BTRX-246040 Study in Participants With Parkinson's Disease With Motor Fluctuations

NCT03608371 | Completed Phase 2 Results FDA Drug

• 1 other identifier: NEP-PD-201
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 4

Brief Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetics and efficacy of BTRX-246040 in participants with Parkinson's Disease who have motor fluctuations and predictable early morning off periods.

Conditions

Parkinson Disease; Motor Disorder

Outcome Measures

Primary Outcomes (1)

• Maximal Change in UPDRS Part III From Predose to Postdose on Day 1

  UPDRS = Unified Parkinson's Disease Rating Scale. The Unified Parkinson's Disease Rating Scale (UPDRS) Part III is a 14-item rating scale, which consists of the motor examination. Each item is scored from 0 (Normal) to 4 (Most severe). The score ranges from 0-56.

  From pre-dose to 8 hours post-dose on Day 1

Secondary Outcomes (5)

• Duration of ON Time on Day 1

  From dose to 8 hours post-dose on Day 1

• Percentage of Participants Who Turned ON on Day 1

  From dose to 8 hours post-dose on Day 1

• Time to ON on Day 1

  From dose to 8 hours post-dose on Day 1

• Area Under the Curve for UPDRS Part III During the 8 Hours of Assessment on Day 1

  From pre-dose to 8 hours post-dose on Day 1

• Change From Pre-dose Dyskinesia Rating (From the UPDRS Part III Motor Response and Dyskinesia Assessment)

  From pre-dose to 8 hours post-dose on Day 1

Study Arms (4)

BTRX-246040 Cohort 1

EXPERIMENTAL

BTRX-246040 40 mg administered orally

Drug: BTRX-246040

BTRX-246040 Cohort 2

EXPERIMENTAL

BTRX-246040 80 mg administered orally

Drug: BTRX-246040

BTRX-246040 Cohort 3

EXPERIMENTAL

BTRX-246040 120 mg administered orally

Drug: BTRX-246040

Placebo Cohorts 1-3

PLACEBO COMPARATOR

Placebo is administered orally at the same number of capsules as active drug in each Cohort. Placebo capsules consist of inactive ingredients and look identical to BTRX-246040.

Drug: Placebo

Interventions

BTRX-246040 DRUG

oral capsule

BTRX-246040 Cohort 1; BTRX-246040 Cohort 2; BTRX-246040 Cohort 3

Placebo DRUG

oral capsule matching BTRX-246040 capsule

Placebo Cohorts 1-3

Eligibility Criteria

• Age: 30 Years - 76 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed with Parkinson's disease (PD), consistent with the United Kingdom PD Society Brain Bank Criteria for the Diagnosis of PD
• Men or women ≥ 30 years old and ≤ 76 years old
• Female participants must be either surgically sterilized or 2 years post menopausal at screening
• Modified Hoehn and Yahr Staging ≤ 3 in the ON state
• Montreal Cognitive Assessment (MoCA) Score ≥ 26
• Currently has a clear and decisive response to levodopa, and receiving a stable dose of levodopa (at least 4 doses per day of levodopa or ≥ 3 doses per day of RytaryTM (carbidopa and levodopa) extended-release capsules for at least 4 weeks prior to screening)
• Experiencing motor fluctuations during waking hours with at least 2 hours of OFF periods each day, including predictable early morning OFF periods, based on participant assessment
• Able to participate in the study in the practically defined OFF state
• All anti-parkinsonian medications maintained at a stable dose for at least 4 weeks prior to the initial Screening Visit with the exception of monoamine oxidase B (MAO-B) inhibitors, which must be maintained at a stable level for at least 8 weeks prior to the screening visit
• Approved by a central Enrollment Authorization Committee as meeting entry criteria and being a suitable candidate for the study
• Male participants agree to use a reliable method of birth control during the study and for at least 90 days following the last dose of BTRX-246040 or placebo
• Informed and given ample time and opportunity to think about his/her participation in this study and has given his/her written informed consent on an Institutional Review Board (IRB) or Independent Ethics Committee (IEC) approved consent form
• Judged to be reliable and able to keep all appointments for clinic visits, tests, and procedures, including venipuncture, and examinations required by the protocol

You may not qualify if:

• Diagnosis of secondary or an atypical Parkinsonian syndrome
• Severe disabling dyskinesia
• Clinically significant psychosis or hallucinations or history of psychosis in past 6 months
• History of previous neurosurgery for PD
• Currently or previously on Duopa/Duodopa
• Currently taking apomorphine
• Has a diagnosis or history of a substance related disorder per Diagnostic and Statistical Manual of Mental Disorders V edition (DSM-V) criteria (including alcohol but excluding nicotine and caffeine), during the 12 months prior to the Screening Visit
• Medical or recreational use of marijuana in the 6 months prior to the Screening Visit
• Has tested positive at the Screening Visit for drugs of abuse (opiates, cannabinoids, methadone, cocaine, and amphetamines \[including ecstasy\])
• Active suicidal ideation within 1 year prior to the Screening Visit as evidenced by answering "yes" to Questions 4 or 5 on the suicidal ideation portion of the Columbia- Suicide Severity Rating Scale (C-SSRS) or attempted suicide within the last 1 year
• Current major depressive episode or a Beck Depression Inventory-II (BDI-II) score above 19. Participants receiving treatment for depression with antidepressants may be enrolled if they have been on a stable daily dose for at least 8 weeks before the Screening Visit and are clinically stable in the opinion of the Principal Investigator
• Currently or within 8 weeks of screening receiving bupropion
• Exposure to neuroleptics (antipsychotic drugs) for more than 1 month within the past 2 years, or any exposure within the past year
• Any malignancy in the 5 years prior to randomization (excluding successfully treated basal cell carcinoma of the skin or cervical carcinoma in situ)
• Current or previous diagnosis of malignant melanoma or the presence of any suspicious skin lesion based on physical exam findings

Sponsors & Collaborators

• BlackThorn Therapeutics, Inc.: lead

Study Sites (4)

BlackThorn Investigator Site

Hallandale, Florida, 33009, United States

Location

BlackThorn Investigator Site

Farmington Hills, Michigan, 48334, United States

Location

BlackThorn Investigator Site

Durham, North Carolina, 27705, United States

Location

BlackThorn Investigator Site

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Parkinson Disease; Motor Disorders

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Mental Disorders

Results Point of Contact

• Title: Study Contact
• Organization: Neumora Therapeutics

clinicaltrials@neumoratx.com

8577600900

Study Officials

• Jane M Tiller, M.D.

  BlackThorn Therapeutics, Inc. (Sponsor)

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: double-blind study
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study consists of 3 sequential, single ascending dose cohorts of 8 participants each with a 6:2 randomization to BTRX-246040 or placebo

Study Record Dates

• First Submitted: May 9, 2018
• First Posted: July 31, 2018
• Study Start: August 31, 2018
• Primary Completion: April 16, 2019
• Study Completion: April 23, 2019
• Last Updated: June 24, 2025
• Results First Posted: June 24, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)