Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
- Registration Number
- NCT03068754
- Lead Sponsor
- Mallinckrodt
- Brief Summary
About 213 people with ALS will participate in this study. There will be locations in North and South America.
During the first part, participants will be randomly assigned to a group (like by flipping a coin). Out of every 3:
* 2 will get the study drug
* 1 will get a look-alike with no drug in it (placebo)
During the second part, everyone will get the study drug.
Participation will help doctors find out if Acthar can help or slow down the symptoms of ALS better than placebo.
- Detailed Description
This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult participants with ALS. Approximately 213 participants will be enrolled.
Following a screening period of up to 28 days, participants with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units \[U\]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3-week taper.
Participants who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase in which all participants will receive Acthar 0.2 mL (16 U) daily.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 143
- Is 18-75 years of age at Screening
- Has ALS symptom onset within 2 years prior to Screening
- Has forced vital capacity (FVC) no higher than 60% at screening
- If taking riluzole, is on a stable dose for 4 weeks before Screening
- Has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type
- Has used any medication within a time period not allowed per protocol
- Has history of Type 1 or Type 2 diabetes mellitus, or any clinically significant infection
- Used edaravone less than 1 week before Screening
- Received any stem cell replacement therapy
- Used steroids within a time period not allowed per protocol
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm D: Extension Period Placebo-Acthar Placebo Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. Arm B: Treatment Period Placebo Placebo Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (Matching Placebo), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39. Arm A: Treatment Period Acthar Acthar Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39. Arm C: Extension Period Acthar-Acthar Acthar Participants who receive Acthar during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. Arm D: Extension Period Placebo-Acthar Acthar Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
- Primary Outcome Measures
Name Time Method Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Baseline, Week 36 The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival.
ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory)
A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.Number of Participants Experiencing an Adverse Event During the Treatment Period by the end of the treatment period (within 36 Weeks) Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events.
Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period by the time of database lock (within 84 weeks) Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events.
- Secondary Outcome Measures
Name Time Method Treatment Period: Spirometry (%) Baseline, Week 36 Spirometry (meaning the measuring of breath) is the most common of the lung function tests. It measures how much air can be inhaled \[Forced Vital Capacity (FVC)\] and exhaled \[(Forced Expiratory Volume in one second (FEV1)\].
Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R Baseline, Week 36 The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory).
The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R Baseline, Week 84 The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory).
The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.
Trial Locations
- Locations (69)
University of California Irvine Medical Center
🇺🇸Orange, California, United States
University of California Los Angeles
🇺🇸Los Angeles, California, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
University of Nebraska Medical Center - Physicians Clinical Neurosciences Center
🇺🇸Omaha, Nebraska, United States
Las Vegas Clinic
🇺🇸Las Vegas, Nevada, United States
Penn State Health Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
University of Vermont Medical Center
🇺🇸Colchester, Vermont, United States
Hospital Español
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Hospital Británico de Buenos Aires
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Clinical Research Institute Saltillo S.A. de C.V.
🇲🇽Saltillo, Coahuila, Mexico
Clinical Research Institute S.C.
🇲🇽San Lucas Tepetlacalco, Tlalnepantla De Baz, Mexico
FAICIC Clinical Researc
🇲🇽Veracruz, Mexico
Emory University
🇺🇸Atlanta, Georgia, United States
George Washington University
🇺🇸Washington, District of Columbia, United States
Neuromuscular Research Center
🇺🇸Phoenix, Arizona, United States
University of Florida - McKnight Brain Institute
🇺🇸Gainesville, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
IADIN
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Fundación Scherbovsky
🇦🇷Ciudad de Mendoza, Mendoza, Argentina
Hospital Universitario "Dr. José Eleuterio González"
🇲🇽Monterrey, Nuevo Leon, Mexico
Mayo Clinic - Arizona
🇺🇸Scottsdale, Arizona, United States
John Hopkins Outpatient Center
🇺🇸Baltimore, Maryland, United States
University of Massachusetts Medical School
🇺🇸Worcester, Massachusetts, United States
Georgetown University
🇺🇸Washington, District of Columbia, United States
Hospital Italiano de Buenos Aires
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Medical College of Wisconsin/Froedtert Hospital
🇺🇸Milwaukee, Wisconsin, United States
Mercy Health- Saint Mary's
🇺🇸Grand Rapids, Michigan, United States
Hospital Nacional Cayetano Heredia
🇵🇪Lima, Peru
Neurology Associates
🇺🇸Lincoln, Nebraska, United States
Jersey Shore University Medical Center
🇺🇸Neptune, New Jersey, United States
DIABAID
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
INEBA
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Providence ALS Center
🇺🇸Portland, Oregon, United States
Wesley Neurology Clinic
🇺🇸Cordova, Tennessee, United States
STAT Research
🇦🇷Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Recherche Sepmus inc
🇨🇦Greenfield Park, Quebec, Canada
Centro de Trastornos del Movimiento (CETRAM)
🇨🇱Santiago, Región Metropolitana, Chile
ILAIM
🇦🇷Ciudad de Córdoba, Córdoba, Argentina
Hospital Nivel IV Carlos Alberto Seguin Escobedo
🇵🇪Arequipa, Peru
Hospital Almenara
🇵🇪Lima, Peru
Edmonton Kaye Clinic
🇨🇦Edmonton, Alberta, Canada
Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )
🇦🇷Ciudad De Santa Fe, Santa Fe, Argentina
Centre de recherché du Centre Hospitalier de l'Universite de Montreal (CRCHUM)
🇨🇦Montréal, Quebec, Canada
Montreal Neurological Institute & Hospital
🇨🇦Montréal, Quebec, Canada
Hospital Nacional IV Alberto Sabogal Sologuren
🇵🇪Callao, Peru
Clinica Dávila
🇨🇱Santiago, Región Metropolitana, Chile
Biomedica Research Group AV Salvador 149, oficina 1101
🇨🇱Santiago, Chile
Centro de Investigaciones Clínicas SAS
🇨🇴Cali, Colombia
SMIQ BRCR Global México
🇲🇽Querétaro City, Querétaro, Mexico
Phylasis Clinicas Research
🇲🇽Mexico City, Mexico
Centro Especializado en Investigación Clínica S.C.
🇲🇽Boca Del Río, Veracruz, Mexico
Instituto Neuro Cardiovascular de las Américas
🇵🇪Lima, Peru
Indiana University-Neuroscience Center of Excellence/Goodman Hall
🇺🇸Indianapolis, Indiana, United States
Keck School of Medicine, University of Southern California
🇺🇸Los Angeles, California, United States
University of California San Diego
🇺🇸La Jolla, California, United States
Loma Linda University Health System, Department of Neurology
🇺🇸Loma Linda, California, United States
Augusta University
🇺🇸Augusta, Georgia, United States
University of Kentucky Chandler Medical Center
🇺🇸Lexington, Kentucky, United States
Columbia Presbyterian Hospital
🇺🇸New York, New York, United States
Temple University School of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
Allegheny General Hospital
🇺🇸Pittsburgh, Pennsylvania, United States
Texas Neurology, P.A.
🇺🇸Dallas, Texas, United States
Austin Neuromuscular Center
🇺🇸Austin, Texas, United States
The Methodist Hospital
🇺🇸Houston, Texas, United States
Swedish Neuroscience Institute
🇺🇸Seattle, Washington, United States
VCU Medical Center
🇺🇸Richmond, Virginia, United States
Colorado Springs Neurological Associates
🇺🇸Colorado Springs, Colorado, United States
California Pacific Medical Center
🇺🇸San Francisco, California, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States