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The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cabaletta 30gr. | Experimental | weekly IV of Cabaletta 30gr. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cabaletta | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety Lab Evaluations | Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 . | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 24 weeks |
| Videofluoroscopy (VFS) Score |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yoseph Caraco, M.D | Hadassah Medical Organization | Principal Investigator |
| Bernard Brais, MD | McGill University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA | Los Angeles | California | 90095 | United States | ||
| Tahseen Mozaffar |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cabaletta 30gr | weekly IV of Cabaletta 30gr Cabaletta |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cabaletta 30gr. | weekly IV of Cabaletta 30gr. Cabaletta |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Safety Lab Evaluations | Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 . | 24 patients were available for analysis at week 24. Not all patients had all labs performed. Missing data accounts for the differing number of patients with specific lab values. | Posted | Mean | Standard Deviation | percentage change from baseline | 24 weeks |
|
|
28 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cabaletta 30gr | weekly IV of Cabaletta 30gr Cabaletta | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Urinary Tract Infection | Infections and infestations | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Warren Wasiewski | Bioblast Pharma | 717-368-0780 | Warren.wasiewski@bioblastpharma.com |
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| ID | Term |
|---|---|
| D039141 | Muscular Dystrophy, Oculopharyngeal |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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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.
| 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. | 28 weeks |
| Orange |
| California |
| 92868 |
| United States |
| Montreal Neurological Institute, McGill University | Montreal | Quebec | H3A 2B4 | Canada |
| Hadassah medical center | Jerusalem | 91120 | Israel |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
|
|
|
| Secondary | 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. | 22 patients had data available for analysis at week 24 | Posted | Mean | Standard Deviation | percentage change from baseline | 24 weeks |
|
|
|
| Secondary | 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. | 11 patients had valid baseline data and week 24 data | Posted | Mean | Standard Deviation | Points on an 8 point scale | 24 Weeks |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | percentage change from baseline | 28 weeks |
|
|
|
| 25 |
| 2 |
| 25 |
| 25 |
| 25 |
| Pneumonia Aspiration | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Aspiration | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | Systematic Assessment |
|
| Administrative Site Bruise | General disorders | Systematic Assessment |
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| Asthenia | General disorders | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
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| Influenza Like Illness | General disorders | Systematic Assessment |
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| Pyrexia | General disorders | Systematic Assessment |
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| Bacteriuria | Infections and infestations | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | Systematic Assessment |
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| Pharyngitis | Infections and infestations | Systematic Assessment |
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| Urinary Tract Infection | Infections and infestations | Systematic Assessment |
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| Procedural Pain | Injury, poisoning and procedural complications | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Back Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Muscle Fatigue | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Pain in Extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Elevated Urine Glucose Levels | Renal and urinary disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |