A Phase 2 interventional study of Cabaletta in Oculopharyngeal Muscular Dystrophy, sponsored by Bioblast Pharma Ltd.. Completed at 4 sites in 3 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2017-10-17.
Sponsored by Bioblast Pharma Ltd. · Phase 2, Interventional, and Treatment
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.
This study's enrollment of 25 is close to the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.
Browse Muscular Dystrophies studies →Bioblast Pharma Ltd. is the lead sponsor of 5 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
weekly IV of Cabaletta 30gr.
Drug: Cabaletta
Safety Lab Evaluations
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Time frame: 24 weeks
Drinking Test Score
Change from baseline in ice water drinking time, in seconds, at week 24. Times greater than 8 seconds to complete the drinking test are considered abnormal.
Time frame: 24 weeks
Videofluoroscopy (VFS) Score
Penetration aspiration score results assessed by VFS comparing baseline, prior to treatment, to week 24. This is an 8 point scale. The higher the number the greater the risk of aspiration. The result reported is the difference from the baseline scores.
Time frame: 24 Weeks
SWAL-QOL, Swallowing Quality of Life Questionnaire
Summary of Quality of Life in Swallowing Disorders total symptom score results over time, change from baseline at weeks 12 and 24 This is a 100 point scale. The higher the number the better the quality of life.
Time frame: 28 weeks
| Milestone | Cabaletta 30gr |
|---|---|
| Started | 25 |
| Completed | 24 |
| Not completed | 1 |
| Withdrew: Death | 1 |
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
| percentage change from baseline | Cabaletta 30gr. |
|---|---|
| White Blood Cell Count | -4.0 ± 16.69 |
| Red Blood Cell Count | 0.1 ± 0.42 |
| Hemoglobin | 0.2 ± 7.98 |
| Hematocrit | 1.1 ± 9.28 |
| Platelets | -0.5 ± 13.42 |
| Alanine Aminotransferase | -0.8 ± 34.59 |
| Aspartate Aminotransferase | -5.7 ± 30.93 |
| Creatinine phosphokinase | 6.8 ± 37.12 |
| Gamma-Glutamyl Transferase | 9.3 ± 28.73 |
| Total Bilirubin | 37.2 ± 46.02 |
| Glucose | 12 ± 15.93 |
| Lactose Dehydrogenase | 23 ± 0.9 |
| Prothrombin Time | 7.1 ± 12.62 |
| International Normalized Ratio | 1.3 ± 8.48 |
Change from baseline in ice water drinking time, in seconds, at week 24. Times greater than 8 seconds to complete the drinking test are considered abnormal.
| percentage change from baseline | Cabaletta 30gr |
|---|---|
| Drinking Test Score | -33.8 ± 34.89 |
Penetration aspiration score results assessed by VFS comparing baseline, prior to treatment, to week 24. This is an 8 point scale. The higher the number the greater the risk of aspiration. The result reported is the difference from the baseline scores.
| Points on an 8 point scale | Cabaletta 30gr |
|---|---|
| Videofluoroscopy (VFS) Score | 0.1 ± 1.27 |
Summary of Quality of Life in Swallowing Disorders total symptom score results over time, change from baseline at weeks 12 and 24 This is a 100 point scale. The higher the number the better the quality of life.
| percentage change from baseline | Cabaletta 30gr |
|---|---|
| SWAL-QOL, Swallowing Quality of Life Questionnaire | 12.4 ± 16.87 |
Collected over 28 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cabaletta 30gr | 1/25 (4%) | 2/25 (8%) | 25/25 (100%) |
| Event | Cabaletta 30gr |
|---|---|
| Urinary Tract InfectionInfections and infestations | 1/25 |
| Pneumonia AspirationRespiratory, thoracic and mediastinal disorders | 1/25 |
| AspirationRespiratory, thoracic and mediastinal disorders | 1/25 |
| Event | Cabaletta 30gr |
|---|---|
| Elevated Urine Glucose LevelsRenal and urinary disorders | 13/25 |
| Procedural PainInjury, poisoning and procedural complications | 10/25 |
| Back PainMusculoskeletal and connective tissue disorders | 7/25 |
| Musculoskeletal PainMusculoskeletal and connective tissue disorders | 5/25 |
| HeadacheNervous system disorders | 5/25 |
| Administrative Site BruiseGeneral disorders | 4/25 |
| FatigueGeneral disorders | 4/25 |
| NasopharyngitisInfections and infestations | 4/25 |
| Abdominal PainGastrointestinal disorders | 3/25 |
| Influenza Like IllnessGeneral disorders | 3/25 |
| Age, Categorical(Participants) | Cabaletta 30gr. |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 20 |
| >=65 years | 5 |
| Age, Continuous(years) | Cabaletta 30gr. |
|---|---|
| Mean | 59.5 ± 8.34 |
| Sex: Female, Male(Participants) | Cabaletta 30gr. |
|---|---|
| Female | 13 |
| Male | 12 |
| Region of Enrollment(participants) | Cabaletta 30gr. |
|---|---|
| Canada | 11 |
| Israel | 14 |
This study is completed, as verified in Sep 2017. You cannot join it, but the record below documents what was studied.
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Bioblast Pharma Ltd.