A Phase 2 Study of CX-8998 in Adults With Tremor Associated With Parkinson's Disease
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|ClinicalTrials.gov Identifier: NCT03436953|
Recruitment Status : Withdrawn (The program was not initiated nor were any patients ever enrolled. The investigations detailed in the submitted protocol were incorporated into another program.)
First Posted : February 19, 2018
Last Update Posted : May 28, 2021
|Condition or disease||Intervention/treatment||Phase|
|Parkinson's Disease Tremor||Drug: CX-8998 Drug: Placebo||Phase 2|
This is a Phase 2, multicenter, double-blind, placebo-controlled, parallel-group study consisting of a screening period of up to 4 weeks, a 4 week randomized double-blind, dose-titration treatment period, followed by a 1 week safety follow-up period after the last dose of study medication, and a scheduled follow-up safety telephone call one week later.
Subjects will be randomized 1:1 to one of two treatment groups. Group A will receive titrating doses of CX-8998 up to 10 mg BID and Group B will receive placebo.
Subjects will participate for a total of up to 12 weeks, including screening, the 4-week treatment period and follow-up.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of CX-8998 for Tremor Associated With Parkinson's Disease|
|Estimated Study Start Date :||December 2019|
|Estimated Primary Completion Date :||September 2020|
|Estimated Study Completion Date :||December 2020|
|Experimental: CX-8998 T-type calcium channel blocker||
T-type calcium channel blocker
|Placebo Comparator: Comparator||
- Change from Baseline to Day 28 on the MDS-UPDRS Tremor Score as scored by the central rater [ Time Frame: Baseline through completion of study treatment period, an average of 28 days ]The MDS-UPDRS is a multi-dimensional scale that assesses the motor and non-motor impact of PD across four parts. Part I: Non-Motor Experiences of Daily Living; Part II: Motor Experiences of Daily Living; Part III: Motor Examination; and Part IV: Motor Complications.
- Change from Baseline to Day 28 on the TETRAS Activity of Daily Living subscale [ Time Frame: Baseline through completion of study treatment period, an average of 28 days ]The ADL section of TETRAS has 12 items, each rated 0, 1, 2, 3 or 4. The maximum total score is 48. Item 1 addresses speech; item 10 addresses occupational impairment; and item 12 assesses social impact. The other 9 items assess activities that are affected primarily by upper limb tremor
- Change from Baseline to Day 28 in accelerometry score [ Time Frame: Baseline through completion of study treatment period, an average of 28 days ]Accelerometry will be used to obtain quantitative measurements of tremor. The Kinesia ONE device will be placed on the index finger and worn in the clinic immediately after execution of the MDS-UPDRS and/or TETRAS performance subscale. A total of 4 tasks will be performed on the left side and then again on the right side to assess resting, postural, kinetic, and lateral wing beating tremor. Each task will be performed for 15 seconds.
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] as assessed by CTCAE v4.0 [ Time Frame: Through study completion, an average of 12 weeks ]Treatment-emergent adverse events are all adverse events occurring during the treatment period or a pretreatment event that worsens in intensity during the treatment period.
- Changes from baseline in QTcF [ Time Frame: Baseline through study completion, an average of 5 weeks ]Fridericia's Correction Formula (QTCF) is a formula which takes into account the physiologic shortening of the QT interval which occurs as the heart rate increases, permitting comparison of the QT interval across a range of rates.
- Percentage of subjects who did not complete the study due to Treatment Emergent Adverse Events as assessed by CTCAE v4.0 [ Time Frame: Duration of study, an average of 12 weeks ]Treatment-emergent adverse events are all adverse events occurring during the treatment period or a pretreatment event that worsens in intensity during the treatment period.
- Percentage of subjects with Serious Adverse Events as assessed by CTCAE v4.0 [ Time Frame: Duration of study, an average of 12 weeks ]Any adverse event that results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect observed in any offspring of the subject conceived during treatment with the study drug or is an important medical event.
- Percentage of subjects with Adverse Events of Special Interest as assessed by CTCAE v4.0 [ Time Frame: Duration of study, an average of 12 weeks ]An adverse event of special interest is a serious adverse event as defined in Outcome 8.
- Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: Screening through study completion, an average of 8 weeks ]The Columbia-Suicide Severity Rating Scale (C-SSRS) is a questionnaire used for suicide assessment. A numerical score is derived from the C-SSRS categories. The score is created at each assessment for each patient and is used for determining treatment emergence. Suicidal Ideation Score: The maximum suicidal ideation category (1-5 on the C-SSRS) present at the assessment.
- Epworth Sleepiness Scale [ Time Frame: Baseline through completion of study treatment period, an average of 28 days ]The Epworth Sleepiness Scale (ESS) is a scale intended to measure daytime sleepiness that is measured by use of a very short questionnaire. The questionnaire asks the subject to rate his or her probability of falling asleep on a scale of increasing probability from 0 to 3 for 8 different situations. The scores are added together to obtain a single number.
- Questionnaire for Impulsive-Compulsive Disorders in Parkinson's Disease-Rating Scale (QUIP-RS) [ Time Frame: Screening through study completion, an average of 8 weeks ]The QUIP-RS was developed to assess the severity of impulse control disorders and related behavior symptoms and to monitor changes in symptoms over time. The QUIP-RS has 4 primary questions (pertaining to commonly reported thoughts, urges/desires, and behaviors associated with impulsive control disorders), each of which is applied to 4 impulsive behaviors (compulsive gambling, buying, eating, and sexual behavior) and 3 related disorders (medication use, punding, and hobbyism). It uses a 5-point scale to gauge the frequency of behaviors. Scores for each behavior and related disorder range from 0 to 16, with a higher score indicating greater severity (ie, frequency) of symptoms. The total QUIP-RS score for all impulsive control disorders and related disorders combined ranges from 0 to 112.
- University of Miami Parkinson's disease Hallucinations Questionnaire (UM-PDHQ) [ Time Frame: Screening, Baseline and Day 28, an average of 28 days ]The UM-PDHQ is a 20-item clinician-administered questionnaire that is completed during a structured interview. Questions are divided into 2 groups: a quantitative group that consists of 6 questions (modality, frequency, duration, insight, emotional burden) and a qualitative group that consists of 14 questions. Scores range from 0-14.
- Hospital Anxiety and Depression Scale (HADS) [ Time Frame: Screening and Day 28, an average of 28 days ]The Hospital Anxiety and Depression Scale (HADS), a self-assessment scale, was developed to detect states of depression, anxiety and emotional distress. The HADS is a 14-item scale that generates ordinal data. Seven of the items relate to anxiety, and 7 of the items relate to depression. Each item on the questionnaire is scored from 0 to 3, with individual scores ranging between 0 and 21 for either anxiety or depression.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03436953
|Study Director:||Stacey Boyer, PhD||Jazz Pharmaceuticals|