Clinical trial NCT03068754: Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
Official title: A Multicenter, Double Blind, Placebo Controlled Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Lead sponsor: Mallinckrodt
Sponsor class: INDUSTRY
First posted: 2017-03-03
Enrollment: 143
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants will be assigned to treatment group randomly (like flipping a coin). They will have a 2 out of 3 chance of receiving the study drug, and a 1 out of 3 chance of receiving placebo during the treatment period. All participants who continue into the extension period receive Acthar.
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. Is 18-75 years of age at Screening
2. Has ALS symptom onset within 2 years prior to Screening
3. Has forced vital capacity (FVC) no higher than 60% at screening
4. If taking riluzole, is on a stable dose for 4 weeks before Screening

Exclusion Criteria:

1. Has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type
2. Has used any medication within a time period not allowed per protocol
3. Has history of Type 1 or Type 2 diabetes mellitus, or any clinically significant infection
4. Used edaravone less than 1 week before Screening
5. Received any stem cell replacement therapy
6. Used steroids within a time period not allowed per protocol

Primary outcomes:
measure: Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Baseline, Week 36
measure: Number of Participants Experiencing an Adverse Event During the Treatment Period
timeFrame: by the end of the treatment period (within 36 Weeks)
measure: Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period
timeFrame: by the time of database lock (within 84 weeks)
Secondary outcomes:
measure: Treatment Period: Spirometry (%)
timeFrame: Baseline, Week 36
measure: Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R
timeFrame: Baseline, Week 36
measure: Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R
timeFrame: Baseline, Week 84
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Neuromuscular Research Center
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic - Arizona
state: Arizona
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Loma Linda
country: United States
facility: Loma Linda University Health System, Department of Neurology
state: California
city: Los Angeles
country: United States
facility: Keck School of Medicine, University of Southern California
state: California
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Orange
country: United States
facility: University of California Irvine Medical Center
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Colorado Springs
country: United States
facility: Colorado Springs Neurological Associates
state: Colorado
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Washington
country: United States
facility: George Washington University
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida - McKnight Brain Institute
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University-Neuroscience Center of Excellence/Goodman Hall
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky Chandler Medical Center
state: Kentucky
city: Baltimore
country: United States
facility: John Hopkins Outpatient Center
state: Maryland
city: Worcester
country: United States
facility: University of Massachusetts Medical School
state: Massachusetts
city: Grand Rapids
country: United States
facility: Mercy Health- Saint Mary's
state: Michigan
city: Lincoln
country: United States
facility: Neurology Associates
state: Nebraska
city: Omaha
country: United States
facility: University of Nebraska Medical Center - Physicians Clinical Neurosciences Center
state: Nebraska
city: Las Vegas
country: United States
facility: Las Vegas Clinic
state: Nevada
city: Neptune
country: United States
facility: Jersey Shore University Medical Center
state: New Jersey
city: New York
country: United States
facility: Columbia Presbyterian Hospital
state: New York
city: Portland
country: United States
facility: Providence ALS Center
state: Oregon
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University School of Medicine
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny General Hospital
state: Pennsylvania
city: Cordova
country: United States
facility: Wesley Neurology Clinic
state: Tennessee
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Dallas
country: United States
facility: Texas Neurology, P.A.
state: Texas
city: Houston
country: United States
facility: The Methodist Hospital
state: Texas
city: Colchester
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Richmond
country: United States
facility: VCU Medical Center
state: Virginia
city: Seattle
country: United States
facility: Swedish Neuroscience Institute
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin/Froedtert Hospital
state: Wisconsin
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: IADIN
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: STAT Research
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: DIABAID
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: INEBA
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: Hospital Español
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aires
country: Argentina
facility: Hospital Británico de Buenos Aires
state: Buenos Aires
city: Ciudad de Córdoba
country: Argentina
facility: ILAIM
state: Córdoba
city: Ciudad de Mendoza
country: Argentina
facility: Fundación Scherbovsky
state: Mendoza
city: Ciudad De Santa Fe
country: Argentina
facility: Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )
state: Santa Fe
city: Edmonton
country: Canada
facility: Edmonton Kaye Clinic
state: Alberta
city: Greenfield Park
country: Canada
facility: Recherche Sepmus inc
state: Quebec
city: Montréal
country: Canada
facility: Centre de recherché du Centre Hospitalier de l'Universite de Montreal (CRCHUM)
state: Quebec
city: Montréal
country: Canada
facility: Montreal Neurological Institute & Hospital
state: Quebec
city: Santiago
country: Chile
facility: Centro de Trastornos del Movimiento (CETRAM)
state: Región Metropolitana
city: Santiago
country: Chile
facility: Clinica Dávila
state: Región Metropolitana
city: Santiago
country: Chile
facility: Biomedica Research Group AV Salvador 149, oficina 1101
city: Cali
country: Colombia
facility: Centro de Investigaciones Clínicas SAS
city: Saltillo
country: Mexico
facility: Clinical Research Institute Saltillo S.A. de C.V.
state: Coahuila
city: Monterrey
country: Mexico
facility: Hospital Universitario "Dr. José Eleuterio González"
state: Nuevo Leon
city: Querétaro City
country: Mexico
facility: SMIQ BRCR Global México
state: Querétaro
city: San Lucas Tepetlacalco
country: Mexico
facility: Clinical Research Institute S.C.
state: Tlalnepantla De Baz
city: Boca Del Río
country: Mexico
facility: Centro Especializado en Investigación Clínica S.C.
state: Veracruz
city: Mexico City
country: Mexico
facility: Phylasis Clinicas Research
city: Veracruz
country: Mexico
facility: FAICIC Clinical Researc
city: Arequipa
country: Peru
facility: Hospital Nivel IV Carlos Alberto Seguin Escobedo
city: Callao
country: Peru
facility: Hospital Nacional IV Alberto Sabogal Sologuren
city: Lima
country: Peru
facility: Hospital Almenara
city: Lima
country: Peru
facility: Instituto Neuro Cardiovascular de las Américas
city: Lima
country: Peru
facility: Hospital Nacional Cayetano Heredia
Study officials:
affiliation: Mallinckrodt
name: Clinical Team Leader
role: STUDY_DIRECTOR
References: