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

NCT03068754 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: MNK14042068
• Study Type: interventional
• Target: 143
• Locations: 7 countries
• Sites: 69

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS

Outcome Measures

Primary Outcomes (3)

• 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

• 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)

• 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 Outcomes (3)

• Treatment Period: Spirometry (%)

  Baseline, Week 36

• Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R

  Baseline, Week 36

• Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R

  Baseline, Week 84

Study Arms (4)

Arm A: Treatment Period Acthar

EXPERIMENTAL

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.

Drug: Acthar

Arm B: Treatment Period Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Arm C: Extension Period Acthar-Acthar

EXPERIMENTAL

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.

Drug: Acthar

Arm D: Extension Period Placebo-Acthar

EXPERIMENTAL

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.

Drug: Acthar; Drug: Placebo

Interventions

Acthar DRUG

Repository corticotropin for subcutaneous injection

Also known as: Trade name: Acthar® Gel, Generic name: repository corticotropin injection

Arm A: Treatment Period Acthar; Arm C: Extension Period Acthar-Acthar; Arm D: Extension Period Placebo-Acthar

Placebo DRUG

Matching placebo for subcutaneous injection

Also known as: Matching Placebo, Reference Product

Arm B: Treatment Period Placebo; Arm D: Extension Period Placebo-Acthar

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mallinckrodt: lead

Study Sites (69)

Neuromuscular Research Center

Phoenix, Arizona, 85028, United States

Location

Mayo Clinic - Arizona

Scottsdale, Arizona, 85259, United States

Location

University of California San Diego

La Jolla, California, 92037, United States

Location

Loma Linda University Health System, Department of Neurology

Loma Linda, California, 92354, United States

Location

Keck School of Medicine, University of Southern California

Los Angeles, California, 90033, United States

Location

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

University of California Irvine Medical Center

Orange, California, 92868, United States

Location

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Colorado Springs Neurological Associates

Colorado Springs, Colorado, 80907, United States

Location

Georgetown University

Washington D.C., District of Columbia, 20007, United States

Location

George Washington University

Washington D.C., District of Columbia, 20037, United States

Location

University of Florida - McKnight Brain Institute

Gainesville, Florida, 32611, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Indiana University-Neuroscience Center of Excellence/Goodman Hall

Indianapolis, Indiana, 46202, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Kentucky Chandler Medical Center

Lexington, Kentucky, 40536, United States

Location

John Hopkins Outpatient Center

Baltimore, Maryland, 21287, United States

Location

University of Massachusetts Medical School

Worcester, Massachusetts, 01655, United States

Location

Mercy Health- Saint Mary's

Grand Rapids, Michigan, 49503, United States

Location

Neurology Associates

Lincoln, Nebraska, 68510, United States

Location

University of Nebraska Medical Center - Physicians Clinical Neurosciences Center

Omaha, Nebraska, 68198, United States

Location

Las Vegas Clinic

Las Vegas, Nevada, 89145, United States

Location

Jersey Shore University Medical Center

Neptune City, New Jersey, 07753, United States

Location

Columbia Presbyterian Hospital

New York, New York, 10032, United States

Location

Providence ALS Center

Portland, Oregon, 97213, United States

Location

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Temple University School of Medicine

Philadelphia, Pennsylvania, 19140, United States

Location

Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

Wesley Neurology Clinic

Cordova, Tennessee, 38018, United States

Location

Austin Neuromuscular Center

Austin, Texas, 78756, United States

Location

Texas Neurology, P.A.

Dallas, Texas, 75214, United States

Location

The Methodist Hospital

Houston, Texas, 77030, United States

Location

University of Vermont Medical Center

Colchester, Vermont, 05401, United States

Location

VCU Medical Center

Richmond, Virginia, 23298, United States

Location

Swedish Neuroscience Institute

Seattle, Washington, 98122, United States

Location

Medical College of Wisconsin/Froedtert Hospital

Milwaukee, Wisconsin, 53226, United States

Location

IADIN

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1015ABR, Argentina

Location

STAT Research

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1023AAB, Argentina

Location

DIABAID

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1061ABD, Argentina

Location

INEBA

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1192AAW, Argentina

Location

Hospital Italiano de Buenos Aires

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1199ABB, Argentina

Location

Hospital Español

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1209AAB, Argentina

Location

Hospital Británico de Buenos Aires

Ciudad Autonoma de Buenos Aires, Buenos Aires, C1280AEB, Argentina

Location

ILAIM

Córdoba, Córdoba Province, X5000BNB, Argentina

Location

Fundación Scherbovsky

Mendoza, Mendoza Province, CP 5500, Argentina

Location

Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )

Santa Fe, Santa Fe Province, S3000ASL, Argentina

Location

Edmonton Kaye Clinic

Edmonton, Alberta, T6G 1Z1, Canada

Location

Recherche Sepmus inc

Greenfield Park, Quebec, J4V 2J2, Canada

Location

Centre de recherché du Centre Hospitalier de l'Universite de Montreal (CRCHUM)

Montreal, Quebec, H2XOA9, Canada

Location

Montreal Neurological Institute & Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

Centro de Trastornos del Movimiento (CETRAM)

Santiago, Santiago Metropolitan, 8380815, Chile

Location

Clinica Dávila

Santiago, Santiago Metropolitan, 8431657, Chile

Location

Biomedica Research Group AV Salvador 149, oficina 1101

Santiago, 7500710, Chile

Location

Centro de Investigaciones Clínicas SAS

Cali, 760036, Colombia

Location

Clinical Research Institute Saltillo S.A. de C.V.

Saltillo, Coahuila, 25020, Mexico

Location

Hospital Universitario "Dr. José Eleuterio González"

Monterrey, Nuevo León, 64460, Mexico

Location

SMIQ BRCR Global México

Querétaro City, Querétaro, 76090, Mexico

Location

Clinical Research Institute S.C.

San Lucas Tepetlacalco, Tlalnepantla De Baz, 54055, Mexico

Location

Centro Especializado en Investigación Clínica S.C.

Boca del Río, Veracruz, 94290, Mexico

Location

Phylasis Clinicas Research

Mexico City, 54769, Mexico

Location

FAICIC Clinical Researc

Veracruz, 91900, Mexico

Location

Hospital Nivel IV Carlos Alberto Seguin Escobedo

Arequipa, 04001, Peru

Location

Hospital Nacional IV Alberto Sabogal Sologuren

Callao, 07016, Peru

Location

Hospital Almenara

Lima, 15033, Peru

Location

Instituto Neuro Cardiovascular de las Américas

Lima, 15074, Peru

Location

Hospital Nacional Cayetano Heredia

Lima, 15102, Peru

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Adrenocorticotropic Hormone

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Melanocortins; Pro-Opiomelanocortin; Hypothalamic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pituitary Hormones, Anterior; Pituitary Hormones; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Nerve Tissue Proteins; Proteins

Results Point of Contact

• Title: Medical Information Call Center
• Organization: Mallinckrodt

clinicaltrials@mnk.com

800-556-3314

Study Officials

• Clinical Team Leader

  Mallinckrodt

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: The Treatment Period is defined as Randomized (2:1, Arm A: Arm B), Double-blind (with care provider and outcomes assessor also blinded). The Extension Period has no masking, because all who participate receive open label study drug.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: February 23, 2017
• First Posted: March 3, 2017
• Study Start: June 22, 2017
• Primary Completion: November 25, 2019
• Study Completion: November 25, 2019
• Last Updated: October 14, 2020
• Results First Posted: September 3, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will not share

Locations

ME (7); US (39); CA (4); AR (10); CL (3); CO (1); PE (5)