Phase 2
N=69
Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1
Bottom Line
View on ClinicalTrials.gov: NCT00577577 ↗Enrolled (actual)
69
Serious AEs
18.8%
Results posted
Jan 2022
Primary outcome: Primary: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance — 12.40; 20.11 meters
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- rhIGF-I/rhIGFBP-3 (Drug); placebo (Drug)
- Age
- Adult, Older Adult · 21+ yrs
- Sex
- All
- Sponsor
- Insmed Incorporated
- Primary completion
- Dec 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance |
12.40; 20.11 | — |
| PRIMARY Change From Baseline in Daily Step Count |
-140; -58 | — |
| PRIMARY Peak Activity Index: Change From Baseline in Number of Steps Walked Per Minute During the 30 Minute Period of Fastest Walking |
0.5; 1.1 | — |
| PRIMARY Sustained Activity Index: Change From Baseline in the Highest Number of Steps Walked Per Minute Over 20 Minutes of Activity |
-0.9; 0.8 | — |
| PRIMARY Change From Baseline in the Percentage of Time That Participants Spent Inactive |
-0.4; -0.0 | — |
| PRIMARY Change From Baseline in Time Taken for Participants to Ascend and Descend 4 Stairs |
0.0; -0.5; -0.1; -0.5 | — |
| PRIMARY Change From Baseline in Time Taken to Traverse 30 Feet |
0.2; -0.2 | — |
| PRIMARY Change From Baseline in Purdue Pegboard Test Scores |
-0.2; 0.3 | — |
| PRIMARY Change From Baseline in Forced Vital Capacity (FVC) Volume While Sitting or Lying Down |
-0.1; 0.0; -0.2; -0.1 | — |
| PRIMARY Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted While Sitting or Lying Down |
0.0; -2.3; -3.8; -3.2 | — |
| PRIMARY Change From Baseline in Manual Muscle Test (MMT) Scores |
0.9; -0.3; -0.4; 0.0; 1.0; -0.3 | — |
| PRIMARY Change From Baseline in Selective Reminding Test T-Scores (Total Word and Delayed Words) |
4.3; 2.9; -3.2; 0.04 | — |
| PRIMARY Change From Baseline in Selective Reminding Test Raw Scores (Cued Recall and Recognition) |
0.0; -0.4; 0.0; 0 | — |
| PRIMARY Change From Baseline in Rey Complex Figure (RCF) Test Scores |
7.5; 4.8; 5.2; 4.8; -1.5; 5.9 | — |
| PRIMARY Change From Baseline in Letter-Number Sequencing (LNS) Test Scores |
1.18; -0.36 | — |
| PRIMARY Change From Baseline in Trail Making Test (TMT) Scores |
2.2; -0.3; 4.0; -0.9 | — |
| PRIMARY Change From Baseline in the Stroop Color Word Test Scores |
-0.4; 2.0; 2.0; 0.9; 1.3; 1.0 | — |
| PRIMARY Change From Baseline to Week 24 Scores on the Beck Depression Inventory II (BDI-II) Questionnaire |
-1.63; -0.14 | — |
| PRIMARY Change From Baseline in Average Fasting Glucose Concentration in the Blood |
-6.8; 0.2 | — |
| PRIMARY Change From Baseline in Average Fasting Insulin Concentration in the Blood |
-5.7; 0.7 | — |
| PRIMARY Change From Baseline in Qualitative Insulin Sensitivity Check Index (QUICKI) |
0.05; -0.01 | — |
| PRIMARY Change From Baseline in Insulin Sensitivity Index-Matsuda (ISI-Matsuda) |
5.5; -1.4 | — |
| PRIMARY Change From Baseline in Total Blood Cholesterol Level |
-7.9; -2.7 | — |
| PRIMARY Change From Baseline in Total Blood Low-density Lipoproteins (LDL) Level |
1.5; 5.0 | — |
| PRIMARY Change From Baseline in Total Blood High-density Lipoproteins (HDL) Level |
0.3; -3.0 | — |
| PRIMARY Change From Baseline in Total Blood Triglycerides Level |
-54.8; -21.7 | — |
| PRIMARY Change From Baseline in Gastro-esophageal Reflux Disease (GERD) Symptom Frequency Questionnaire (GSFQ) Scores |
-6.4; -0.5 | — |
| PRIMARY Change From Baseline in Gastrointestinal Symptom Rating Scale for Irritable Bowel Syndrome (GSRS-IBS) Questionnaire Scores |
-0.29; -0.02 | — |
| PRIMARY Change From Baseline in Swallowing Disturbance Questionnaire (SDQ) Scores |
0.29; -0.34 | — |
| PRIMARY Change From Baseline in Short Form (36) (SF-36) Questionnaire Scores |
1.4; -0.5; 1.2; 1.2 | — |
| PRIMARY Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Severity Scores |
0.0; 0.5; -0.2; 0.6 | — |
| PRIMARY Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Interference Scores |
0.0; -0.5 | — |
Summary
To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients
Eligibility Criteria
Inclusion Criteria (list is not inclusive):
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
- Able to self-administer study medication by subcutaneous injection or caregiver is available to administer study medication
Exclusion Criteria (list is not inclusive):
- Congenital DM1
- Weight greater than 100 kg or body mass index greater than 30 kg/m2
- Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone, investigational agent within 60 days of screening
- Current diagnosis or history of malignancy expect for surgically cured skin cancer or pilomatricoma
- Changes in lipid lowering medications during the 3 months prior to screening
- Diaphragmatic weakness such that patients are unable to tolerate the supine position, or swallowing impairment such that patients are unable to maintain nutrition without use of gastrostomy.
- Major psychiatric illness (major depression, bipolar disorder or schizophrenia) within twelve months of screening
- History of non-compliance with other therapies
Data sourced from ClinicalTrials.gov (NCT00577577). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.