Phase 2
Completed 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
Source: ClinicalTrials.gov NCT00577577 ↗Enrolled (actual)
69
Serious AEs
18.8%
Results posted
Jan 2022
Primary outcomePrimary: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance — 12.40; 20.11 meters
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
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 | — |
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. Informational only — not medical advice.