A Phase 2 Study of NEU-411 in Companion Diagnostic-Positive Participants with Early Parkinson’s Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Neuron23 Inc.

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)

2025-05-19

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Neuron23, Inc.

External identifiers

EU CT number

2024-515448-22-00

ClinicalTrials.gov

NCT06680830

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy, Others, Pharmacodynamic

To evaluate the safety and primary efficacy of NEU-411 in participants with LRRK2-driven Parkinson’s disease

Secondary objectives 1

1. To evaluate the secondary safety and efficacy of NEU-411 in participants with LRRK2-driven Parkinson's disease

Conditions and MedDRA coding

Parkinson

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Aged 40-80 years at time of screening, inclusive
2. Diagnosis of clinically established or clinically probable Parkinson's disease
3. LRRK2-driven PD using the investigational companion diagnostic genetic test (CDx)
4. Modified Hoehn and Yahr (mH&Y) of 1 to 2.5
5. Additional inclusion criteria are outlined in the full study protocol.

Exclusion criteria 4

1. Secondary or atypical parkinsonian syndromes
2. Uncontrolled diabetes mellitus with hemoglobin A1c (HbA1c) >8%
3. Other significant medical conditions (as determined by medical history, examination, or clinical investigations at screening)
4. Additional exclusion criteria are outlined in the full study protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Change from baseline in the Roche digital biomarker score using the Roche Parkinson's Disease application (v3.0) compared to placebo Time Frame: From enrollment to the end of treatment at 52 weeks
2. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo Time Frame: From enrollment to the end of study at 54 weeks

Secondary endpoints 1

1. Change from baseline to Week 52 in motor function/nonmotor as measured by Movement Disorder Society’s Unified Parkinson’s Disease Rating Scale (MDS-UPDRS)

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Neuron23 Inc.

Sponsor organisation

Neuron23 Inc.

Address

343 Oyster Point Boulevard Suite 120

City

South San Francisco

Postcode

94080-1913

Country

United States

Scientific contact point

Organisation

Neuron23 Inc.

Contact name

Fatta Nahab, MD FAAN FANA

Public contact point

Organisation

Neuron23 Inc.

Contact name

Tom DeMelfi, MS

Third parties 1

Sponsor responsibilities

Article 77 compliance

Neuron23 Inc.

Contact point sponsor

Neuron23 Inc.

Article 77 implementation

Neuron23 Inc.

Locations

3 EU/EEA countries · 21 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 108 files

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

Application history

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