Trial Outcomes & Findings for Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients (NCT NCT02015481)

NCT ID: NCT02015481

Last Updated: 2017-10-17

Results Overview

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

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 25 participants
• Primary outcome timeframe: 24 weeks
• Results posted on: 2017-10-17

Participant Flow

Participant milestones

Measure	Cabaletta 30gr weekly IV of Cabaletta 30gr Cabaletta
Overall Study STARTED	25
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Cabaletta 30gr weekly IV of Cabaletta 30gr Cabaletta
Overall Study Death	1

Baseline Characteristics

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

Baseline characteristics by cohort

Measure	Cabaletta 30gr. n=25 Participants weekly IV of Cabaletta 30gr. Cabaletta
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants
Age, Continuous	59.5 years STANDARD_DEVIATION 8.34 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Region of Enrollment Canada	11 participants n=5 Participants
Region of Enrollment Israel	14 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Population: 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.

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

Outcome measures

Measure	Cabaletta 30gr. n=24 Participants weekly IV of Cabaletta 30gr. Cabaletta
Safety Lab Evaluations White Blood Cell Count	-4.0 percentage change from baseline Standard Deviation 16.69
Safety Lab Evaluations Red Blood Cell Count	0.1 percentage change from baseline Standard Deviation 0.42
Safety Lab Evaluations Hemoglobin	0.2 percentage change from baseline Standard Deviation 7.98
Safety Lab Evaluations Hematocrit	1.1 percentage change from baseline Standard Deviation 9.28
Safety Lab Evaluations Platelets	-0.5 percentage change from baseline Standard Deviation 13.42
Safety Lab Evaluations Alanine Aminotransferase	-0.8 percentage change from baseline Standard Deviation 34.59
Safety Lab Evaluations Aspartate Aminotransferase	-5.7 percentage change from baseline Standard Deviation 30.93
Safety Lab Evaluations Creatinine phosphokinase	6.8 percentage change from baseline Standard Deviation 37.12
Safety Lab Evaluations Gamma-Glutamyl Transferase	9.3 percentage change from baseline Standard Deviation 28.73
Safety Lab Evaluations Total Bilirubin	37.2 percentage change from baseline Standard Deviation 46.02
Safety Lab Evaluations Glucose	12 percentage change from baseline Standard Deviation 15.93
Safety Lab Evaluations Lactose Dehydrogenase	23 percentage change from baseline Standard Deviation 0.9
Safety Lab Evaluations Prothrombin Time	7.1 percentage change from baseline Standard Deviation 12.62
Safety Lab Evaluations International Normalized Ratio	1.3 percentage change from baseline Standard Deviation 8.48

SECONDARY outcome

Timeframe: 24 weeks

Population: 22 patients had data available for analysis at week 24

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.

Outcome measures

Measure	Cabaletta 30gr. n=22 Participants weekly IV of Cabaletta 30gr. Cabaletta
Drinking Test Score	-33.8 percentage change from baseline Standard Deviation 34.89

SECONDARY outcome

Timeframe: 24 Weeks

Population: 11 patients had valid baseline data and week 24 data

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.

Outcome measures

Measure	Cabaletta 30gr. n=11 Participants weekly IV of Cabaletta 30gr. Cabaletta
Videofluoroscopy (VFS) Score	0.1 Points on an 8 point scale Standard Deviation 1.27

SECONDARY outcome

Timeframe: 28 weeks

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.

Outcome measures

Measure	Cabaletta 30gr. n=24 Participants weekly IV of Cabaletta 30gr. Cabaletta
SWAL-QOL, Swallowing Quality of Life Questionnaire	12.4 percentage change from baseline Standard Deviation 16.87

Adverse Events

Cabaletta 30gr

Serious events: 2 serious events

Other events: 25 other events

Deaths: 1 deaths

Serious adverse events

Measure	Cabaletta 30gr n=25 participants at risk weekly IV of Cabaletta 30gr Cabaletta
Infections and infestations Urinary Tract Infection	4.0% 1/25 • Number of events 1 • 28 weeks
Respiratory, thoracic and mediastinal disorders Pneumonia Aspiration	4.0% 1/25 • Number of events 1 • 28 weeks
Respiratory, thoracic and mediastinal disorders Aspiration	4.0% 1/25 • Number of events 1 • 28 weeks

Other adverse events

Measure	Cabaletta 30gr n=25 participants at risk weekly IV of Cabaletta 30gr Cabaletta
Gastrointestinal disorders Abdominal Pain	12.0% 3/25 • Number of events 3 • 28 weeks
Gastrointestinal disorders Constipation	8.0% 2/25 • Number of events 2 • 28 weeks
Gastrointestinal disorders Dysphagia	8.0% 2/25 • Number of events 2 • 28 weeks
Gastrointestinal disorders Toothache	8.0% 2/25 • Number of events 2 • 28 weeks
General disorders Administrative Site Bruise	16.0% 4/25 • Number of events 5 • 28 weeks
General disorders Asthenia	8.0% 2/25 • Number of events 2 • 28 weeks
General disorders Fatigue	16.0% 4/25 • Number of events 5 • 28 weeks
General disorders Influenza Like Illness	12.0% 3/25 • Number of events 3 • 28 weeks
General disorders Pyrexia	8.0% 2/25 • Number of events 2 • 28 weeks
Infections and infestations Bacteriuria	8.0% 2/25 • Number of events 3 • 28 weeks
Infections and infestations Nasopharyngitis	16.0% 4/25 • Number of events 5 • 28 weeks
Infections and infestations Pharyngitis	8.0% 2/25 • Number of events 3 • 28 weeks
Infections and infestations Urinary Tract Infection	12.0% 3/25 • Number of events 4 • 28 weeks
Injury, poisoning and procedural complications Procedural Pain	40.0% 10/25 • Number of events 14 • 28 weeks
Musculoskeletal and connective tissue disorders Arthralgia	8.0% 2/25 • Number of events 3 • 28 weeks
Musculoskeletal and connective tissue disorders Back Pain	28.0% 7/25 • Number of events 10 • 28 weeks
Musculoskeletal and connective tissue disorders Muscle Fatigue	12.0% 3/25 • Number of events 4 • 28 weeks
Musculoskeletal and connective tissue disorders Musculoskeletal Pain	20.0% 5/25 • Number of events 5 • 28 weeks
Musculoskeletal and connective tissue disorders Myalgia	12.0% 3/25 • Number of events 3 • 28 weeks
Musculoskeletal and connective tissue disorders Pain in Extremity	12.0% 3/25 • Number of events 3 • 28 weeks
Nervous system disorders Headache	20.0% 5/25 • Number of events 10 • 28 weeks
Renal and urinary disorders Elevated Urine Glucose Levels	52.0% 13/25 • Number of events 35 • 28 weeks
Respiratory, thoracic and mediastinal disorders Cough	12.0% 3/25 • Number of events 3 • 28 weeks
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	8.0% 2/25 • Number of events 2 • 28 weeks

Additional Information

Warren Wasiewski

Bioblast Pharma

Phone: 717-368-0780

Email: Warren.wasiewski@bioblastpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place