Trial · NCT03068754

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

NCT03068754, Phase 2, Phase 3, Terminated, Mallinckrodt; results first posted 2020-09-03

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS). Trials, record NCT03068754. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03068754

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
NCT number: NCT03068754
Status: Terminated
Phases: Phase 2, Phase 3
Enrollment: 143 participants, actual
Lead sponsor: Mallinckrodt
Conditions: Amyotrophic Lateral Sclerosis
Interventions: Acthar, Placebo
First posted: 2017-03-03
Last updated: 2020-10-14
Results first posted: 2020-09-03

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	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.
Primary	Number of Participants Experiencing an Adverse Event During the Treatment Period	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.
Primary	Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period	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.
Secondary	Treatment Period: Spirometry (%)	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 \[(Forced Expiratory Volume in one second (FEV1)\].
Secondary	Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R	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.
Secondary	Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R	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.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	Baseline, Week 36	Safety Population	score on a scale	MEAN
PRIMARY	Number of Participants Experiencing an Adverse Event During the Treatment Period	by the end of the treatment period (within 36 Weeks)	Safety Population	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period	by the time of database lock (within 84 weeks)	Safety Population	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Treatment Period: Spirometry (%)	Baseline, Week 36	Safety population	liters	MEAN
SECONDARY	Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R	Baseline, Week 36	Safety Population, with an actual score at the given time point	score on a scale	MEAN
SECONDARY	Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R	Baseline, Week 84	OLE population with scores at the given week; baseline is defined as the value at randomization (week 0)	units on a scale	MEAN

Source ClinicalTrials.gov

Results by group30

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	Arm A: RTP Acthar Gel	Baseline	34.7	Standard Deviation 6.01	score on a scale
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	Arm B: RTP Placebo	Baseline	34.3	Standard Deviation 5.84	score on a scale
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	Arm A: RTP Acthar Gel	Week 36	26.4	Standard Deviation 9.34	score on a scale
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)	Arm B: RTP Placebo	Week 36	30.8	Standard Deviation 7.55	score on a scale
Number of Participants Experiencing an Adverse Event During the Treatment Period	Arm A: RTP Acthar Gel	Serious Treatment Emergent Adverse Events	13		Participants
Number of Participants Experiencing an Adverse Event During the Treatment Period	Arm B: RTP Placebo	Serious Treatment Emergent Adverse Events	6		Participants
Number of Participants Experiencing an Adverse Event During the Treatment Period	Arm A: RTP Acthar Gel	Non-serious Treatment-Emergent Adverse Events	74		Participants
Number of Participants Experiencing an Adverse Event During the Treatment Period	Arm B: RTP Placebo	Non-serious Treatment-Emergent Adverse Events	40		Participants
Number of Participants Experiencing an Adverse Event During the Treatment Period	Arm A: RTP Acthar Gel	Death	2		Participants
Number of Participants Experiencing an Adverse Event During the Treatment Period	Arm B: RTP Placebo	Death	3		Participants

1 to 10 of 30Source ClinicalTrials.gov

See all 30 results by group

Participant flow64

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Randomized Treatment Period (RTP)	STARTED	Arm A: Randomized Treatment Period (RTP) Acthar Gel	95
Randomized Treatment Period (RTP)	STARTED	Arm B: RTP Placebo	48
Randomized Treatment Period (RTP)	STARTED	Arm C: Open Label Extension Period (OLE) Acthar Gel-Acthar Gel	0
Randomized Treatment Period (RTP)	STARTED	Arm D: OLE Placebo-Acthar Gel	0
Randomized Treatment Period (RTP)	Intention to Treat (ITT) Population	Arm A: Randomized Treatment Period (RTP) Acthar Gel	95
Randomized Treatment Period (RTP)	Intention to Treat (ITT) Population	Arm B: RTP Placebo	48
Randomized Treatment Period (RTP)	Intention to Treat (ITT) Population	Arm C: Open Label Extension Period (OLE) Acthar Gel-Acthar Gel	0
Randomized Treatment Period (RTP)	Intention to Treat (ITT) Population	Arm D: OLE Placebo-Acthar Gel	0
Randomized Treatment Period (RTP)	Safety Population	Arm A: Randomized Treatment Period (RTP) Acthar Gel	95
Randomized Treatment Period (RTP)	Safety Population	Arm B: RTP Placebo	47

1 to 10 of 64Source ClinicalTrials.gov

See all 64 participant flow

Adverse events384

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Constipation	Gastrointestinal disorders	Arm A: RTP Acthar Gel	13	95	15
other	Constipation	Gastrointestinal disorders	Arm B: RTP Placebo	4	47	5
other	Constipation	Gastrointestinal disorders	Arm C: OLE Acthar Gel-Acthar Gel	2	25	3
other	Constipation	Gastrointestinal disorders	Arm D: OLE Placebo-Acthar Gel	2	8	2
other	Dry Mouth	Gastrointestinal disorders	Arm A: RTP Acthar Gel	5	95	5
other	Dry Mouth	Gastrointestinal disorders	Arm B: RTP Placebo	0	47	0
other	Dry Mouth	Gastrointestinal disorders	Arm C: OLE Acthar Gel-Acthar Gel	1	25	1
other	Dry Mouth	Gastrointestinal disorders	Arm D: OLE Placebo-Acthar Gel	1	8	1
other	Dysphagia	Gastrointestinal disorders	Arm A: RTP Acthar Gel	5	95	5
other	Dysphagia	Gastrointestinal disorders	Arm B: RTP Placebo	5	47	5

1 to 10 of 384Source ClinicalTrials.gov

See all 384 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Arm A: RTP Acthar Gel	2	95
Total, all-cause mortality	Arm B: RTP Placebo	3	47
Total, all-cause mortality	Arm C: OLE Acthar Gel-Acthar Gel	1	25
Total, all-cause mortality	Arm D: OLE Placebo-Acthar Gel	2	8
Total, other adverse events	Arm A: RTP Acthar Gel	71	95
Total, other adverse events	Arm B: RTP Placebo	34	47
Total, other adverse events	Arm C: OLE Acthar Gel-Acthar Gel	18	25
Total, other adverse events	Arm D: OLE Placebo-Acthar Gel	7	8
Total, serious adverse events	Arm A: RTP Acthar Gel	13	95
Total, serious adverse events	Arm B: RTP Placebo	6	47

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03068754

GET https://conductscience.org/api/trials/v1/NCT03068754/results?page=2

API reference For AI assistants