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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03608371
• Date of registration: 09/05/2018
• Prospective Registration: Yes
• Primary sponsor: BlackThorn Therapeutics, Inc.
• Public title: BTRX-246040 Study in Subjects With Parkinson's Disease With Motor Fluctuations
• Scientific 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
• Date of first enrolment: August 31, 2018
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03608371
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jane M Tiller, M.D.
• Affiliation: BlackThorn Therapeutics, Inc. (Sponsor)

Key inclusion & exclusion criteria

Inclusion Criteria:

Diagnosed with Parkinson's disease (consistent with the UK PD Society Brain Bank Criteria
for the Diagnosis of PD) Men or women = 30 years old and = 76 years old Female subjects
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 four 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 subject 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 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 subjects 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.

Exclusion Criteria:

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 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
one 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 the protocol-defined threshhold. Subjects
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 PI 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 subject 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 subject 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 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 subject's participation at study entry.

ECG abnormalities at Visit 1 (screening) or Visit 2 that, in the judgment of the
Investigator, are clinically significant related to the subject's participation

Using the following concomitant medications (contact the Sponsor-designated medical monitor
to determine eligibility when in doubt):

1. proton pump inhibitors within 5 half-lives of Screening

2. 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). Contact the Sponsor-designated medical monitor to determine
eligibility when in doubt.

Previous completion or withdrawal from this study or any other study in


Age minimum: 30 Years
Age maximum: 76 Years
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson Disease

Motor Disorder

Intervention(s)

Drug: BTRX-246040

Drug: Placebo

Primary Outcome(s)

Change in Motor Function [Time Frame: from pre-dose to post-dose on Day 1]

Secondary Outcome(s)

Area under the curve for UPDRS Part III during the 8 hours of assessment on Day 1 [Time Frame: from pre-dose to post-dose on Day 1]

Percentage of subjects that turn ON on Day 1 [Time Frame: from pre-dose to post-dose on Day 1]

Time to ON on Day 1 [Time Frame: from pre-dose to post-dose on Day 1]

Change from pre-dose UPDRS dyskinesia score [Time Frame: from pre-dose to post-dose on Day 1]

Duration of ON time on Day 1 [Time Frame: from pre-dose to post-dose on Day 1]

Secondary ID(s)

NEP-PD-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors