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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03068754
Recruitment Status : Active, not recruiting
First Posted : March 3, 2017
Last Update Posted : July 22, 2019
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Acthar Drug: Placebo Phase 2 Phase 3

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 143 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
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.
Masking: Double (Participant, Investigator)
Masking Description:

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.

Primary Purpose: Treatment
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
Actual Study Start Date : June 22, 2017
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : October 2021


Arm Intervention/treatment
Experimental: Arm A: Treatment Period 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.

Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
  • Trade name: Acthar® Gel
  • Generic name: repository corticotropin injection

Placebo Comparator: Arm B: Treatment Period 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.

Drug: Placebo
Matching placebo for subcutaneous injection
Other Names:
  • Matching Placebo
  • Reference Product

Experimental: Arm C: Extension Period 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.
Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
  • Trade name: Acthar® Gel
  • Generic name: repository corticotropin injection

Experimental: Arm D: Extension Period Placebo-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.
Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
  • Trade name: Acthar® Gel
  • Generic name: repository corticotropin injection

Drug: Placebo
Matching placebo for subcutaneous injection
Other Names:
  • Matching Placebo
  • Reference Product




Primary Outcome Measures :
  1. Treatment Period: Scores on a scale for telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: Baseline through 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 score of 48. Higher scores represent better function.


  2. Number of participants experiencing an adverse event during the Treatment Period [ Time Frame: by the end of the treatment period (within approximately 2 years) ]
    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). Clinically significant changes in safety measures are recorded as adverse events.

  3. Number of participants experiencing an adverse event by the end of the Trial [ Time Frame: by the time of database lock (within approximately 4 years) ]
    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 :
  1. Treatment Period: Spirometry (%) [ Time Frame: Baseline through Week 36 ]
    The trained investigator (or designee) administers pulmonary function tests Categories: Forced volume capacity (FVC), forced expiratory volume in 1 second (FEV1)

  2. Treatment Period: Scores on a scale for investigator-administered ALSFRS-R [ Time Frame: Baseline through 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 score of 48. Higher scores represent better function.


  3. Extension Period: Scores on a scale for investigator-administered ALSFRS-R [ Time Frame: during the extension period (within approximately 2 years) ]

    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.




Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03068754


  Show 69 Study Locations
Sponsors and Collaborators
Mallinckrodt
Investigators
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Study Director: Clinical Team Leader Mallinckrodt

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Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT03068754     History of Changes
Other Study ID Numbers: MNK14042068
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: July 22, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Mallinckrodt:
ALS

Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs