Study Stopped
Closed after complete treatment of 32 patients after 21 months' recruitment.
Pharmacodynamic Study of CK-2017357 in Patients With Generalized Myasthenia Gravis
A Phase II, Double-Blind, Randomized, Three-Way Crossover, Placebo-Controlled, Pharmacodynamic Study of CK-2017357 in Patients With Generalized Myasthenia Gravis on Standard Therapy
2 other identifiers
interventional
32
1 country
15
Brief Summary
The primary objective of this early-stage clinical study is to demonstrate an effect of single doses of CK-2017357 on measures of skeletal muscle function and fatigability in patients with generalized myasthenia gravis (MG).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Dec 2010
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2010
CompletedFirst Submitted
Initial submission to the registry
December 28, 2010
CompletedFirst Posted
Study publicly available on registry
December 30, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2012
CompletedSeptember 24, 2019
September 1, 2019
1.8 years
December 28, 2010
September 18, 2019
Conditions
Outcome Measures
Primary Outcomes (3)
Quantitative Myasthenia Gravis score (QMG)
A quantitative motor assessment of muscular weakness on a scale of 0 to 3 with 0 representing "none" and 3 representing "severe". Muscular assessments included in the overall score are effects on double vision, ptosis, facial muscles, swallowing, speech, outstretched arms and legs, forced vital capacity, hand grip strength, and ability to lift head.
1 day
Pulmonary Function Test (VC in liters)
Forced Vital Capacity
1 day
Manual Muscle Test (MMT)
Sum of strength or function values assessed by physician on the neck, shoulder, hip and ankle
1 day
Secondary Outcomes (7)
Characterize dose and plasma concentrations of CK-2017357 and QMG
2 days
Characterize dose and plasma concentrations of CK-2017357 and Forced Vital Capacity (FVC)
2 days
Characterize dose and plasma concentrations of CK-2017357 and Manual Muscle Test (MMT)
2 days
Number of patients with adverse events
4 weeks
Modified MG Symptom Score
2 days
- +2 more secondary outcomes
Study Arms (6)
Treatment Sequence 1
EXPERIMENTALDosing Period 1 Placebo; Dosing Period 2 CK-2017357 250 mg; Dosing Period 3 CK-2017357 500 mg
Treatment Sequence 2
EXPERIMENTALDosing Period 1 Placebo; Dosing Period 2 CK-2017357 500 mg; Dosing Period 3 CK-2017357 250 mg
Treatment Sequence 3
EXPERIMENTALDosing Period 1 CK-2017357 250 mg; Dosing Period 2 Placebo; Dosing Period 3 CK-2017357 500 mg
Treatment Sequence 4
EXPERIMENTALDosing Period 1 CK-2017357 250 mg; Dosing Period 2 CK-2017357 500 mg; Dosing Period 3 Placebo
Treatment Sequence 5
EXPERIMENTALDosing Period 1 CK-2017357 500 mg; Dosing Period 2 Placebo; Dosing Period 3 CK-2017357 250 mg
Treatment Sequence 6
EXPERIMENTALDosing Period 1 CK-2017357 500 mg; Dosing Period 2 CK-2017357 250 mg; Dosing Period 3 Placebo
Interventions
Matching placebo in capsules administered as a single oral dose.
250 mg CK-2017357 in capsules administered as a single oral dose.
500 mg CK-2017357 in capsules administered as a single oral dose.
