A Study to Determine if BHV-8000 is Effective, Safe and Tolerable as a Treatment for Adults Living With Early Parkinson's Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biohaven Therapeutics Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2025-12-22

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Biohaven Therapeutics, Ltd.

External identifiers

EU CT number

2025-521113-13-00

ClinicalTrials.gov

NCT06976268

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Dose response, Efficacy, Pharmacokinetic, Pharmacogenetic, Pharmacodynamic

To evaluate the efficacy of 2 dose levels of BHV-8000 compared to placebo by delaying the time to the first qualifying worsening event on the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part II.

Secondary objectives 5

1. To evaluate the efficacy of BHV-8000 compared to placebo on the change from baseline to Week 48 on the MDS-UPDRS Part III.
2. To evaluate the efficacy of BHV-8000 compared to placebo on the change from baseline to Week 48 on the Clinician Global Impression of Severity (CGI-S) score.
3. To evaluate the efficacy of BHV-8000 compared to placebo on the change from baseline to Week 48 on the dopamine transporter single photon emission computed tomography (DaT-SPECT) scan Striatal Binding Ratio (SBR) in the putamen ipsilateral to the clinically most affected side.
4. To evaluate the efficacy of BHV-8000 compared to placebo on the change from baseline to Week 48 on the Parkinson’s Disease Composite Score – Function (PARCOMS-Function).
5. To assess the safety and tolerability of BHV-8000.

Conditions and MedDRA coding

Early Parkinson’s Disease

Study design 4 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Male or female participants 40 to 85 years of age, inclusive, at the time of informed consent
2. Meet the diagnostic criteria for "Probable PD" as assessed on the Movement Disorder Society(MDS) Clinical Diagnostic Criteria for PD as assessed by the Investigator.
3. Have a clinician-documented diagnosis of idiopathic PD with an onset within 2 years of the Screening Visit

Exclusion criteria 5

1. Medical history indicating a Parkinsonian syndrome other than idiopathic PD, including, but not limited to, progressive supranuclear gaze palsy, multiple system atrophy, drug-induced Parkinsonism, essential tremor, or primary dystonia.
2. Diagnosis of clinically significant central nervous system (CNS) disease other than PD.
3. Participants who are current smokers (defined as smoking [in any form, e.g., tobacco smoke, electronic cigarettes, etc.] ).
4. Treatment with PD medication(s)
5. Any other condition(s) that may compromise participant safety, interfere with study conduct, or jeopardize the potential proper interpretation of study results, in the opinion of the investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Time to first qualifying worsening event on Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II [Time Frame: Up to 48 Weeks]. To evaluate the efficacy of BHV-8000 compared to placebo. This objective is measured by assessing the time to prespecified worsening on MDS-UPDRS Part II (motor experiences of daily living per self-administered questionnaire). MDS-UPDRS Part II is a 52-point scale with a higher total score representing more severe disability.

Secondary endpoints 6

1. Change in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III from Baseline to Week 48 [Time Frame: Baseline to Week 48]. This objective is measured by assessing the change in MDS-UPDRS Part III (motor examination conducted by rater). MDS-UPDRS Part III is a 132-point scale with a higher total score representing a greater degree of motor impairment.
2. Change in Clinical Global Impression of Severity (CGI-S) from Baseline to Week 48 [Time Frame: Baseline to Week 48]. To evaluate the efficacy of BHV-8000 compared to placebo. This objective is measured by assessing the change in severity of a participant's illness as determined by the managing clinician. The CGI-S is a 7-point scale (1 - 7) with 7 representing the most extremely ill participants.
3. Change in DaT-SPECT scan from Baseline to Week 48 [Time Frame: Baseline to Week 48]. To evaluate the efficacy of BHV-8000 compared to placebo. This objective is measured by change in DaT-SPECT Striatal Binding Ratio (SBR) in the putamen (assessing the activity of the dopamine transporters). Reduced uptake of the radiotracer is indicative of a decreased number of dopamine-secreting cells and suggestive of disease progression.
4. Change in Parkinson's Disease Composite Score - Function (PARCOMS-Function) from Baseline to Week 48 [Time Frame: Baseline to Week 48].To compare the efficacy of BHV-8000 compared to placebo.This objective is measured by changes in the Parkinson's Disease Composite Score-Function (PARCOMS-Function) score.The PARCOMS-Function is a composite of select items taken from the MDS-UPDRS Part II and the PDQ-39. The PARCOMS-Function is a 100-point scale with higher scores representing greater disfunction
5. Number of Participants with Deaths, Serious AEs (SAEs), AEs Leading to Study Drug Discontinuation, and moderate or severe AEs [Time Frame: Baseline to Week 48]. To assess the safety and tolerability of BHV-8000. This objective will be measured by assessing the number of unique participants with deaths, SAEs, AEs leading to discontinuation, and moderate and severe AEs.
6. Number of participants with clinically significant laboratory abnormalities [Time Frame: Baseline to Week 48]. To assess the safety and tolerability of BHV-8000. This objective will be measured by assessing the number of unique participants with treatment-emergent Grade 3 and 4 laboratory abnormalities.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Auxiliary 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biohaven Therapeutics Ltd.

Sponsor organisation

Biohaven Therapeutics Ltd.

Address

215 Church Street

City

New Haven

Postcode

06510-1803

Country

United States

Scientific contact point

Organisation

Biohaven Therapeutics Ltd.

Contact name

Research and Development Medical Director

Public contact point

Organisation

Biohaven Therapeutics Ltd.

Contact name

Research and Development Medical Director

Third parties 19

Locations

10 EU/EEA countries · 101 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 166 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications