Trial Outcomes & Findings for ACTH in Progressive Forms of MS (NCT NCT01950234)
NCT ID: NCT01950234
Last Updated: 2025-02-11
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
59 participants
Primary outcome timeframe
Month 36
Results posted on
2025-02-11
Participant Flow
Participant milestones
| Measure |
ACTH
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Overall Study
STARTED
|
29
|
30
|
|
Overall Study
COMPLETED
|
11
|
9
|
|
Overall Study
NOT COMPLETED
|
18
|
21
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
ACTH in Progressive Forms of MS
Baseline characteristics by cohort
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
Total
n=59 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
56.5 years
STANDARD_DEVIATION 9.3 • n=5 Participants
|
51.5 years
STANDARD_DEVIATION 10.2 • n=7 Participants
|
53.9 years
STANDARD_DEVIATION 9.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
58 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Month 36Outcome measures
| Measure |
ACTH
n=14 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=9 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Proportion of Patients Exhibiting a 20% Worsening in T25FW at 36 Months
|
3 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study noticing any changes in menstrual cycle or period.
Outcome measures
| Measure |
ACTH
n=12 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=11 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Menstrual Changes [Female]
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Month 36The number is a T-score. There is no maximum or minimum value. Mean change in average of 5 DEXA scan values dual-energy x-ray absorptiometry; measures bone mineral density. Higher numbers are better. A DEXA T-score has a mean of 0 and standard deviation of 1, meaning that a T-score represents how many standard deviations a person's bone density is away from the average bone density of a healthy young adult, with a score of 0 indicating average bone density. Higher scores are better, indicating better bone density. A T-score of -1 to +1 is considered normal bone density, a score between -1 and -2.5 indicates osteopenia (low bone mass), and a score of -2.5 or lower indicates osteoporosis.
Outcome measures
| Measure |
ACTH
n=14 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=10 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: DEXA Scans
|
-0.41 units on a scale
Standard Deviation 0.49
|
0 units on a scale
Standard Deviation 0.5
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that they bruise more easily
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Bruising
|
7 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that have noticed swelling in their ankles
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Swelling Ankles
|
15 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that have noticed losing more hair
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Hair Loss
|
7 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that have noticed in increase in acne
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Acne
|
2 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that they have been having trouble sleeping
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Insomnia
|
12 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that they have noticed changes in mood
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Mood Change
|
12 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that they have had upset stomach, indigestion, diarrhea, or bloating
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: GI Upset
|
5 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study that noticed feeling tired or worn out
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Fatigue
|
16 Participants
|
14 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study noticing a metallic taste in their mouth
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Unpleasant Taste
|
4 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Month 36Count of participants self reporting at any time throuhgout the study noticing any blurred vision
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Blurred Vision
|
4 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed moon facies over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Moon Facies
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed skin thinning over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Skin Thinning
|
3 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed hirsuitism over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Hirsuitism
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed bruising over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Bruising
|
4 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed ankle swelling over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Ankle Swelling
|
7 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Month 36Any new physician assessed hair loss over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Hair Loss
