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

• Sponsor: Bioblast Pharma Ltd. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02015481
• Collaborator: (none)
• Enrollment: 25
• Locations: 4
• Arm: 1
• Duration (Months): 26
• Patients Per Site: 6.3
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Condition or Disease	Intervention/Treatment	Phase
Oculopharyngeal Muscular Dystrophy	Drug: Cabaletta	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 25 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
• Study Start Date: Feb 1, 2014
• Actual Primary Completion Date: Apr 1, 2016
• Actual Study Completion Date: Apr 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cabaletta 30gr. weekly IV of Cabaletta 30gr.	Drug: Cabaletta

Outcome Measures

Primary Outcome Measures

1. Safety Lab Evaluations [24 weeks]

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

Secondary Outcome Measures

1. Drinking Test Score [24 weeks]

   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.

2. Videofluoroscopy (VFS) Score [24 Weeks]

   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.

3. SWAL-QOL, Swallowing Quality of Life Questionnaire [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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCLA	Los Angeles	California	United States	90095
2	Tahseen Mozaffar	Orange	California	United States	92868
3	Montreal Neurological Institute, McGill University	Montreal	Quebec	Canada	H3A 2B4
4	Hadassah medical center	Jerusalem		Israel	91120

Sponsors and Collaborators

• Bioblast Pharma Ltd.

Investigators

• Principal Investigator: Yoseph Caraco, M.D, Hadassah Medical Organization
• Principal Investigator: Bernard Brais, MD, McGill University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Bioblast Pharma Ltd.
• ClinicalTrials.gov Identifier: NCT02015481
• Other Study ID Numbers: BBCO-001
• First Posted: Dec 19, 2013
• Last Update Posted: Oct 17, 2017
• Last Verified: Sep 1, 2017

• Keywords provided by Bioblast Pharma Ltd.: Case control, safety, tolerability, efficacy.
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Oculopharyngeal

Study Results

Participant Flow

Arm/Group Title	Cabaletta 30gr
Arm/Group Description	weekly IV of Cabaletta 30gr Cabaletta
Period Title: Overall Study
STARTED	25
COMPLETED	24
NOT COMPLETED	1

Baseline Characteristics

Arm/Group Title	Cabaletta 30gr.
Arm/Group Description	weekly IV of Cabaletta 30gr. Cabaletta
Overall Participants	25
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	20 80%
>=65 years	5 20%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	59.5 (8.34)
Sex: Female, Male (Count of Participants)
Female	13 52%
Male	12 48%
Region of Enrollment (participants) [Number]
Canada	11 44%
Israel	14 56%

Outcome Measures

1. Primary Outcome

Title	Safety Lab Evaluations
Description	Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Time Frame	24 weeks

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Cabaletta 30gr.
Arm/Group Description	weekly IV of Cabaletta 30gr. Cabaletta
Measure Participants	24
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)

2. Secondary Outcome

Title	Drinking Test Score
Description	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

Outcome Measure Data

Analysis Population Description

22 patients had data available for analysis at week 24

Arm/Group Title	Cabaletta 30gr
Arm/Group Description	weekly IV of Cabaletta 30gr Cabaletta
Measure Participants	22
Mean (Standard Deviation) [percentage change from baseline]	-33.8 (34.89)

3. Secondary Outcome

Title	Videofluoroscopy (VFS) Score
Description	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

Outcome Measure Data

Analysis Population Description

11 patients had valid baseline data and week 24 data

Arm/Group Title	Cabaletta 30gr
Arm/Group Description	weekly IV of Cabaletta 30gr Cabaletta
Measure Participants	11
Mean (Standard Deviation) [Points on an 8 point scale]	0.1 (1.27)

4. Secondary Outcome

Title	SWAL-QOL, Swallowing Quality of Life Questionnaire
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Cabaletta 30gr
Arm/Group Description	weekly IV of Cabaletta 30gr Cabaletta
Measure Participants	24
Mean (Standard Deviation) [percentage change from baseline]	12.4 (16.87)

Adverse Events

Time Frame	28 weeks
Arm/Group Title	Cabaletta 30gr
Arm/Group Description	weekly IV of Cabaletta 30gr Cabaletta
All Cause Mortality
	Cabaletta 30gr
	Affected / at Risk (%)	# Events
Total	1/25 (4%)
Serious Adverse Events
	Cabaletta 30gr
	Affected / at Risk (%)	# Events
Total	2/25 (8%)
Infections and infestations
Urinary Tract Infection	1/25 (4%)	1
Respiratory, thoracic and mediastinal disorders
Pneumonia Aspiration	1/25 (4%)	1
Aspiration	1/25 (4%)	1
Other (Not Including Serious) Adverse Events
	Cabaletta 30gr
	Affected / at Risk (%)	# Events
Total	25/25 (100%)
Gastrointestinal disorders
Abdominal Pain	3/25 (12%)	3
Constipation	2/25 (8%)	2
Dysphagia	2/25 (8%)	2
Toothache	2/25 (8%)	2
General disorders
Administrative Site Bruise	4/25 (16%)	5
Asthenia	2/25 (8%)	2
Fatigue	4/25 (16%)	5
Influenza Like Illness	3/25 (12%)	3
Pyrexia	2/25 (8%)	2
Infections and infestations
Bacteriuria	2/25 (8%)	3
Nasopharyngitis	4/25 (16%)	5
Pharyngitis	2/25 (8%)	3
Urinary Tract Infection	3/25 (12%)	4
Injury, poisoning and procedural complications
Procedural Pain	10/25 (40%)	14
Musculoskeletal and connective tissue disorders
Arthralgia	2/25 (8%)	3
Back Pain	7/25 (28%)	10
Muscle Fatigue	3/25 (12%)	4
Musculoskeletal Pain	5/25 (20%)	5
Myalgia	3/25 (12%)	3
Pain in Extremity	3/25 (12%)	3
Nervous system disorders
Headache	5/25 (20%)	10
Renal and urinary disorders
Elevated Urine Glucose Levels	13/25 (52%)	35
Respiratory, thoracic and mediastinal disorders
Cough	3/25 (12%)	3
Oropharyngeal Pain	2/25 (8%)	2

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.