A Phase IIb, Multi-National, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Efficacy of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) (BENEFIT-ALS)
Study Purpose:
The purpose of this research study is to evaluate the safety and effectiveness of CK-2017357 when taken with or without riluzole (also called Rilutek®) in patients with Amyotrophic Lateral Sclerosis (ALS).
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Not enrollingPhase:
Phase IIStudy Chair(s)/Principal Investigator(s):
Jeremy Shefner, MD, PhD (SUNY Upstate Medical University)
Clinicaltrials.gov ID (11 digit #):
NCT01709149Neals Affiliated?
YesCoordinating Center Contact Information
Jinsy Andrews, MD / .(JavaScript must be enabled to view this email address) / 650-624-2929
.(JavaScript must be enabled to view this email address) United States
Full Study Summary:
The length of the study, including screening, dosing, and follow-up, is approximately 20 weeks. After a one-week open-label phase during which all patients will receive CK-2017357 125 milligrams (mg) twice daily, patients will be randomized one to one (fifty-fifty) to receive double-blind CK-2017357 or matching placebo. The CK-2017357/placebo dose will be increased no faster than weekly to each patient's highest tolerated daily dose, with a maximum of 250 mg twice daily. The dose may be decreased based on tolerability. Patients will continue treatment at the highest tolerated dose to complete a total of 12 weeks of double-blind treatment. Patients may be on riluzole or not on riluzole at study entry. Patients not on riluzole must stay off riluzole. Patients on riluzole who are getting double-blind CK-2017357 will be given riluzole at half the labeled dosage (50 mg once a day instead of 50 mg twice a day). Blood tests for safety will be performed. Information about any side effects that may occur will also be collected.
Study Sponsor:
CytokineticsParticipant Duration:
approximately 20 weeks
Estimated Enrollment:
400Estimated Study Start Date:
10/31/2012Estimated Study Completion Date:
11/02/2016Posting Last Modified Date:
02/28/2013Date Study Added to alsconsortium.org:
10/21/2012-
More Information
***Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use during the conduct of this study.
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Eligibility Criteria
Gender:
Female, MaleMinimum Age:
18Maximum Age:
N/ATime since Symptom Onset:
Time since Diagnosis:
Can participants use Riluzole?
Yes
Inclusion Criteria:
Able to comprehend and willing to sign an Informed Consent Form (ICF)
Male or female 18 years of age or older
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)
Upright Slow Vital Capacity (SVC) >60 % of predicted for age, height and sex
At least 4 of the 12 ALSFRS-R questions must be scored 2 or 3
Diminished but measurable maximum voluntary grip strength in at least one hand; i.e., between 10 and 40 pounds (females) and 10 and 60 pounds (males)
Able to swallow tablets without crushing
A caregiver (if one is needed) who can and will observe and report the patient's status
Pre-study clinical laboratory findings within normal range or, if outside of the normal range, deemed not clinically significant by the Investigator
Male patients must 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 during and for 10 weeks after the end of the study
Female patients must be post-menopausal (≥ 1 year) or sterilized, or, if of childbearing potential, not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the course of the study, and use contraceptive drugs or devices as detailed in item 10 for the duration of the study and for 10 weeks after the end of the study
Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use during the conduct of this study.
Exclusion Criteria:
Any use of non-invasive positive pressure ventilation (NIPPV, e.g. continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP]) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
Patients with a diaphragm pacing system (DPS) at study entry or who anticipate DPS placement during the course of the study
Body Mass Index (BMI) of 19.0 kg/m2 or lower
Unwilling to discontinue theophylline-containing medications during study participation
Serum chloride < 100 mg/deciliter (dL)
Neurological impairment due to a condition other than ALS, including history of transient ischemic attack (TIA) within the past year
Presence at screening of any medically significant cardiac, pulmonary, gastrointestinal (GI), musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data
Has taken any investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing
Previously received CK-2017357 in any previous clinical trial -
Site Contact Information
Barrow Neurology
Gale Kittle / .(JavaScript must be enabled to view this email address) / 602-406-4792
Phoenix, Arizona 85013
United StatesUniversity of California, San Diego
Aaryn Belfer / .(JavaScript must be enabled to view this email address) / 858-246-0247
La Jolla, California 92093
United StatesUC Irvine ALS & Neuromuscular Center
Brian Minton / .(JavaScript must be enabled to view this email address) / 714-456-8520
Orange, California 92868
United StatesCoordinated Clinical Research
Luci Barbie / .(JavaScript must be enabled to view this email address) / 619-297-3023
San Diego, California 92037
United StatesCalifornia Pacific Medical Center Forbes Norris MDA/ALS Research Center
Marguerite Engel / .(JavaScript must be enabled to view this email address) / 415-600-3758
San Francisco, California 94115
United StatesHospital for Special Care
Agnes Koczon-Jaremko / .(JavaScript must be enabled to view this email address) / 860-612-6356
New Britain, Connecticut 06053
United StatesMayo Clinic Florida
Catherine Ruiz / .(JavaScript must be enabled to view this email address) / 904-953-6523
Jacksonville, Florida 32224
United StatesEmory University
Meraida Polak / .(JavaScript must be enabled to view this email address) / 404-778-3807
Atlanta, Georgia 30322
United StatesGeorgia Health Sciences University
Brandy Quarles / .(JavaScript must be enabled to view this email address) / 706-721-2681
Augusta, Georgia 30912
United StatesIndiana University Department of Neurology
Sandra Guingrich / .(JavaScript must be enabled to view this email address) / 317-963-7382
Indianapolis, Indiana 46202
United StatesUniversity of Iowa Hospitals and Clinics
Jeri Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
Iowa City, Iowa 52242
United StatesUniversity of Kansas
Maureen Walsh / .(JavaScript must be enabled to view this email address) / 913-588-0645
Kansas City, Kansas 66160
United StatesJohns Hopkins University
Kristen Riley / .(JavaScript must be enabled to view this email address) / 410-955-8511
Baltimore, Maryland 21205
United StatesMassachusetts General Hospital
Lorena Loci / .(JavaScript must be enabled to view this email address) / 617-724-0783
Boston, Massachusetts 02114
United StatesUniversity of Massachusetts Medical School
Diane McKenna-Yasek, RN, BSN / .(JavaScript must be enabled to view this email address) / 508-856-4697
Worcester, Massachusetts 01655
United StatesUniversity of Michigan
Jayna Ballard / .(JavaScript must be enabled to view this email address) / 734-763-9037
Ann Arbor, Michigan 48109
United StatesHenry Ford Hospital
Helen Foley / .(JavaScript must be enabled to view this email address) / 313-916-3955
Detroit, Michigan 48202
United StatesSt Mary's Healthcare
Lynn Cherney / .(JavaScript must be enabled to view this email address) / 616-685-5091
Grand Rapids, Michigan 49503
United StatesHennepin County Medical Center - Berman Center for Research
Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-341-7923
Minneapolis, Minnesota 55415
United StatesSaint Louis University
