AzTrials
AzTrials

Parkinson DiseasePhase 2NCT03608371

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

A Phase 2 study testing BTRX-246040 in people with Parkinson Disease and Motor Disorder, run by BlackThorn Therapeutics, Inc. at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 30 to 76 years
Conditions the study is for
Parkinson Disease, Motor Disorder
What is being tested?
BTRX-246040
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
24 participants
Where
4 sitesUnited States
Study dates
31 Aug 2018to 23 Apr 2019
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 30 to 76 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
Show all 13Show fewer
  • 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

Who cannot join

  • 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
Show all 33Show fewer
  • 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
  • Any other clinically significant medical condition or circumstance prior to randomization that, in the opinion of the Investigator, could affect participant safety, preclude evaluation of response, interfere with the ability to comply with study procedures, or prohibit completion of the study (e.g., uncontrolled diabetes mellitus, renal or hepatic impairment, coronary artery disease, evidence of significant active cardiac, respiratory, or hematologic disease, cancer with < 5 year remission (excluding successfully treated basal cell carcinoma of the skin or cervical carcinoma in situ), chronic pain, fibromyalgia or gastric bypass)
  • Prior seizures (other than childhood febrile seizure) or other condition that would place the participant at increased risk of seizures or is taking anticonvulsants for seizure control
  • History of serious head injury (e.g., skull fracture, cerebral contusion, or trauma resulting in prolonged unconsciousness), intracranial neoplasm or hemorrhage
  • Orthostatic hypotension that is symptomatic or requires medication
  • Participation in another study of an investigational medicinal product (IMP) or medical device currently or in the last 30 days or within 5 half-lives of the IMP (whichever is longer) prior to Screening
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels ≥ 2x upper limit of normal (ULN) or glomerular filtration rate ≤ 60 mL/min at Visit 1 (screening)
  • Nephritic syndrome, end-stage renal disease (and using renal replacement therapy such as hemodialysis or peritoneal dialysis), or a serum creatinine ≥ 2 mg/dL (≥ 172 μmol/L) at the Screening Visit
  • Any other clinically significant abnormalities (i.e., laboratory deviations requiring acute medical intervention or further medical evaluation) in laboratory results at screening including clinical chemistries, hematology, and urinalysis, that, in the judgment of the Investigator, should preclude a participant's participation at study entry
  • Electrocardiogram (ECG) abnormalities at Visit 1 (screening) or Visit 2 that, in the judgment of the Investigator, are clinically significant related to the participant's participation
  • Using the following concomitant medications (contact the Sponsor-designated medical monitor to determine eligibility when in doubt):
  • proton pump inhibitors within 5 half-lives of Screening
  • fluoxetine and irreversible monoamine oxidase inhibitors within 4 weeks of Screening
  • Currently taking or have taken, within 5 half-lives of Screening, any medications or supplements that are strong inhibitors or inducers of CYP3A4
  • A known hypersensitivity to gelatin capsules
  • Investigator site personnel directly affiliated with this study, and/or their immediate families. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted
  • Employees of the Sponsor or of any third-party organizations involved in study who require exclusion of their employees
  • Have participated in a clinical trial or any other type of medical research judged by the Investigator to be scientifically or medically incompatible with this study within 30 days prior to Visit 1 (screening)
  • Previous completion or withdrawal from this study or any other study investigating BTRX-246040 (previously called LY2940094)

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • BlackThorn Investigator Site

    Dallas, Texas, 75390

  • BlackThorn Investigator Site

    Durham, North Carolina, 27705

  • BlackThorn Investigator Site

    Farmington Hills, Michigan, 48334

  • BlackThorn Investigator Site

    Hallandale, Florida, 33009

Other trials in United States

About this study

Official title: Phase 2A, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Efficacy of BTRX-246040 in Parkinson's Disease Subjects With Motor Fluctuations

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.

Read the detailed description

Study treatment is 1 day and total duration of the study is up to 36 days, including an approximate 28-day screening period. The study consists of 3 sequential, ascending dose cohorts of 8 participants each with a 6:2 randomization to BTRX-246040 or placebo. The planned dosing for each cohort is 40, 80, and 120 mg. After enrollment of the first cohort is completed, doses for subsequent cohorts may be modified based on review of the available data (safety, tolerability, efficacy, and pharmacokinetics) by an unblinded Dosing Review Committee (DRC). A similar review and determination of dosing for the subsequent cohort is to be performed after completion of each cohort and based on all data available from previous cohorts.

Participants who meet entry criteria assessed at the screening visit (up to 28 days prior to Day 1) present to the clinic on the morning of Day 1 (treatment day) in the practically defined OFF state (having withheld anti-parkinsonian medications after 10:00 PM the evening prior). Participants are to be dosed with study drug and remain on site for an 8-hour observation period with Unified Parkinson's Disease Rating Scale (UPDRS) Part III motor response, dyskinesia rating and ON/OFF status assessed pre-dose, every 30 minutes for 4 hours post-dose, and then hourly for 4 additional post-dose hours (i.e., 8 hours total post-dose) prior to being discharged. Blood for pharmacokinetics are to be collected 6 times at scheduled intervals within the 8-hour observation period. A follow-up safety visit is scheduled 7 days later.

Being tested
BTRX-246040, Placebo
Medical subject headings
Parkinson Disease, Motor Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BTRX-246040Drug

    oral capsule

  • PlaceboDrug

    oral capsule matching BTRX-246040 capsule

Study groups in NCT03608371
Study groupRoleWhat they receive
BTRX-246040 Cohort 1ExperimentalBTRX-246040 40 mg administered orally
Receives: Drug: BTRX-246040
BTRX-246040 Cohort 2ExperimentalBTRX-246040 80 mg administered orally
Receives: Drug: BTRX-246040
BTRX-246040 Cohort 3ExperimentalBTRX-246040 120 mg administered orally
Receives: Drug: BTRX-246040
Placebo Cohorts 1-3Placebo comparatorPlacebo 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.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

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

Secondary outcomes (5)

  1. Duration of ON Time on Day 1

    ON is the typical functional state when patients are receiving medication and have a good response.

    OFF is the typical functional state when patients have a poor response in spite of taking medications.

    Measured: From dose to 8 hours post-dose on Day 1

  2. Percentage of Participants Who Turned ON on Day 1

    ON is the typical functional state when patients are receiving medication and have a good response.

    OFF is the typical functional state when patients have a poor response in spite of taking medications.

    Measured: From dose to 8 hours post-dose on Day 1

  3. Time to ON on Day 1

    ON is the typical functional state when patients are receiving medication and have a good response.

    OFF is the typical functional state when patients have a poor response in spite of taking medications.

    Measured: From dose to 8 hours post-dose on Day 1

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

    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). UDPRS Part III was assessed pre-dose and every 30 minutes for 4 hours and then hourly for another 4 hours (i.e., 8 hours following study drug administration) on Day 1.

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

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

    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). An additional single item to rate dyskinesia was added to the UDPRS and was rated from 0 (no dyskinesia) to 4 (severe dyskinesia: maximal amplitude and present during most of the examinations).

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Jul 2018
  2. Study start31 Aug 2018
  3. Primary completion16 Apr 2019
  4. Study completion23 Apr 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Model description
The study consists of 3 sequential, single ascending dose cohorts of 8 participants each with a 6:2 randomization to BTRX-246040 or placebo
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03608371
Sponsor's protocol ID
NEP-PD-201
Status last verified
Jun 2025
First submitted
9 May 2018
Last updated
24 Jun 2025
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BTRX-246040 in people with Parkinson Disease and Motor Disorder, run by BlackThorn Therapeutics, Inc. at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximal Change in UPDRS Part III From Predose to Postdose on Day 1 (time frame: From pre-dose to 8 hours post-dose on Day 1). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03608371.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in