Title

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
  • Phase

    Phase 2
  • Study Type

    Interventional
  • Intervention/Treatment

    trehalose ...
  • Study Participants

    25
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
Study Started
Feb 28
2014
Primary Completion
Apr 30
2016
Study Completion
Apr 30
2016
Results Posted
Aug 23
2017
Last Update
Oct 17
2017

Drug Cabaletta

Cabaletta 30gr. Experimental

weekly IV of Cabaletta 30gr.

Criteria

Inclusion Criteria:

Males and females
18 - 80 years (inclusive) of age
Clinically and genetically diagnosed as OPMD
Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
Patients who provide written informed consent to participate in the study
Body Mass Index (BMI) <30 kg/m2

Exclusion Criteria:

Diabetes mellitus type 1 or 2
Other major diseases, e.g.: renal failure (creatinine clearance <60ml/min), liver failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers, tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis
Uncontrolled heart disease , CHF,
Other neuromuscular diseases
Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection (e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates), esophageal rings and webs, spastic motility disorders of the esophagus.
History of malignancy (except non-invasive skin malignancy)
History of neck irradiation
Pregnant or currently lactating women
Obesity (BMI≥ 30) and associated morbidity
Prior pharyngeal myotomy
Weight loss of more than 10% in the last 12 months.
Known hypersensitivity to any ingredients in the injection
Patient receiving anticoagulant treatment (e.g. warfarin)

Summary

Cabaletta 30gr

All Events

Event Type Organ System Event Term Cabaletta 30gr

Safety Lab Evaluations

Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .

Cabaletta 30gr.

Alanine Aminotransferase

-0.8
percentage change from baseline (Mean)
Standard Deviation: 34.59

Aspartate Aminotransferase

-5.7
percentage change from baseline (Mean)
Standard Deviation: 30.93

Creatinine phosphokinase

6.8
percentage change from baseline (Mean)
Standard Deviation: 37.12

Gamma-Glutamyl Transferase

9.3
percentage change from baseline (Mean)
Standard Deviation: 28.73

Glucose

12.0
percentage change from baseline (Mean)
Standard Deviation: 15.93

Hematocrit

1.1
percentage change from baseline (Mean)
Standard Deviation: 9.28

Hemoglobin

0.2
percentage change from baseline (Mean)
Standard Deviation: 7.98

International Normalized Ratio

1.3
percentage change from baseline (Mean)
Standard Deviation: 8.48

Lactose Dehydrogenase

23.0
percentage change from baseline (Mean)
Standard Deviation: 0.9

Platelets

-0.5
percentage change from baseline (Mean)
Standard Deviation: 13.42

Prothrombin Time

7.1
percentage change from baseline (Mean)
Standard Deviation: 12.62

Red Blood Cell Count

0.1
percentage change from baseline (Mean)
Standard Deviation: 0.42

Total Bilirubin

37.2
percentage change from baseline (Mean)
Standard Deviation: 46.02

White Blood Cell Count

-4.0
percentage change from baseline (Mean)
Standard Deviation: 16.69

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.

Cabaletta 30gr

-33.8
percentage change from baseline (Mean)
Standard Deviation: 34.89

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.

Cabaletta 30gr

0.1
Points on an 8 point scale (Mean)
Standard Deviation: 1.27

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.

Cabaletta 30gr

12.4
percentage change from baseline (Mean)
Standard Deviation: 16.87

Age, Continuous

59.5
years (Mean)
Standard Deviation: 8.34

Age, Categorical

Region of Enrollment

Sex: Female, Male

Overall Study

Cabaletta 30gr

Drop/Withdrawal Reasons

Cabaletta 30gr