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Amyotrophic Lateral SclerosisPhase 2/3NCT03068754

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

A Phase 2/3 study testing Acthar in people with Amyotrophic Lateral Sclerosis, run by Mallinckrodt at 69 sites in 7 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

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). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Acthar
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
143 participants
Where
69 sitesin 7 countries
Study dates
22 Jun 2017to 25 Nov 2019
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

69 sites in 7 countries.

69 sites in 7 countries

United States39 sites
  • Emory University

    Atlanta, Georgia, 30322

  • Augusta University

    Augusta, Georgia, 30912

  • Austin Neuromuscular Center

    Austin, Texas, 78756

  • John Hopkins Outpatient Center

    Baltimore, Maryland, 21287

  • University of Vermont Medical Center

    Colchester, Vermont, 05401

  • Colorado Springs Neurological Associates

    Colorado Springs, Colorado, 80907

  • Wesley Neurology Clinic

    Cordova, Tennessee, 38018

  • Texas Neurology, P.A.

    Dallas, Texas, 75214

  • University of Florida - McKnight Brain Institute

    Gainesville, Florida, 32611

  • Mercy Health- Saint Mary's

    Grand Rapids, Michigan, 49503

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • The Methodist Hospital

    Houston, Texas, 77030

  • Indiana University-Neuroscience Center of Excellence/Goodman Hall

    Indianapolis, Indiana, 46202

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • University of California San Diego

    La Jolla, California, 92037

  • Las Vegas Clinic

    Las Vegas, Nevada, 89145

  • University of Kentucky Chandler Medical Center

    Lexington, Kentucky, 40536

  • Neurology Associates

    Lincoln, Nebraska, 68510

  • Loma Linda University Health System, Department of Neurology

    Loma Linda, California, 92354

  • Keck School of Medicine, University of Southern California

    Los Angeles, California, 90033

  • University of California Los Angeles

    Los Angeles, California, 90095

  • Medical College of Wisconsin/Froedtert Hospital

    Milwaukee, Wisconsin, 53226

  • Jersey Shore University Medical Center

    Neptune City, New Jersey, 07753

  • Columbia Presbyterian Hospital

    New York, New York, 10032

  • University of Nebraska Medical Center - Physicians Clinical Neurosciences Center

    Omaha, Nebraska, 68198

  • University of California Irvine Medical Center

    Orange, California, 92868

  • Temple University School of Medicine

    Philadelphia, Pennsylvania, 19140

  • Neuromuscular Research Center

    Phoenix, Arizona, 85028

  • Allegheny General Hospital

    Pittsburgh, Pennsylvania, 15212

  • Providence ALS Center

    Portland, Oregon, 97213

  • VCU Medical Center

    Richmond, Virginia, 23298

  • California Pacific Medical Center

    San Francisco, California, 94115

  • University of California San Francisco

    San Francisco, California, 94143

  • Mayo Clinic - Arizona

    Scottsdale, Arizona, 85259

  • Swedish Neuroscience Institute

    Seattle, Washington, 98122

  • University of South Florida

    Tampa, Florida, 33612

  • Georgetown University

    Washington D.C., District of Columbia, 20007

  • George Washington University

    Washington D.C., District of Columbia, 20037

  • University of Massachusetts Medical School

    Worcester, Massachusetts, 01655

Other trials in United States

Argentina10 sites
  • IADIN

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1015ABR

  • STAT Research

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1023AAB

  • DIABAID

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1061ABD

  • INEBA

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1192AAW

  • Hospital Italiano de Buenos Aires

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1199ABB

  • Hospital Español

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1209AAB

  • Hospital Británico de Buenos Aires

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1280AEB

  • ILAIM

    Córdoba, Córdoba Province, X5000BNB

  • Fundación Scherbovsky

    Mendoza, Mendoza Province, CP 5500

  • Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )

    Santa Fe, Santa Fe Province, S3000ASL

Other trials in Argentina

Mexico7 sites
  • Centro Especializado en Investigación Clínica S.C.

    Boca del Río, Veracruz, 94290

  • Phylasis Clinicas Research

    Mexico City, 54769

  • Hospital Universitario "Dr. José Eleuterio González"

    Monterrey, Nuevo León, 64460

  • SMIQ BRCR Global México

    Querétaro City, Querétaro, 76090

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

    Saltillo, Coahuila, 25020

  • Clinical Research Institute S.C.

    San Lucas Tepetlacalco, Tlalnepantla De Baz, 54055

  • FAICIC Clinical Researc

    Veracruz, 91900

Other trials in Mexico

Peru5 sites
  • Hospital Nivel IV Carlos Alberto Seguin Escobedo

    Arequipa, 04001

  • Hospital Nacional IV Alberto Sabogal Sologuren

    Callao, 07016

  • Hospital Almenara

    Lima, 15033

  • Instituto Neuro Cardiovascular de las Américas

    Lima, 15074

  • Hospital Nacional Cayetano Heredia

    Lima, 15102

Other trials in Peru

Canada4 sites
  • Edmonton Kaye Clinic

    Edmonton, Alberta, T6G 1Z1

  • Recherche Sepmus inc

    Greenfield Park, Quebec, J4V 2J2

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

    Montreal, Quebec, H2XOA9

  • Montreal Neurological Institute & Hospital

    Montreal, Quebec, H3A 2B4

Other trials in Canada

Chile3 sites
  • Centro de Trastornos del Movimiento (CETRAM)

    Santiago, Santiago Metropolitan, 8380815

  • Clinica Dávila

    Santiago, Santiago Metropolitan, 8431657

  • Biomedica Research Group AV Salvador 149, oficina 1101

    Santiago, 7500710

Other trials in Chile

Colombia1 site
  • Centro de Investigaciones Clínicas SAS

    Cali, 760036

Other trials in Colombia

About this study

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

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.

Read the 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.

Being tested
Acthar, Placebo
Keywords
ALS
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ActharDrug

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

    Repository corticotropin for subcutaneous injection

  • PlaceboDrug

    Also called Matching Placebo, Reference Product

    Matching placebo for subcutaneous injection

Study groups in NCT03068754
Study groupRoleWhat they receive
Arm A: Treatment Period ActharExperimentalParticipants 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.
Receives: Drug: Acthar
Arm B: Treatment Period PlaceboPlacebo comparatorParticipants 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.
Receives: Drug: Placebo
Arm C: Extension Period Acthar-ActharExperimentalParticipants 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.
Receives: Drug: Acthar
Arm D: Extension Period Placebo-ActharExperimentalParticipants 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.
Receives: Drug: Acthar; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Baseline, Week 36

  2. 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.

    Measured: by the end of the treatment period (within 36 Weeks)

  3. 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.

    Measured: by the time of database lock (within 84 weeks)

Secondary outcomes (3)
  1. 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)].

    Measured: Baseline, Week 36

  2. 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.

    Measured: Baseline, Week 36

  3. 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.

    Measured: Baseline, Week 84

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Mar 2017
  2. Study start22 Jun 2017
  3. Primary completion25 Nov 2019
  4. Study completion25 Nov 2019

Why it stopped: As recommended by the study's independent Data and Safety Monitoring Board (DSMB)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
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.
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
143 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03068754
Sponsor's protocol ID
MNK14042068
Status last verified
Sep 2020
First submitted
23 Feb 2017
Last updated
14 Oct 2020
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Acthar in people with Amyotrophic Lateral Sclerosis, run by Mallinckrodt at 69 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 14 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 69 sites in United States, Argentina, Mexico, Peru, Canada and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) (time frame: Baseline, Week 36). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2020.

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