AnneMarie Fann / .(JavaScript must be enabled to view this email address) / 314-977-4868
St. Louis, Missouri 63104
United StatesWashington University
Charlie Wulf / .(JavaScript must be enabled to view this email address) / 314-362-6980
St. Louis, Missouri 63110
United StatesNeurology Associates
Becky Weber / .(JavaScript must be enabled to view this email address) / 402-483-7226
Lincoln, Nebraska 68506
United StatesDartmouth Hitchcock Medical Center
Jessica Harrington, CCRC / .(JavaScript must be enabled to view this email address) / 603-650-4649
Lebanon, New Hampshire 03756
United StatesHospital for Special Surgery
Nicole Kassebaum / .(JavaScript must be enabled to view this email address) / 646-797-8592
New York, New York 10021
United StatesUniversity of Rochester
Farheen Hussain / .(JavaScript must be enabled to view this email address) / 585-275-4715
Rochester, New York 14642
United StatesSUNY Upstate Medical University
Tanya Perry / .(JavaScript must be enabled to view this email address) / 315-464-4998
Syracuse, New York 13120
United StatesCarolinas Medical Center Department of Neurology
Carissa Ingram / .(JavaScript must be enabled to view this email address) / 704-446-0836
Charlotte, North Carolina 27406
United StatesDuke University
Karen Grace, RN, BSN / .(JavaScript must be enabled to view this email address) / 909-668-2844
Durham, North Carolina 27705
United StatesWake Forest University, School of Medicine
Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / 336-713-8577
Winston-Salem, North Carolina 27157
United StatesOhio State University Department of Neurology
Sharon Chelnick / .(JavaScript must be enabled to view this email address) / 614-293-4973
Columbus, Ohio 43221
United StatesProvidence ALS Center
Kimberly Siebers, RN, BSN / .(JavaScript must be enabled to view this email address) / 503-962-1171
Portand, Oregon 97213
United StatesOregon Health & Science University
Diana Dimitrova / .(JavaScript must be enabled to view this email address) / 503-494-7269
Portland, Oregon 97239
United StatesPenn State Hershey Neuroscience Clinics
Beth Stephens / .(JavaScript must be enabled to view this email address) / 717-531-0003 ext 283395
Hershey, Pennsylvania 17033
United StatesDrexel Neurology
Christine Barr, RN / .(JavaScript must be enabled to view this email address) / 267-507-2633
Philadelphia, Pennsylvania 19107
United StatesUniversity of Pennsylvania
Kelly L Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
Philadelphia, Pennsylvania 19107
United StatesUniversity of Pittsburgh
Danielle Rowlands / .(JavaScript must be enabled to view this email address) / 412-648-9053
Pittsburgh, Pennsylvania 15213
United StatesVanderbilt University Medical Center
Diana Davis / .(JavaScript must be enabled to view this email address) / 615-322-8957
Nashville, Tennessee 37232
United StatesTexas Neurology
Janine McCloskey / .(JavaScript must be enabled to view this email address) / jmccloskey@texasneurology.com
Dallas, Texas 75214
United StatesBaylor College of Medicine
Claire MacAdam / .(JavaScript must be enabled to view this email address) / 713-798-5694
Houston, Texas 77030
United StatesUniversity of Texas Health Science Center at San Antonio
Pamela Kittrell, RN, MSN, CCRC / .(JavaScript must be enabled to view this email address) / 210-450-0524
San Antonio, Texas 78229
United StatesUniversity of Virginia
Amruta Joshi / .(JavaScript must be enabled to view this email address) / 434-982-0293
Charlottesville, Virginia 22908
United StatesThe George Washington University
Angela Kelly / .(JavaScript must be enabled to view this email address) / 202-741-2717
Washington D.C. 20037
United StatesWest Virginia University Department of Neurology
Patricia A Altemus, MS / .(JavaScript must be enabled to view this email address) / 304-598-4000 ext 75656
Morgantown, West Virginia 26506
United StatesMedical College of Wisconsin
Clara Schindler Propsom / .(JavaScript must be enabled to view this email address) / 414-805-3260
Milwaukee, Wisconsin 53226
United StatesHeritage Medical Research
Janet Petrillo / .(JavaScript must be enabled to view this email address) / 403-210-7006
Calgary, Alberta T2N 4Z6
CanadaUniversity of Alberta Hospital
Michael Kreuzer / .(JavaScript must be enabled to view this email address) / 780-407-2944
Edmonton, Alberta B3H 3A7
CanadaUniversity of British Columbia
Marife Fabros / .(JavaScript must be enabled to view this email address) / 604-737-6319
Vancouver, British Columbia V5Z 2G9
CanadaStan Cassidy Centre for Rehabilitation
Susan Brophy / .(JavaScript must be enabled to view this email address) / 506-447-4294
Fredericton, New Brunswick E3B 0C7
CanadaQE II Health Sciences Centre
Margo MacFarlane / .(JavaScript must be enabled to view this email address) / (902) 473-2728
Halifax, Nova Scotia B3H 3A7
CanadaLondon Health Sciences
Joan Martin / .(JavaScript must be enabled to view this email address) / 905-521-2100 ext 75232
Hamilton, Ontario L8S 4K1
CanadaMcMaster University Medical Centre
Joan Martin / .(JavaScript must be enabled to view this email address) / 905-521-2100 ext 75232
Hamilton, Ontario L8S 4K1
CanadaQueen's University: Kingston General
Adriana Breen / .(JavaScript must be enabled to view this email address) / 613-549-6666
Kingston, Ontario K7L 2V7
CanadaUniv. of Toronto - Sunnybrook Health Sciences Centre
Kristiana Salmon / .(JavaScript must be enabled to view this email address) / 514-398-1779
Toronto, Ontario M4N 3M5
CanadaHapital Notre Dame (CHUM) Centre Hospitalier de l'Universite de Montreal
Ann Rusnov / .(JavaScript must be enabled to view this email address) / 514 890 8000
Montreal, Quebec H2L4M1
CanadaMontreal Neurological Institute
Marc Lemieux / .(JavaScript must be enabled to view this email address) / 514-398-2667
Montreal, Quebec H3A 2B4
CanadaCHU de Quebec: Hopital l'Enfant-Jesus
Lucie Morel / .(JavaScript must be enabled to view this email address) / 418-649-0252
Quebec, Quebec G1J 1Z4
CanadaCharite Universitatsmedizin
Dorit Strassenburg / .(JavaScript must be enabled to view this email address) / 49-30-450-6603-96
Berlin, 13353
GermanyHannover Medical School
Chantal Fischer / .(JavaScript must be enabled to view this email address) / 0049 (0) 511 532-8333
Hannover, 30625
GermanyUniversity of Ulm
Sabine Raubold / .(JavaScript must be enabled to view this email address) / 49-731- 500 63006
Ulm, 89081
GermanyCHRU de Lille - Hôpital Roger Salengro
Francine Niset-Ratel / .(JavaScript must be enabled to view this email address) / 0033 (0)6 07 50 34 20
Lille, F-59037
FranceCHU de Limoges - Hôpital Dupuytren
Olivier Villeneuve / .(JavaScript must be enabled to view this email address) / 0033 (0)5 55 05 65 59
Limoges, 87042
FranceCHU Montepellier
Sébastien Alphandéry / .(JavaScript must be enabled to view this email address) / 0033 (0)4 67 52 93 28
Montpellier, 34295
FranceHapital de la Salpatrière
Lucette Lacomblez / .(JavaScript must be enabled to view this email address) / 0033 (0)1 42 16 24 72
Paris, Cedex 13
FranceWalton Centre for Neurology and Neurosurgery
Louise Pate / .(JavaScript must be enabled to view this email address) / +44 (0)1515295666
Liverpool, L9 7LJ
United KingdomKings College Hospital NHS Foundation Trust
Andrew Dougherty / .(JavaScript must be enabled to view this email address) / +44 (0)20 7848 5355
London, SE5 8AF
United KingdomJohn Radcliffe Hospital
Edna Gonzalvo / .(JavaScript must be enabled to view this email address) / +44 (0)1865231556
Oxford, OX3 9DU
United KingdomSheffield Institute for Translational Neuroscience
Theresa Walsh / .(JavaScript must be enabled to view this email address) / 44 -0-114-222-2226
Sheffield,
United KingdomBarts and the London MND & the Centre Royal London Hospital
Anna Belin / .(JavaScript must be enabled to view this email address) / 44-020- 788-27150
Whitechapel, London,
United KingdomTrinity College, Beaumont Hospital
Liz Fogarty / .(JavaScript must be enabled to view this email address) / 00353-1-809-3874
Dublin, 9
Ireland