Trial · Interventional

TerminatedPhase 2/3Results postedRegistry data

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

NCTNCT03068754Other idMNK14042068

Open at ClinicalTrials.govAsk about this trial

This trial was stopped early.

Reason given at the registry: As recommended by the study's independent Data and Safety Monitoring Board (DSMB)

Results were posted for this trial.

Overview

Status: Terminated
Phase: Phase 2/3
Enrollment: 143 participants (actual)
Sponsor: Mallinckrodt · Industry
Design: Randomized · Double masking
Countries: 7 countries
United States, Argentina, Canada, Chile, Colombia, Mexico, Peru
Start: 22 Jun 2017
Primary completion: 25 Nov 2019
Completion: 25 Nov 2019
First posted: 3 Mar 2017
Last update posted: 14 Oct 2020
Conditions: Amyotrophic Lateral Sclerosis

Timeline

1 trial · 1 stopped early · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2017201820192020

Ph 2/3
Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
NCT03068754
22 Jun 2017 to 25 Nov 2019
Terminated
Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

ActharDrug
PlaceboDrug

Arms4

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.
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.
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.
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.

Primary outcomes3

Outcomes the sponsor listed as primary.

Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time frame · Baseline, Week 36
Registry description
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)
Registry description
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)
Registry description
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 outcomes(3)

Treatment Period: Spirometry (%)
Time frame · Baseline, Week 36
Registry description
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
Registry description
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
Registry description
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.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· Baseline, Week 36

Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

Group	Value (score on a scale)	Spread
Arm A: RTP Acthar Gel	34.7	6.01
Arm B: RTP Placebo	34.3	5.84

Primary outcomeCount of participants· by the end of the treatment period (within 36 Weeks)

Number of Participants Experiencing an Adverse Event During the Treatment Period

Group	Value (Participants)
Arm A: RTP Acthar Gel	13
Arm B: RTP Placebo	6

Primary outcomeCount of participants· by the time of database lock (within 84 weeks)

Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period

Group	Value (Participants)
Arm C: OLE Acthar Gel-Acthar Gel	4
Arm D: OLE Placebo-Acthar Gel	2

Secondary and other outcome results(3)

Secondary outcomeMean· Baseline, Week 36

Treatment Period: Spirometry (%)

Group	Value (liters)	Spread
Arm A: RTP Acthar Gel	85.0	18.31
Arm B: RTP Placebo	82.1	17.60

Secondary outcomeMean· Baseline, Week 36

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

Group	Value (score on a scale)	Spread
Arm A: RTP Acthar Gel	35.8	6.05
Arm B: RTP Placebo	35.4	6.23

Secondary outcomeMean· Baseline, Week 84

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

Group	Value (units on a scale)	Spread
Arm C: OLE Acthar Gel-Acthar Gel	36.8	5.33
Arm D: OLE Placebo-Acthar Gel	39.6	1.81

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
Arm A: RTP Acthar GelParticipants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), 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.	2 / 95	13 / 95	71 / 95
Arm B: RTP PlaceboParticipants 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.	3 / 47	6 / 47	34 / 47
Arm C: OLE Acthar Gel-Acthar GelParticipants who receive Acthar Gel 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 Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.	1 / 25	4 / 25	18 / 25
Arm D: OLE Placebo-Acthar GelParticipants 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.	2 / 8	2 / 8	7 / 8

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: from 18 Years up to 75 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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

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