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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03068754
• Date of registration: 23/02/2017
• Prospective Registration: Yes
• Primary sponsor: Mallinckrodt
• Public title: Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
• Scientific 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
• Date of first enrolment: June 22, 2017
• Target sample size: 143
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03068754
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2/Phase 3

Countries of recruitment

Argentina, Canada, Chile, Colombia, Mexico, Peru, United States

Contacts

• Name: Clinical Team Leader
• Affiliation: Mallinckrodt

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Placebo

Drug: Acthar

Primary Outcome(s)

Number of Participants Experiencing an Adverse Event During the Treatment Period [Time Frame: by the end of the treatment period (within 36 Weeks)]

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)]

Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [Time Frame: Baseline, Week 36]

Secondary Outcome(s)

Treatment Period: Spirometry (%) [Time Frame: Baseline, Week 36]

Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R [Time Frame: Baseline, Week 84]

Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R [Time Frame: Baseline, Week 36]

Secondary ID(s)

MNK14042068

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/09/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03068754