A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

• ClinicalTrials.gov Identifier: NCT01906658
• Recruitment Status: Completed
• First Posted: July 24, 2013
• Results First Posted: January 6, 2017
• Last Update Posted: January 6, 2017
• Sponsor: Mallinckrodt
• Information provided by (Responsible Party): Mallinckrodt

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis
• Intervention: Drug: Repository corticotropin injection
• Enrollment: 43

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Acthar 80 U (1.0 mL) Subcutaneous (SC) Twice Weekly	Acthar 24 U (0.3 mL) SC Daily	Acthar 56 U (0.7 mL) SC Twice Weekly	Acthar 16 U (0.2 mL) SC Daily
Arm/Group Description	Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Period Title: Overall Study
Started	11	11	10	11
Completed	9	10	7	11
Not Completed	2	1	3	0

Baseline Characteristics

Arm/Group Title		Acthar 80 U (1.0 mL) SC Twice Weekly	Acthar 24 U (0.3 mL) SC Daily	Acthar 56 U (0.7 mL) SC Twice Weekly	Acthar 16 U (0.2 mL) SC Daily	Total
Arm/Group Description		Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Total of all reporting groups
Overall Number of Baseline Participants		11	11	10	11	43
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	11 participants	11 participants	10 participants	11 participants	43 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	4 36.4%	7 63.6%	6 60.0%	9 81.8%	26 60.5%
	>=65 years	7 63.6%	4 36.4%	4 40.0%	2 18.2%	17 39.5%
Gender; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	11 participants	11 participants	10 participants	11 participants	43 participants
	Female	4 36.4%	5 45.5%	4 40.0%	4 36.4%	17 39.5%
	Male	7 63.6%	6 54.5%	6 60.0%	7 63.6%	26 60.5%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	11 participants	11 participants	10 participants	11 participants	43 participants
		11	11	10	11	43

Outcome Measures

1. Primary Outcome

Title	Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication
Description	[Not Specified]
Time Frame	Baseline to Week 8

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Acthar 80 U (1.0 mL) SC Twice Weekly	Acthar 24 U (0.3 mL) SC Daily	Acthar 56 U (0.7 mL) SC Twice Weekly	Acthar 16 U (0.2 mL) SC Daily
Arm/Group Description:	Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Overall Number of Participants Analyzed	11	11	10	11
Measure Type: Count of Participants; Unit of Measure: Participants
	2 18.2%	0 0.0%	2 20.0%	0 0.0%

2. Secondary Outcome

Title	Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation
Description	[Not Specified]
Time Frame	Baseline to Week 8

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Acthar 80 U (1.0 mL) SC Twice Weekly	Acthar 24 U (0.3 mL) SC Daily	Acthar 56 U (0.7 mL) SC Twice Weekly	Acthar 16 U (0.2 mL) SC Daily
Arm/Group Description:	Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Overall Number of Participants Analyzed	11	11	10	11
Measure Type: Count of Participants; Unit of Measure: Participants
	2 18.2%	0 0.0%	2 20.0%	0 0.0%

3. Secondary Outcome

Title	Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication
Description	[Not Specified]
Time Frame	Baseline to Week 8

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Acthar 80 U (1.0 mL) SC Twice Weekly	Acthar 24 U (0.3 mL) SC Daily	Acthar 56 U (0.7 mL) SC Twice Weekly	Acthar 16 U (0.2 mL) SC Daily
Arm/Group Description:	Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Overall Number of Participants Analyzed	11	11	10	11
Measure Type: Count of Participants; Unit of Measure: Participants
	0 0.0%	0 0.0%	0 0.0%	0 0.0%

4. Secondary Outcome

Title	Proportion of Subjects With Treatment Emergent Suicidality
Description	[Not Specified]
Time Frame	Baseline to Week 36

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Acthar 80 U (1.0 mL) SC Twice Weekly	Acthar 24 U (0.3 mL) SC Daily	Acthar 56 U (0.7 mL) SC Twice Weekly	Acthar 16 U (0.2 mL) SC Daily
Arm/Group Description:	Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks	Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Overall Number of Participants Analyzed	11	11	10	11
Measure Type: Count of Participants; Unit of Measure: Participants
	1 9.1%	3 27.3%	0 0.0%	0 0.0%

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Acthar 80 U (1.0 mL) SC Twice Weekly		Acthar 24 U (0.3 mL) SC Daily		Acthar 56 U (0.7 mL) SC Twice Weekly		Acthar 16 U (0.2 mL) SC Daily
Arm/Group Description	Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks		Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks		Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks		Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily Repository corticotropin injection: Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
All-Cause Mortality
	Acthar 80 U (1.0 mL) SC Twice Weekly		Acthar 24 U (0.3 mL) SC Daily		Acthar 56 U (0.7 mL) SC Twice Weekly		Acthar 16 U (0.2 mL) SC Daily
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--		--/--		--/--
Serious Adverse Events
	Acthar 80 U (1.0 mL) SC Twice Weekly		Acthar 24 U (0.3 mL) SC Daily		Acthar 56 U (0.7 mL) SC Twice Weekly		Acthar 16 U (0.2 mL) SC Daily
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/11 (18.18%)		3/11 (27.27%)		1/10 (10.00%)		1/11 (9.09%)
Cardiac disorders
Supraventricular tachycardia	0/11 (0.00%)	0	0/11 (0.00%)	0	0/10 (0.00%)	0	1/11 (9.09%)	1
Gastrointestinal disorders
Gastrointestinal haemorrhage	0/11 (0.00%)	0	0/11 (0.00%)	0	0/10 (0.00%)	0	1/11 (9.09%)	1
Infections and infestations
Pneumonia	1/11 (9.09%)	1	0/11 (0.00%)	0	0/10 (0.00%)	0	0/11 (0.00%)	0
Injury, poisoning and procedural complications
Feeding tube complication	0/11 (0.00%)	0	0/11 (0.00%)	0	1/10 (10.00%)	1	0/11 (0.00%)	0
Metabolism and nutrition disorders
Hypokalaemia	0/11 (0.00%)	0	1/11 (9.09%)	1	0/10 (0.00%)	0	0/11 (0.00%)	0
Nervous system disorders
Seizures	1/11 (9.09%)	1	0/11 (0.00%)	0	0/10 (0.00%)	0	0/11 (0.00%)	0
Respiratory, thoracic and mediastinal disorders
Respiratory arrest	1/11 (9.09%)	1	0/11 (0.00%)	0	0/10 (0.00%)	0	0/11 (0.00%)	0
Atelectasis	1/11 (9.09%)	1	0/11 (0.00%)	0	0/10 (0.00%)	0	0/11 (0.00%)	0
Respiratory failure	0/11 (0.00%)	0	2/11 (18.18%)	2	1/10 (10.00%)	1	0/11 (0.00%)	0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Acthar 80 U (1.0 mL) SC Twice Weekly		Acthar 24 U (0.3 mL) SC Daily		Acthar 56 U (0.7 mL) SC Twice Weekly		Acthar 16 U (0.2 mL) SC Daily
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	11/11 (100.00%)		11/11 (100.00%)		9/10 (90.00%)		11/11 (100.00%)
Eye disorders
Vision blurred	1/11 (9.09%)		2/11 (18.18%)		0/10 (0.00%)		3/11 (27.27%)
Gastrointestinal disorders
Salivary hypersecretion	3/11 (27.27%)		1/11 (9.09%)		3/10 (30.00%)		2/11 (18.18%)
Diarrhea	0/11 (0.00%)	0	1/11 (9.09%)	1	3/10 (30.00%)	3	1/11 (9.09%)	1
General disorders
Edema, peripheral	4/11 (36.36%)		2/11 (18.18%)		2/10 (20.00%)		6/11 (54.55%)
Asthenia	4/11 (36.36%)		1/11 (9.09%)		1/10 (10.00%)		2/11 (18.18%)
Edema	2/11 (18.18%)		3/11 (27.27%)		1/10 (10.00%)		0/11 (0.00%)
Injury, poisoning and procedural complications
Fall	4/11 (36.36%)		1/11 (9.09%)		2/10 (20.00%)		2/11 (18.18%)
Musculoskeletal and connective tissue disorders
Muscular weakness	4/11 (36.36%)		3/11 (27.27%)		5/10 (50.00%)		4/11 (36.36%)
Muscle spasms	2/11 (18.18%)		1/11 (9.09%)		2/10 (20.00%)		5/11 (45.45%)
Back pain	1/11 (9.09%)		0/11 (0.00%)		4/10 (40.00%)		3/11 (27.27%)
Muscle atrophy	0/11 (0.00%)		0/11 (0.00%)		3/10 (30.00%)		2/11 (18.18%)
Nervous system disorders
Headache	0/11 (0.00%)		3/11 (27.27%)		5/10 (50.00%)		4/11 (36.36%)
Dizziness	0/11 (0.00%)		1/11 (9.09%)		2/10 (20.00%)		3/11 (27.27%)
Psychiatric disorders
Anxiety	3/11 (27.27%)		0/11 (0.00%)		2/10 (20.00%)		1/11 (9.09%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Institution/Investigator shall not, without Sponsor's prior written consent, independently publish or otherwise disclose any results of the study until a Multi-Center Publication is published. If a Multi-Center Publication is not submitted for publication within the specified timeframe, Institution and Principal Investigator shall have the right to publish and present the results of Institution's and Principal Investigator's activities solely in accordance with the Sponsor's written provisions.

Results Point of Contact

• Name/Title: Lawrence Hill
• Organization: Mallinckrodt Pharmaceuticals
• Phone: 908-238-6370
• EMail: lawrence.hill@mallinckrodt.com

• Responsible Party: Mallinckrodt
• ClinicalTrials.gov Identifier: NCT01906658
• Other Study ID Numbers: QSC01-ALS-01
• First Submitted: July 15, 2013
• First Posted: July 24, 2013
• Results First Submitted: November 8, 2016
• Results First Posted: January 6, 2017
• Last Update Posted: January 6, 2017