Trial Outcomes & Findings for Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1) (NCT NCT00233519)
NCT ID: NCT00233519
Last Updated: 2012-06-25
Results Overview
Safety and tolerability was measured via interval laboratory studies,electrocardiograms, echocardiograms, ultrasounds of the abdomen and pelvis, dual energy x-ray absorptiometry (DEXA) studies, chest and neck x-rays, and serial physical examinations. The participants had six inpatient evaluations at the University of Rochester General Clinical Research Center (Weeks 0, 8, 16, 24, 28, and 40) and nine outpatient evaluations. Patients also completed side effects diaries to record any adverse events in the interval time between inpatient and outpatient evaluations.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
17 participants
Primary outcome timeframe
24 weeks
Results posted on
2012-06-25
Participant Flow
Participant milestones
| Measure |
Cohort 1- Two Escalating Doses of iPlex
N=6 This group received self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks.
|
Cohort 2-Three Escalating Doses of iPlex
N=9 Following approval of the safety monitoring committee who reviewed the data on the cohort 1, the second cohort received consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection.
|
|---|---|---|
|
Overall Study
STARTED
|
7
|
10
|
|
Overall Study
COMPLETED
|
6
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
Baseline characteristics by cohort
| Measure |
Cohort 1- Two Escalating Doses of iPlex
n=7 Participants
N=6 This group received self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks.
|
Cohort 2-Three Escalating Doses of iPlex
n=10 Participants
N=9 Following approval of the safety monitoring committee who reviewed the data on the cohort 1, the second cohort received consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection.
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
10 participants
n=7 Participants
|
17 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeksSafety and tolerability was measured via interval laboratory studies,electrocardiograms, echocardiograms, ultrasounds of the abdomen and pelvis, dual energy x-ray absorptiometry (DEXA) studies, chest and neck x-rays, and serial physical examinations. The participants had six inpatient evaluations at the University of Rochester General Clinical Research Center (Weeks 0, 8, 16, 24, 28, and 40) and nine outpatient evaluations. Patients also completed side effects diaries to record any adverse events in the interval time between inpatient and outpatient evaluations.
Outcome measures
| Measure |
Cohort 1- Two Escalating Doses of iPlex
n=6 Participants
N=6 This group received self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks.
|
Cohort 2-Three Escalating Doses of iPlex
n=9 Participants
N=9 Following approval of the safety monitoring committee who reviewed the data on the cohort 1, the second cohort received consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection.
|
|---|---|---|
|
The Number of Study Participants Who Safely Tolerated Somatokine
|
6 participants
|
9 participants
|
Adverse Events
Cohort 1- Two Escalating Doses of iPlex
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2-Three Escalating Doses of iPlex
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1- Two Escalating Doses of iPlex
n=6 participants at risk
N=6 This group received self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks.
|
Cohort 2-Three Escalating Doses of iPlex
n=9 participants at risk
N=9 Following approval of the safety monitoring committee who reviewed the data on the cohort 1, the second cohort received consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection.
|
|---|---|---|
|
Hepatobiliary disorders
Acute Inflammation of Gallbladder
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
Other adverse events
| Measure |
Cohort 1- Two Escalating Doses of iPlex
n=6 participants at risk
N=6 This group received self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks.
|
Cohort 2-Three Escalating Doses of iPlex
n=9 participants at risk
N=9 Following approval of the safety monitoring committee who reviewed the data on the cohort 1, the second cohort received consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection.
|
|---|---|---|
|
Psychiatric disorders
Mood alteration/depression
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle pain
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Neck stiffness
|
16.7%
1/6 • Number of events 2 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Papules
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Rubor
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Skin excision
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Skin tag-moles
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Small raised red dots
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Tenderness
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Nervous system disorders
Tremor
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Twisted ankle
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Upper posterior pain
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Vascular disorders
Venostasis
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • Number of events 2 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
ankle fracture
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Cardiac disorders
Atrial flutter
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Basal Cell Carcinoma
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Eye disorders
Bilateral swelling of optic nerves
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
blisters
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Eye disorders
blurred vision
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
bruising
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Change in size of moles
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
General disorders
Chills
|
16.7%
1/6 • Number of events 2 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Gastrointestinal disorders
Cold sensation in esophagus
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Congestion
|
50.0%
3/6 • Number of events 3 • 40 weeks
|
22.2%
2/9 • Number of events 3 • 40 weeks
|
|
Gastrointestinal disorders
Constipation
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
cramping
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Cut on top of head
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cyst
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Reproductive system and breast disorders
Cyst left ovary
|
0.00%
0/2 • 40 weeks
|
20.0%
1/5 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Nervous system disorders
dizziness
|
0.00%
0/6 • 40 weeks
|
22.2%
2/9 • Number of events 2 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Edema
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Blood and lymphatic system disorders
Edema
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Metabolism and nutrition disorders
Elevated thyroid stimulating hormone (TSH) level
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Eye disorders
Eyelid dysfunction
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Fell on ice, hit left knee
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Eye disorders
Film on both eyes
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
General disorders
Flu-like symptoms
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Hepatobiliary disorders
Gallbladder pain
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 3 • 40 weeks
|
|
Hepatobiliary disorders
Gallstone
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Infections and infestations
Gingivitis
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Reproductive system and breast disorders
Gynecomastia
|
25.0%
1/4 • Number of events 1 • 40 weeks
|
0.00%
0/4 • 40 weeks
|
|
Eye disorders
Halos
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
General disorders
Headache
|
33.3%
2/6 • Number of events 2 • 40 weeks
|
33.3%
3/9 • Number of events 7 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Heel pain
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Endocrine disorders
Hot flashes/flushes
|
0.00%
0/6 • 40 weeks
|
22.2%
2/9 • Number of events 2 • 40 weeks
|
|
Metabolism and nutrition disorders
Hyperkelmia
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
0.00%
0/6 • 40 weeks
|
33.3%
3/9 • Number of events 4 • 40 weeks
|
|
Vascular disorders
Hypovolemia
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Increase in moles
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
General disorders
Increased need for sleep
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
|
General disorders
Increased thirst
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Injection site reaction
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
77.8%
7/9 • Number of events 8 • 40 weeks
|
|
Renal and urinary disorders
Kidney stone
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Left chest pain
|
33.3%
2/6 • Number of events 2 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Left knee pain
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Left pinky pain
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Musculoskeletal and connective tissue disorders
Lower back pain
|
16.7%
1/6 • Number of events 1 • 40 weeks
|
0.00%
0/9 • 40 weeks
|
|
Skin and subcutaneous tissue disorders
Lump
|
0.00%
0/6 • 40 weeks
|
11.1%
1/9 • Number of events 1 • 40 weeks
|
Additional Information
Richard T. Moxley, III MD
University of Rochester Medical Center
Phone: 585-275-5006
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee National Institutes of Health (NIH) can review the content of planned submissions prior to public release (3 weeks notice requested) to insure integrity of the NIH and the Data Safety Monitoring Board (DMSB)staff.
- Publication restrictions are in place
Restriction type: OTHER