Eligibility Criteria
You may qualify if:
- Ability to comprehend and willing to sign an Informed Consent Form (ICF)
- Ability to understand written and oral English language
- Males and females between 18 and 80 years of age, inclusive
- Patient's signs and symptoms not better explained by another disease process
- Established diagnosis of MG defined as clinical evidence of muscle weakness and positive AChR-binding antibody titer (\>0.02 nmol/L)
- Myasthenia Gravis Foundation of America (MGFA) clinical classification II or III
- Stable MG disease for 4 weeks prior to randomization
- Ability to refrain from IVIg treatments during the course of the study
- Ability to refrain from cholinesterase-inhibitors (e.g. pyridostigmine) for 12 hours before each dose
- Ability to perform all elements of the QMG
- Grade of 2 or 3 in two or more of the following muscle groups as measured by QMG: right or left arm flexion, head lift, and right or left leg raise at 45° Note: Patients may re-screen if they fail due to inadequate weakness from taking pyridostigmine within 12 hours of screening
- Body mass index (BMI) of 18.0 to 36.0 kg/m2, inclusive
- Pre-study clinical laboratory findings (including troponin I \[TnI\] and creatine phosphokinase \[CPK\]) within the normal range, or if outside of the normal range, deemed not clinically significant by the Investigator
- For female patients only: Agreement to use a double barrier during sexual intercourse (1 hormonal, plus 1 barrier method, or 2 simultaneous barrier methods) birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures)
- For male patients only: Agreement either to use a condom during sexual intercourse with female partners who are of reproductive potential and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide, or oral contraceptives) for the duration of the study and 10 weeks after the end of the study or to abstain from sexual intercourse for the duration of the study and 10 weeks after the end of the study
You may not qualify if:
- History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue
- Other major chronic or debilitating illnesses within six months prior to study entry
- Hepatic insufficiency (defined as ALT or AST \> 3x ULN, or total bilirubin \> 3 mg/dL)
- Renal insufficiency (defined as serum creatinine \> 2.5 mg/dL or receiving dialysis)
- Other myasthenic syndromes (e.g. Lambert Eaton syndrome; inherited myasthenic syndrome)
- Female patients who are premenopausal and are: (a) pregnant on the basis of a serum pregnancy test, (b) breast-feeding, or (c) not using an effective method of double barrier (1 hormonal plus 1 barrier method or 2 simultaneous barrier methods) birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures)
- Receipt of IVIg or plasmapheresis treatment within 6 weeks prior to the first dose of study drug
- Changes to immunosuppressive treatments (i.e., prednisone) within 6 weeks prior to the first dose of study drug
- Rituxan treatment within 3 months prior to study entry
- Participation in any other investigational study drug or device trial in which receipt of an investigational study drug or device occurred within 30 days prior to dosing
- Any prior treatment with CK-2017357
- Recent history of alcoholism or drug abuse, or significant behavioral or psychiatric problems, or other conditions which in the Investigator's opinion may impair ability to adequately comply with the requirements of the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
UCSF - Fresno
Fresno, California, 93721, United States
University of California - Irvine
Orange, California, 92868, United States
California Pacific Medical Center
San Francisco, California, 94115, United States
Stanford University
Stanford, California, 94305, United States
Hospital for Special Care
New Britain, Connecticut, 06053, United States
The University of Kansas Medical Center
Kansas City, Kansas, 26506, United States
Johns Hopkins
Baltimore, Maryland, 21205, United States
Neurocare Center for Research
Newton, Massachusetts, 02459, United States
University of North Carolina
Chapel Hill, North Carolina, 27599, United States
Duke University
Durham, North Carolina, 27710, United States
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19107, United States
West Penn Allegheny Health System
Pittsburgh, Pennsylvania, 15212, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
University of Texas Health Science Center
San Antonio, Texas, 78229, United States
University of Virginia Health System
Charlottesville, Virginia, 22908, United States
Related Publications (1)
Sanders DB, Rosenfeld J, Dimachkie M, Meng L, Malik FI. A Study to Evaluate Efficacy, Safety and Tolerability of Single Doses of Tirasemtiv in Patients with Myasthenia Gravis. 65th Annual Meeting of the American Academy of Neurology, San Diego, CA, March 2013
RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Andrew Wolff, MD, FACC
Cytokinetics, Inc.
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 28, 2010
First Posted
December 30, 2010
Study Start
December 1, 2010
Primary Completion
October 1, 2012
Study Completion
October 1, 2012
Last Updated
September 24, 2019
Record last verified: 2019-09