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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)

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ClinicalTrials.gov Identifier: NCT00233519
Recruitment Status : Completed
First Posted : October 5, 2005
Results First Posted : February 1, 2010
Last Update Posted : June 25, 2012
Sponsor:
Collaborators:
National Institute of Neurological Disorders and Stroke (NINDS)
Imsmed Incorporated
Information provided by (Responsible Party):
Richard T Moxley, University of Rochester

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Myotonic Dystrophy
Intervention Drug: SomatoKine/IPLEX
Enrollment 17
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex
Hide Arm/Group Description 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. 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.
Period Title: Overall Study
Started 7 10
Completed 6 9
Not Completed 1 1
Arm/Group Title Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex Total
Hide Arm/Group Description 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. 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 of all reporting groups
Overall Number of Baseline Participants 7 10 17
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 10 participants 17 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
7
 100.0%
10
 100.0%
17
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 10 participants 17 participants
Female
3
  42.9%
6
  60.0%
9
  52.9%
Male
4
  57.1%
4
  40.0%
8
  47.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 10 participants 17 participants
7 10 17
1.Primary Outcome
Title The Number of Study Participants Who Safely Tolerated Somatokine
Hide Description 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.
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 6 9
Measure Type: Number
Unit of Measure: participants
6 9
Time Frame 40 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex
Hide Arm/Group Description 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. 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.
All-Cause Mortality
Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/6 (0.00%)      1/9 (11.11%)    
Hepatobiliary disorders     
Acute Inflammation of Gallbladder  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1- Two Escalating Doses of iPlex Cohort 2-Three Escalating Doses of iPlex
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/6 (50.00%)      7/9 (77.78%)    
Blood and lymphatic system disorders     
Edema  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Cardiac disorders     
Atrial flutter  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Endocrine disorders     
Hot flashes/flushes  1  0/6 (0.00%)  0 2/9 (22.22%)  2
Eye disorders     
Bilateral swelling of optic nerves  1  0/6 (0.00%)  0 1/9 (11.11%)  1
blurred vision  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Eyelid dysfunction  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Film on both eyes  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Halos  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Gastrointestinal disorders     
Abdominal pain  1  1/6 (16.67%)  2 0/9 (0.00%)  0
Cold sensation in esophagus  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Constipation  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Diarrhea  1  1/6 (16.67%)  1 1/9 (11.11%)  1
Vomiting  1  0/6 (0.00%)  0 1/9 (11.11%)  1
General disorders     
Chills  1  1/6 (16.67%)  2 0/9 (0.00%)  0
Flu-like symptoms  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Headache  1  2/6 (33.33%)  2 3/9 (33.33%)  7
Increased need for sleep  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Increased thirst  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Hepatobiliary disorders     
Gallbladder pain  1  0/6 (0.00%)  0 1/9 (11.11%)  3
Gallstone  1  1/6 (16.67%)  1 1/9 (11.11%)  1
Infections and infestations     
Gingivitis  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Metabolism and nutrition disorders     
Elevated thyroid stimulating hormone (TSH) level  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Hyperkelmia  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Hypoglycemia  1  0/6 (0.00%)  0 3/9 (33.33%)  4
Musculoskeletal and connective tissue disorders     
ankle fracture  1  0/6 (0.00%)  0 1/9 (11.11%)  1
cramping  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Fell on ice, hit left knee  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Heel pain  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Left chest pain  1  2/6 (33.33%)  2 0/9 (0.00%)  0
Left knee pain  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Left pinky pain  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Lower back pain  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Muscle pain  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Muscle weakness  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Neck stiffness  1  1/6 (16.67%)  2 1/9 (11.11%)  1
Tenderness  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Twisted ankle  1  1/6 (16.67%)  1 1/9 (11.11%)  1
Upper posterior pain  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Cyst  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Nervous system disorders     
dizziness  1  0/6 (0.00%)  0 2/9 (22.22%)  2
Tremor  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Psychiatric disorders     
Mood alteration/depression  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Renal and urinary disorders     
Kidney stone  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Reproductive system and breast disorders     
Cyst left ovary  1  0/2 (0.00%)  0 1/5 (20.00%)  1
Gynecomastia  1  1/4 (25.00%)  1 0/4 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Congestion  1  3/6 (50.00%)  3 2/9 (22.22%)  3
Sore throat  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Skin and subcutaneous tissue disorders     
Alopecia  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Basal Cell Carcinoma  1  0/6 (0.00%)  0 1/9 (11.11%)  1
blisters  1  0/6 (0.00%)  0 1/9 (11.11%)  1
bruising  1  1/6 (16.67%)  1 1/9 (11.11%)  1
Change in size of moles  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Cut on top of head  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Dermatitis  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Edema  1  1/6 (16.67%)  1 1/9 (11.11%)  1
Increase in moles  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Injection site reaction  1  1/6 (16.67%)  1 7/9 (77.78%)  8
Lump  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Papules  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Pruritis  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Rubor  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Skin excision  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Skin tag-moles  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Small raised red dots  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Vascular disorders     
Hypovolemia  1  0/6 (0.00%)  0 1/9 (11.11%)  1
Venostasis  1  1/6 (16.67%)  1 0/9 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
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.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Richard T. Moxley, III MD
Organization: University of Rochester Medical Center
Phone: 585-275-5006
EMail: RichardT_Moxley@urmc.rochester.edu
Layout table for additonal information
Responsible Party: Richard T Moxley, University of Rochester
ClinicalTrials.gov Identifier: NCT00233519     History of Changes
Other Study ID Numbers: 5 U54 NS048843-03 A
First Submitted: October 3, 2005
First Posted: October 5, 2005
Results First Submitted: August 13, 2009
Results First Posted: February 1, 2010
Last Update Posted: June 25, 2012