|
4 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed cataracts over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Cataracts
|
2 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Month 36New physician assessed myopathy over study duration
Outcome measures
| Measure |
ACTH
n=29 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Myopathy
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Month 36Change in hemoglobin A1c
Outcome measures
| Measure |
ACTH
n=14 Participants
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=10 Participants
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Safety and Tolerability of ACTH: Change in Hemoglobin A1c
|
0.14 percent
Standard Deviation 0.36
|
0.05 percent
Standard Deviation 0.32
|
Adverse Events
ACTH
Serious events: 8 serious events
Other events: 29 other events
Deaths: 0 deaths
Placebo
Serious events: 7 serious events
Other events: 29 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ACTH
n=29 participants at risk
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 participants at risk
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
Gastrointestinal disorders
Gastrointestinal
|
13.8%
4/29 • Number of events 6 • 36 Months
|
6.7%
2/30 • Number of events 3 • 36 Months
|
|
General disorders
infection
|
6.9%
2/29 • Number of events 2 • 36 Months
|
10.0%
3/30 • Number of events 4 • 36 Months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal
|
6.9%
2/29 • Number of events 2 • 36 Months
|
6.7%
2/30 • Number of events 3 • 36 Months
|
|
Respiratory, thoracic and mediastinal disorders
respiratory
|
6.9%
2/29 • Number of events 2 • 36 Months
|
0.00%
0/30 • 36 Months
|
Other adverse events
| Measure |
ACTH
n=29 participants at risk
ACTH administered subcutaneously as a pulsed regimen of 3 consecutive days per month
ACTH: Acthar gel
|
Placebo
n=30 participants at risk
Placebo subcutaneous injections administered on 3 consecutive days per month
Placebo: Placebo
|
|---|---|---|
|
General disorders
Alopecia
|
13.8%
4/29 • Number of events 4 • 36 Months
|
20.0%
6/30 • Number of events 7 • 36 Months
|
|
Renal and urinary disorders
Bladder function
|
17.2%
5/29 • Number of events 5 • 36 Months
|
6.7%
2/30 • Number of events 2 • 36 Months
|
|
General disorders
Bruising
|
24.1%
7/29 • Number of events 7 • 36 Months
|
36.7%
11/30 • Number of events 11 • 36 Months
|
|
Cardiac disorders
cardiovascular
|
48.3%
14/29 • Number of events 17 • 36 Months
|
26.7%
8/30 • Number of events 10 • 36 Months
|
|
General disorders
dermatologic
|
24.1%
7/29 • Number of events 10 • 36 Months
|
33.3%
10/30 • Number of events 12 • 36 Months
|
|
General disorders
Dysgeusia
|
13.8%
4/29 • Number of events 4 • 36 Months
|
6.7%
2/30 • Number of events 2 • 36 Months
|
|
General disorders
Edema
|
48.3%
14/29 • Number of events 24 • 36 Months
|
30.0%
9/30 • Number of events 10 • 36 Months
|
|
General disorders
Fatigue
|
41.4%
12/29 • Number of events 12 • 36 Months
|
40.0%
12/30 • Number of events 12 • 36 Months
|
|
Gastrointestinal disorders
Gastrointestinal
|
31.0%
9/29 • Number of events 26 • 36 Months
|
43.3%
13/30 • Number of events 15 • 36 Months
|
|
General disorders
genitourinary
|
0.00%
0/29 • 36 Months
|
6.7%
2/30 • Number of events 3 • 36 Months
|
|
General disorders
Infection
|
31.0%
9/29 • Number of events 25 • 36 Months
|
43.3%
13/30 • Number of events 22 • 36 Months
|
|
General disorders
Injection site reaction
|
10.3%
3/29 • Number of events 12 • 36 Months
|
20.0%
6/30 • Number of events 6 • 36 Months
|
|
General disorders
Metabolic
|
10.3%
3/29 • Number of events 3 • 36 Months
|
0.00%
0/30 • 36 Months
|
|
General disorders
Metabolic/endocrine
|
20.7%
6/29 • Number of events 8 • 36 Months
|
16.7%
5/30 • Number of events 5 • 36 Months
|
|
General disorders
Mood
|
37.9%
11/29 • Number of events 14 • 36 Months
|
36.7%
11/30 • Number of events 12 • 36 Months
|
|
General disorders
Musculoskeletal
|
75.9%
22/29 • Number of events 83 • 36 Months
|
53.3%
16/30 • Number of events 46 • 36 Months
|
|
General disorders
Neoplasm
|
13.8%
4/29 • Number of events 6 • 36 Months
|
0.00%
0/30 • 36 Months
|
|
Nervous system disorders
Neurologic
|
51.7%
15/29 • Number of events 38 • 36 Months
|
30.0%
9/30 • Number of events 17 • 36 Months
|
|
General disorders
other
|
31.0%
9/29 • Number of events 14 • 36 Months
|
6.7%
2/30 • Number of events 2 • 36 Months
|
|
Renal and urinary disorders
renal
|
0.00%
0/29 • 36 Months
|
3.3%
1/30 • Number of events 3 • 36 Months
|
|
Respiratory, thoracic and mediastinal disorders
respiratory
|
6.9%
2/29 • Number of events 3 • 36 Months
|
3.3%
1/30 • Number of events 2 • 36 Months
|
|
General disorders
sleep
|
48.3%
14/29 • Number of events 19 • 36 Months
|
30.0%
9/30 • Number of events 9 • 36 Months
|
|
General disorders
visual
|
44.8%
13/29 • Number of events 16 • 36 Months
|
26.7%
8/30 • Number of events 11 • 36 Months
|
|
General disorders
weight gain
|
27.6%
8/29 • Number of events 10 • 36 Months
|
26.7%
8/30 • Number of events 9 • 36 Months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place