NCT02015481

Brief Summary

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Feb 2014

Geographic Reach
3 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 8, 2013

Completed
11 days until next milestone

First Posted

Study publicly available on registry

December 19, 2013

Completed
1 month until next milestone

Study Start

First participant enrolled

February 1, 2014

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2016

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

August 23, 2017

Completed
Last Updated

October 17, 2017

Status Verified

September 1, 2017

Enrollment Period

2.2 years

First QC Date

December 8, 2013

Results QC Date

July 21, 2017

Last Update Submit

September 15, 2017

Conditions

Oculopharyngeal Muscular Dystrophy

Keywords

Case control, safety, tolerability, efficacy.

Outcome Measures

Primary Outcomes (1)

  • Safety Lab Evaluations

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

    24 weeks

Secondary Outcomes (3)

  • Drinking Test Score

    24 weeks

  • Videofluoroscopy (VFS) Score

    24 Weeks

  • SWAL-QOL, Swallowing Quality of Life Questionnaire

    28 weeks

Study Arms (1)

Cabaletta 30gr.

EXPERIMENTAL

weekly IV of Cabaletta 30gr.

Drug: Cabaletta

Interventions

CabalettaDRUG
Cabaletta 30gr.

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Males and females
  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

UCLA

Los Angeles, California, 90095, United States

Location

Tahseen Mozaffar

Orange, California, 92868, United States

Location

Montreal Neurological Institute, McGill University

Montreal, Quebec, H3A 2B4, Canada

Location

Hadassah medical center

Jerusalem, 91120, Israel

Location

MeSH Terms

Conditions

Muscular Dystrophy, Oculopharyngeal

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Warren Wasiewski
Organization
Bioblast Pharma
Warren.wasiewski@bioblastpharma.com
717-368-0780

Study Officials

  • Yoseph Caraco, M.D

    Hadassah Medical Organization

    PRINCIPAL INVESTIGATOR

  • Bernard Brais, MD

    McGill University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 8, 2013

First Posted

December 19, 2013

Study Start

February 1, 2014

Primary Completion

April 1, 2016

Study Completion

April 1, 2016

Last Updated

October 17, 2017

Results First Posted

August 23, 2017

Record last verified: 2017-09

Locations

US(2)CA(1)IL(1)