Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
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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 |
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Recruitment Status :
Terminated
(As recommended by the study's independent Data and Safety Monitoring Board (DSMB))
First Posted : March 3, 2017
Results First Posted : September 3, 2020
Last Update Posted : October 14, 2020
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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 |
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.
| 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 |
| Actual Primary Completion Date : | November 25, 2019 |
| Actual Study Completion Date : | November 25, 2019 |
| Arm | Intervention/treatment |
|---|---|
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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:
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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:
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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.
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Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
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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.
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Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
Drug: Placebo Matching placebo for subcutaneous injection
Other Names:
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- Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: Baseline, 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 (summed) score of 48. Higher scores represent better function.
- Number of Participants Experiencing an Adverse Event During the Treatment Period [ Time Frame: by the end of the treatment period (within 36 Weeks) ]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.
- Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period [ Time Frame: by the time of database lock (within 84 weeks) ]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.
- Treatment Period: Spirometry (%) [ Time Frame: Baseline, Week 36 ]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)].
- Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R [ Time Frame: Baseline, 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 (summed) score of 48. Higher scores represent better function.
- Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R [ Time Frame: Baseline, Week 84 ]
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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
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
| Study Director: | Clinical Team Leader | Mallinckrodt |
Documents provided by Mallinckrodt:
| Responsible Party: | Mallinckrodt |
| ClinicalTrials.gov Identifier: | NCT03068754 |
| Other Study ID Numbers: |
MNK14042068 |
| First Posted: | March 3, 2017 Key Record Dates |
| Results First Posted: | September 3, 2020 |
| Last Update Posted: | October 14, 2020 |
| Last Verified: | September 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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ALS |
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Amyotrophic Lateral Sclerosis Adrenocorticotropic Hormone Motor Neuron Disease Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases Neuromuscular Diseases |
Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs |