of PD according to MDS-Criteria.

Known heterozygous severe GBA mutation (based on PD-related pathogenicity).

MoCA ≥ 21.

HY in dopaminergic ON ≤3.

Disqualifiers

Known pathogenic mutation carriers of the following familial PD genes: PRKN, PINK1, DJ1, LRRK2.

Medical history indicating a Parkinsonian syndrome other than sporadic PD (progressive supranuclear palsy, multiple system atrophy, drug-induced parkinsonism, essential tremor, primary dystonia).

A diagnosis of a significant CNS disease other than PD.

Previous, current or planned (within next 2 years) treatment with Deep Brain Stimulation (DBS) or ablation with high-intensity focused ultrasound or planned treatment with these within the next 2 years.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Prasinezumab

    Drug

    Prasinezumab 1500 mg monthly infusion

  • Sodium Chloride

    Drug

    Saline infusion (0,9 % sodium chloride) monthly infusion

Treatment groups

120 Participants
are divided into 2 treatment groups
Group A: PrasinezumabExperimental treatment 1 intervention
Group B: Sodium chloride 0,9% infusionPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Parkinson's Disease Cognitive Composite Score (PDCCS)

To assess the efficacy of prasinezumab compared with placebo on cognitive function at week 104 measured by the Parkinson's Disease Cognitive Composite Score (PDCCS). Composite score that includes the following cognitive tests: * LNS (Attention) * Derived-Sem. Fluency-Animal T-Score (Executive function) * Derived-Delayed Recall T-Score (Memory) * MoCA (Global cognition) Per single Test: Group Mean and Standard Deviation (SD) at Baseline = reference ZBaseline = (xBaseline - Group MeanBaseline) / SDBaseline ZFollow-up = (xFollow-up - Group MeanBaseline) / SDBaseline Mean of Z-Scores of relevant Tests per Patient per Visit Min-Max: n.a. (higher values mean a better outcome)

Time frame
week 104

Secondary outcomes

1

Cognitive function (MoCA_z)

Cognitive function measured by MoCA\_z (Montreal Cognitive Assessment demographically-corrected z-value) Min-Max: 0 - 30 (higher values mean a better outcome)

Time frame
week 104
2

PD-MCI

Percentage of participants with diagnosis of PD-MCI defined by MDS Level II criteria

Time frame
week 104
3

PDD

Percentage of participants with diagnosis of PDD

Time frame
week 104
4

MDS-UPDRS I-IV

International Parkinson and Movement Disorder Society (MDS) Unified Parkinson's Disease Rating Scale (UPDRS) part I-IV Min-Max: 0 - 272 (lower values mean a better outcome)

Time frame
week 104

Other outcomes

1

Exploratory: cognitive function (MoCA_z)

Cognitive function measured by PDCCS in the prasinezumab and placebo group separately compared to MoCA\_z values (Montreal Cognitive Assessment demographically-corrected z-value) PDCCS is a composite score (see primary endpoint for details)

Time frame
week 104
2

Exploratory: Cognitive function (MoCA ≥ 26)

Cognitive function measured by PDCCS in the prasinezumab versus placebo group in participants who were cognitively normal at baseline as defined by MoCA ≥ 26. PDCCS is a composite score (see primary endpoint for details)

Time frame
week 104
3

Exploratory: Cognitive function (PD-MCI)

Cognitive function measured by PDCCS in the prasinezumab versus placebo group in participants who had PD-MCI at baseline as defined by MoCA ≤ 25. PDCCS is a composite score (see primary endpoint for details)

Time frame
week 104
4

Exploratory: IADL

Cognitive-driven Instrumental Activities of Daily Living (IADL) as measured by the FAQ (Functional Activities Questionnaire) cognitive score provided by the patient and caregiver separately. Min-Max: 0-30 (lower values mean a better outcome)

Time frame
week 104

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University Hospital Tuebingen

Lead sponsor

Source ClinicalTrials.gov

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