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

Mallinckrodt

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: Acthar

Study type: Interventional

Funder types: Industry

Identifiers

NCT03068754

MNK14042068

Details and patient eligibility

About

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.

Full 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.

Enrollment: 143 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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143 participants in 4 patient groups, including a placebo group

Arm A: Treatment Period Acthar

Experimental group

Description:

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.

Treatment:

Drug: Acthar

Arm B: Treatment Period Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo

Arm C: Extension Period Acthar-Acthar

Experimental group

Description:

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.

Treatment:

Drug: Acthar

Arm D: Extension Period Placebo-Acthar

Experimental group

Description:

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.

Treatment:

Drug: Acthar

Drug: Placebo