Trial Outcomes & Findings for Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy (NCT NCT01207908)
NCT ID: NCT01207908
Last Updated: 2021-01-20
Results Overview
Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
44 participants
Primary outcome timeframe
6 months
Results posted on
2021-01-20
Participant Flow
Participant milestones
| Measure |
IGF-1
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
Once daily steroid treatment (Deflazacort or Prednisone) administered orally for DMD.
|
|---|---|---|
|
Overall Study
STARTED
|
22
|
22
|
|
Overall Study
COMPLETED
|
17
|
21
|
|
Overall Study
NOT COMPLETED
|
5
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
IGF-1
n=17 Participants
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
n=21 Participants
Standard daily steroid treatment for DMD
|
Total
n=38 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.56 Years
STANDARD_DEVIATION 1.28 • n=5 Participants
|
8.70 Years
STANDARD_DEVIATION 1.79 • n=7 Participants
|
8.64 Years
STANDARD_DEVIATION 1.67 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsOutcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Outcome measures
| Measure |
IGF-1
n=17 Participants
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
n=21 Participants
Once daily steroid treatment (Deflazacort or Prednisone) administered orally for DMD.
|
|---|---|---|
|
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
|
3.4 meters
Standard Deviation 32.4
|
-5.1 meters
Standard Deviation 50.2
|
SECONDARY outcome
Timeframe: 6 monthsOutcome Measure Data Table shows change of height velocity at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Outcome measures
| Measure |
IGF-1
n=17 Participants
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
n=21 Participants
Once daily steroid treatment (Deflazacort or Prednisone) administered orally for DMD.
|
|---|---|---|
|
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
|
2.60 cm/year
Standard Deviation 1.64
|
-0.06 cm/year
Standard Deviation 1.69
|
SECONDARY outcome
Timeframe: 6 monthsOutcome Measure Data Table shows change of North Star Ambulatory Assessment (NSAA) score at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1). The NSAA is a 17-item scale that grades performance of various functional skills on a scale from 0 (unable), 1 (complete independently but with modifications), and 2 (complete without compensation). The range of NSAA score is from 0 to 34. The higher score indicates better motor function.
Outcome measures
| Measure |
IGF-1
n=17 Participants
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
n=21 Participants
Once daily steroid treatment (Deflazacort or Prednisone) administered orally for DMD.
|
|---|---|---|
|
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
|
-0.94 score on a scale
Standard Deviation 2.41
|
-0.4 score on a scale
Standard Deviation 3.0
|
Adverse Events
IGF-1
Serious events: 1 serious events
Other events: 20 other events
Deaths: 0 deaths
Standard Steroid Treatment Alone
Serious events: 0 serious events
Other events: 21 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
IGF-1
n=22 participants at risk
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
n=22 participants at risk
Standard daily steroid treatment for DMD
|
|---|---|---|
|
Nervous system disorders
Intracranial hypertension
|
4.5%
1/22 • Number of events 1 • 6 months
|
0.00%
0/22 • 6 months
|
Other adverse events
| Measure |
IGF-1
n=22 participants at risk
IGF-1 plus standard steroid treatment
IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
|
Standard Steroid Treatment Alone
n=22 participants at risk
Standard daily steroid treatment for DMD
|
|---|---|---|
|
Nervous system disorders
Headache
|
50.0%
11/22 • 6 months
|
40.9%
9/22 • 6 months
|
|
Gastrointestinal disorders
Nausea and vomiting
|
31.8%
7/22 • 6 months
|
22.7%
5/22 • 6 months
|
|
Nervous system disorders
seizure
|
0.00%
0/22 • 6 months
|
4.5%
1/22 • 6 months
|
|
Nervous system disorders
papilledema
|
4.5%
1/22 • 6 months
|
0.00%
0/22 • 6 months
|
|
Skin and subcutaneous tissue disorders
stinging at injection site
|
13.6%
3/22 • 6 months
|
—
0/0 • 6 months
|
|
General disorders
fever
|
9.1%
2/22 • 6 months
|
13.6%
3/22 • 6 months
|
|
Skin and subcutaneous tissue disorders
rash and skin welt
|
13.6%
3/22 • 6 months
|
4.5%
1/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
lipohypertrophy
|
18.2%
4/22 • 6 months
|
0.00%
0/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Foot pain
|
4.5%
1/22 • 6 months
|
4.5%
1/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Finger injury
|
4.5%
1/22 • 6 months
|
4.5%
1/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Leg injury
|
4.5%
1/22 • 6 months
|
0.00%
0/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.5%
1/22 • 6 months
|
13.6%
3/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Finger infection
|
4.5%
1/22 • 6 months
|
0.00%
0/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Leg pain
|
9.1%
2/22 • 6 months
|
18.2%
4/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Arm weakness
|
4.5%
1/22 • 6 months
|
0.00%
0/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Leg numbness
|
4.5%
1/22 • 6 months
|
0.00%
0/22 • 6 months
|
Additional Information
Dr. Meilan Rutter
Cincinnati Children's Hospital Medical Center
Phone: 513-636-4744
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place