Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03068754
• Other study ID #: MNK14042068
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: June 22, 2017
• Est. completion date: November 25, 2019

Study information

• Verified date: September 2020
• Source: Mallinckrodt
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Terminated
• Enrollment: 143
• Est. completion date: November 25, 2019
• Est. primary completion date: November 25, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Acthar
Repository corticotropin for subcutaneous injection
• Placebo
Matching placebo for subcutaneous injection

Locations

Country	Name	City	State
Argentina	DIABAID	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	Hospital Británico de Buenos Aires	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	Hospital Español	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	Hospital Italiano de Buenos Aires	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	IADIN	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	INEBA	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	STAT Research	Ciudad Autonoma de Buenos Aires	Buenos Aires
Argentina	ILAIM	Ciudad de Córdoba	Córdoba
Argentina	Fundación Scherbovsky	Ciudad de Mendoza	Mendoza
Argentina	Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )	Ciudad De Santa Fe	Santa Fe
Canada	Edmonton Kaye Clinic	Edmonton	Alberta
Canada	Recherche Sepmus inc	Greenfield Park	Quebec
Canada	Centre de recherché du Centre Hospitalier de l'Universite de Montreal (CRCHUM)	Montréal	Quebec
Canada	Montreal Neurological Institute & Hospital	Montréal	Quebec
Chile	Biomedica Research Group AV Salvador 149, oficina 1101	Santiago
Chile	Centro de Trastornos del Movimiento (CETRAM)	Santiago	Región Metropolitana
Chile	Clinica Dávila	Santiago	Región Metropolitana
Colombia	Centro de Investigaciones Clínicas SAS	Cali
Mexico	Centro Especializado en Investigación Clínica S.C.	Boca Del Río	Veracruz
Mexico	Phylasis Clinicas Research	Mexico City
Mexico	Hospital Universitario "Dr. José Eleuterio González"	Monterrey	Nuevo Leon
Mexico	SMIQ BRCR Global México	Querétaro City	Querétaro
Mexico	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
Peru	Hospital Nivel IV Carlos Alberto Seguin Escobedo	Arequipa
Peru	Hospital Nacional IV Alberto Sabogal Sologuren	Callao
Peru	Hospital Almenara	Lima
Peru	Hospital Nacional Cayetano Heredia	Lima
Peru	Instituto Neuro Cardiovascular de las Américas	Lima
United States	Emory University	Atlanta	Georgia
United States	Augusta University	Augusta	Georgia
United States	Austin Neuromuscular Center	Austin	Texas
United States	John Hopkins Outpatient Center	Baltimore	Maryland
United States	University of Vermont Medical Center	Colchester	Vermont
United States	Colorado Springs Neurological Associates	Colorado Springs	Colorado
United States	Wesley Neurology Clinic	Cordova	Tennessee
United States	Texas Neurology, P.A.	Dallas	Texas
United States	University of Florida - McKnight Brain Institute	Gainesville	Florida
United States	Mercy Health- Saint Mary's	Grand Rapids	Michigan
United States	Penn State Health Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	The Methodist Hospital	Houston	Texas
United States	Indiana University-Neuroscience Center of Excellence/Goodman Hall	Indianapolis	Indiana
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of California San Diego	La Jolla	California
United States	Las Vegas Clinic	Las Vegas	Nevada
United States	University of Kentucky Chandler Medical Center	Lexington	Kentucky
United States	Neurology Associates	Lincoln	Nebraska
United States	Loma Linda University Health System, Department of Neurology	Loma Linda	California
United States	Keck School of Medicine, University of Southern California	Los Angeles	California
United States	University of California Los Angeles	Los Angeles	California
United States	Medical College of Wisconsin/Froedtert Hospital	Milwaukee	Wisconsin
United States	Jersey Shore University Medical Center	Neptune	New Jersey
United States	Columbia Presbyterian Hospital	New York	New York
United States	University of Nebraska Medical Center - Physicians Clinical Neurosciences Center	Omaha	Nebraska
United States	University of California Irvine Medical Center	Orange	California
United States	Temple University School of Medicine	Philadelphia	Pennsylvania
United States	Neuromuscular Research Center	Phoenix	Arizona
United States	Allegheny General Hospital	Pittsburgh	Pennsylvania
United States	Providence ALS Center	Portland	Oregon
United States	VCU Medical Center	Richmond	Virginia
United States	California Pacific Medical Center	San Francisco	California
United States	University of California San Francisco	San Francisco	California
United States	Mayo Clinic - Arizona	Scottsdale	Arizona
United States	Swedish Neuroscience Institute	Seattle	Washington
United States	University of South Florida	Tampa	Florida
United States	George Washington University	Washington	District of Columbia
United States	Georgetown University	Washington	District of Columbia
United States	University of Massachusetts Medical School	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Mallinckrodt

Countries where clinical trial is conducted

United States,  Argentina,  Canada,  Chile,  Colombia,  Mexico,  Peru, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	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.	Baseline, Week 36
Primary	Number of Participants Experiencing an Adverse Event During the Treatment Period	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.	by the end of the treatment period (within 36 Weeks)
Primary	Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period	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.	by the time of database lock (within 84 weeks)
Secondary	Treatment Period: Spirometry (%)	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)].	Baseline, Week 36
Secondary	Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R	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.	Baseline, Week 36
Secondary	Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R	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.	Baseline, Week 84