A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
This study has been completed.
Sponsor:
Cytokinetics
Information provided by:
Cytokinetics
ClinicalTrials.gov Identifier:
NCT01089010
First received: March 16, 2010
Last updated: May 11, 2011
Last verified: May 2011
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Purpose
The primary objective of this study is to demonstrate a pharmacodynamic effect of CK 2017357 on measures of skeletal muscle function or fatigability in patients with ALS.
| Condition | Intervention | Phase |
|---|---|---|
|
Amyotrophic Lateral Sclerosis |
Drug: Placebo Drug: 250 mg CK-2017357 Drug: 500 mg CK-2017357 |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A Phase II, Double-Blind, Randomized, Placebo-Controlled, Three-Way Crossover, Pharmacokinetic and Pharmacodynamic Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) |
Resource links provided by NLM:
Genetics Home Reference related topics:
amyotrophic lateral sclerosis
MedlinePlus related topics:
Amyotrophic Lateral Sclerosis
U.S. FDA Resources
Further study details as provided by Cytokinetics:
Primary Outcome Measures:
- The primary objective of this study is to demonstrate a pharmacodynamic effect of CK-2017357 on measures of skeletal muscle function or fatigability in patients with ALS. [ Time Frame: 2 days ] [ Designated as safety issue: No ]
In this hypothesis-generating early Phase II study, multiple assessments of skeletal muscle function will be made without specifying a single primary endpoint, including:
- ALS Functional Assessment
- Maximum Grip Strength (bilateral)
- Maximum Grip Strength Fatigability (bilateral)
- Shoulder Extension Fatigue (bilateral)
- Slow Vital Capacity
- Maximal Voluntary Ventilation
- Sniff Inspiratory Pressure
- Maximum Voluntary Muscle Contraction of multiple bilateral muscle groups using Hand Held Dynamometry
- Repeated Sub-Maximum Grip Strength Fatigability (bilateral)
Secondary Outcome Measures:
- To evaluate and characterize the relationship, if any, between the plasma concentration of CK-2017357 and its pharmacodynamic effects (PK/PD relationship) [ Time Frame: 2 day ] [ Designated as safety issue: No ]
- To evaluate the safety and tolerability of 2 doses of CK-2017357 given as single doses administered orally to patients with ALS [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
- To evaluate the effect of CK-2017357 on patient and investigator determined global functional assessment [ Time Frame: 2 days ] [ Designated as safety issue: No ]
| Enrollment: | 67 |
| Study Start Date: | March 2010 |
| Study Completion Date: | November 2010 |
| Primary Completion Date: | November 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Three-way crossover
2 oral dose levels of CK-2017357 and placebo
|
Drug: Placebo
Matching placebo in capsules administered as a single oral dose.
Drug: 250 mg CK-2017357
250 mg CK-2017357 in capsules administered as a single oral dose.
Drug: 500 mg CK-2017357
500 mg CK-2017357 in capsules administered as a single oral dose.
|
Detailed Description:
This study is a Phase II, double-blind, randomized, placebo-controlled, three-way crossover study of CK-2017357 in patients with ALS. 36 to 72 patients will be randomized to one of six different treatment sequences. Each treatment sequence consists of three dosing periods; in each dosing period¸ patients receive a single oral dose of placebo, 250 mg of CK-2017357, or 500 mg of CK-2017357. All six treatment sequences will enroll approximately the same number of patients. A washout period of at least 6 days (to a maximum of 10 days) will be employed between the doses for each patient. This study is designed to assess the effect of CK-2017357 on maximal voluntary muscle strength, on the development of fatigue at maximal and sub-maximal voluntary muscle contraction, and on selected pulmonary function parameters. The plasma concentration of CK-2017357 will be measured at selected time points after each of two single doses of CK-2017357 in men and women. The plasma concentration versus time data obtained in this study may be used to develop a population PK model and estimate inter-subject variability of PK parameters in this target patient population, in particular between male and female study patients.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Able to comprehend and willing to sign an Informed Consent Form (ICF)
- A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) (Brooks, et al., 2000)
- Males or Females 18 years of age or older
- Body mass index (BMI) of 18.0 to 30.0 kg/m2, inclusive
- Maximum voluntary grip strength between 10 & 40 pounds (females) and 10 & 60 pounds (males)
- Able to maintain grip contraction for 15 seconds
- Upright Slow Vital Capacity (SVC) ≥40% of predicted for age, height, and sex
- Able to perform pulmonary function tests
- Pre-study clinical laboratory findings (including troponin I (TnI) and CPK) within normal range, or if outside of the normal range, deemed not clinically significant by the Investigator
- For female patients only: The patient is post-menopausal (≥ 1 year) or sterilized, or if she is of childbearing potential, she is not breastfeeding, her pregnancy test is negative, she has no intention to become pregnant during the course of the study, and she is using contraceptive drugs or devices. For male patients only: Male patients agree for the duration of the study and 10 weeks after the end of the study 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) or the male patient must agree to abstain from sexual intercourse for 10 weeks after the end of the study.
Exclusion Criteria:
- Significant hepatic/renal insufficiency as defined by Upper Limit of Normal (ULN) laboratory findings
- Life expectancy <3 months
- Participation in any trial in which receipt of investigational study drug occurred within 30 days prior to dosing
- Any prior treatment with CK-2017357
- In the opinion of the Investigator, the patient is not suitable to participate in the study
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01089010
Please refer to this study by its ClinicalTrials.gov identifier: NCT01089010
Locations
| United States, Arizona | |
| Phoenix Neurological Associates, Ltd. | |
| Phoenix, Arizona, United States, 85018 | |
| United States, California | |
| University Neurology Associates | |
| Fresno, California, United States, 93701 | |
| California Pacific Medical Center | |
| San Francisco, California, United States, 94115 | |
| United States, Florida | |
| Mayo Clinic Florida | |
| Jacksonville, Florida, United States, 32224 | |
| United States, Kentucky | |
| University of Kentucky | |
| Lexington, Kentucky, United States, 40536 | |
| United States, Maryland | |
| Johns Hopkins Hospital | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Massachusetts General Hospital | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Missouri | |
| Washington University | |
| St. Louis, Missouri, United States, 63110 | |
| United States, New York | |
| SUNY Upstate Medical Center | |
| Syracuse, New York, United States, 13210 | |
| United States, North Carolina | |
| Duke University | |
| Durham, North Carolina, United States, 27705 | |
| United States, Oregon | |
| Providence ALS Center | |
| Portland, Oregon, United States, 97213 | |
| United States, Pennsylvania | |
| Drexel University College of Medicine, Dept of Neurology | |
| Philadelphia, Pennsylvania, United States, 19102 | |
| Penn State | |
| University Park, Pennsylvania, United States, 17033 | |
| United States, Texas | |
| The University of Texas Health Science Center at San Antonio | |
| San Antonio, Texas, United States, 78229 | |
| United States, Vermont | |
| University of Vermont | |
| Burlington, Vermont, United States, 05401 | |
Sponsors and Collaborators
Cytokinetics
Investigators
| Study Chair: | Jeremy M Shefner, MD, PhD | State University of New York - Upstate Medical University |
More Information
No publications provided
| Responsible Party: | Andrew Wolff, MD, FACC, Chief Medical Officer, Cytokinetics, Inc. |
| ClinicalTrials.gov Identifier: | NCT01089010 History of Changes |
| Other Study ID Numbers: | CY 4021 |
| Study First Received: | March 16, 2010 |
| Last Updated: | May 11, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Amyotrophic Lateral Sclerosis Motor Neuron Disease Sclerosis Central Nervous System Diseases Metabolic Diseases Nervous System Diseases |
Neurodegenerative Diseases Neuromuscular Diseases Pathologic Processes Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies |
ClinicalTrials.gov processed this record on February 27, 2015