Trial Outcomes & Findings for A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis (NCT NCT01906658)
NCT ID: NCT01906658
Last Updated: 2017-01-06
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
43 participants
Primary outcome timeframe
Baseline to Week 8
Results posted on
2017-01-06
Participant Flow
Participant milestones
| Measure |
Acthar 80 U (1.0 mL) Subcutaneous (SC) Twice Weekly
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 24 U (0.3 mL) SC Daily
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 56 U (0.7 mL) SC Twice Weekly
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 16 U (0.2 mL) SC Daily
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 Study
STARTED
|
11
|
11
|
10
|
11
|
|
Overall Study
COMPLETED
|
9
|
10
|
7
|
11
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
3
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
Baseline characteristics by cohort
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 Participants
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 24 U (0.3 mL) SC Daily
n=11 Participants
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 56 U (0.7 mL) SC Twice Weekly
n=10 Participants
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 16 U (0.2 mL) SC Daily
n=11 Participants
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
n=43 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
26 Participants
n=31 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
17 Participants
n=31 Participants
|
|
Gender
Female
|
4 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
4 Participants
n=7 Participants
|
17 Participants
n=31 Participants
|
|
Gender
Male
|
7 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
26 Participants
n=31 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=99 Participants
|
11 participants
n=107 Participants
|
10 participants
n=206 Participants
|
11 participants
n=7 Participants
|
43 participants
n=31 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 8Outcome measures
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 Participants
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 24 U (0.3 mL) SC Daily
n=11 Participants
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 56 U (0.7 mL) SC Twice Weekly
n=10 Participants
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 16 U (0.2 mL) SC Daily
n=11 Participants
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
|
|---|---|---|---|---|
|
Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication
|
2 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 8Outcome measures
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 Participants
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 24 U (0.3 mL) SC Daily
n=11 Participants
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 56 U (0.7 mL) SC Twice Weekly
n=10 Participants
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 16 U (0.2 mL) SC Daily
n=11 Participants
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
|
|---|---|---|---|---|
|
Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation
|
2 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 8Outcome measures
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 Participants
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 24 U (0.3 mL) SC Daily
n=11 Participants
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 56 U (0.7 mL) SC Twice Weekly
n=10 Participants
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 16 U (0.2 mL) SC Daily
n=11 Participants
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
|
|---|---|---|---|---|
|
Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 36Outcome measures
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 Participants
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 24 U (0.3 mL) SC Daily
n=11 Participants
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 56 U (0.7 mL) SC Twice Weekly
n=10 Participants
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 16 U (0.2 mL) SC Daily
n=11 Participants
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
|
|---|---|---|---|---|
|
Proportion of Subjects With Treatment Emergent Suicidality
|
1 Participants
|
3 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
Acthar 80 U (1.0 mL) SC Twice Weekly
Serious events: 2 serious events
Other events: 11 other events
Deaths: 0 deaths
Acthar 24 U (0.3 mL) SC Daily
Serious events: 3 serious events
Other events: 11 other events
Deaths: 0 deaths
Acthar 56 U (0.7 mL) SC Twice Weekly
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Acthar 16 U (0.2 mL) SC Daily
Serious events: 1 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 participants at risk
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 24 U (0.3 mL) SC Daily
n=11 participants at risk
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 56 U (0.7 mL) SC Twice Weekly
n=10 participants at risk
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 16 U (0.2 mL) SC Daily
n=11 participants at risk
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
|
|---|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory arrest
|
9.1%
1/11 • Number of events 1
|
0.00%
0/11
|
0.00%
0/10
|
0.00%
0/11
|
|
Nervous system disorders
Seizures
|
9.1%
1/11 • Number of events 1
|
0.00%
0/11
|
0.00%
0/10
|
0.00%
0/11
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
9.1%
1/11 • Number of events 1
|
0.00%
0/11
|
0.00%
0/10
|
0.00%
0/11
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/11
|
18.2%
2/11 • Number of events 2
|
10.0%
1/10 • Number of events 1
|
0.00%
0/11
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/11
|
9.1%
1/11 • Number of events 1
|
0.00%
0/10
|
0.00%
0/11
|
|
Injury, poisoning and procedural complications
Feeding tube complication
|
0.00%
0/11
|
0.00%
0/11
|
10.0%
1/10 • Number of events 1
|
0.00%
0/11
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.00%
0/11
|
0.00%
0/11
|
0.00%
0/10
|
9.1%
1/11 • Number of events 1
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/11
|
0.00%
0/11
|
0.00%
0/10
|
9.1%
1/11 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
9.1%
1/11 • Number of events 1
|
0.00%
0/11
|
0.00%
0/10
|
0.00%
0/11
|
Other adverse events
| Measure |
Acthar 80 U (1.0 mL) SC Twice Weekly
n=11 participants at risk
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 24 U (0.3 mL) SC Daily
n=11 participants at risk
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 56 U (0.7 mL) SC Twice Weekly
n=10 participants at risk
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 16 U (0.2 mL) SC Daily
n=11 participants at risk
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
|
|---|---|---|---|---|
|
General disorders
Edema, peripheral
|
36.4%
4/11
|
18.2%
2/11
|
20.0%
2/10
|
54.5%
6/11
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
36.4%
4/11
|
27.3%
3/11
|
50.0%
5/10
|
36.4%
4/11
|
|
Nervous system disorders
Headache
|
0.00%
0/11
|
27.3%
3/11
|
50.0%
5/10
|
36.4%
4/11
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
18.2%
2/11
|
9.1%
1/11
|
20.0%
2/10
|
45.5%
5/11
|
|
Injury, poisoning and procedural complications
Fall
|
36.4%
4/11
|
9.1%
1/11
|
20.0%
2/10
|
18.2%
2/11
|
|
General disorders
Asthenia
|
36.4%
4/11
|
9.1%
1/11
|
10.0%
1/10
|
18.2%
2/11
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.1%
1/11
|
0.00%
0/11
|
40.0%
4/10
|
27.3%
3/11
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
27.3%
3/11
|
9.1%
1/11
|
30.0%
3/10
|
18.2%
2/11
|
|
Psychiatric disorders
Anxiety
|
27.3%
3/11
|
0.00%
0/11
|
20.0%
2/10
|
9.1%
1/11
|
|
General disorders
Edema
|
18.2%
2/11
|
27.3%
3/11
|
10.0%
1/10
|
0.00%
0/11
|
|
Nervous system disorders
Dizziness
|
0.00%
0/11
|
9.1%
1/11
|
20.0%
2/10
|
27.3%
3/11
|
|
Eye disorders
Vision blurred
|
9.1%
1/11
|
18.2%
2/11
|
0.00%
0/10
|
27.3%
3/11
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/11
|
9.1%
1/11 • Number of events 1
|
30.0%
3/10 • Number of events 3
|
9.1%
1/11 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Muscle atrophy
|
0.00%
0/11
|
0.00%
0/11
|
30.0%
3/10
|
18.2%
2/11
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER