MedDataX Logo

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

NCT ID: NCT02015481

Last Updated: 2017-10-17

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-02-28

Study Completion Date

2016-04-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Oculopharyngeal Muscular Dystrophy

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Case control, safety, tolerability, efficacy.

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Cabaletta 30gr.

weekly IV of Cabaletta 30gr.

Group Type EXPERIMENTAL

Cabaletta

Intervention Type DRUG

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Cabaletta

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Bioblast Pharma Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Yoseph Caraco, M.D

Role: PRINCIPAL_INVESTIGATOR

Hadassah Medical Organization

Bernard Brais, MD

Role: PRINCIPAL_INVESTIGATOR

McGill University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

UCLA

Los Angeles, California, United States

Site Status

Tahseen Mozaffar

Orange, California, United States

Site Status

Montreal Neurological Institute, McGill University

Montreal, Quebec, Canada

Site Status

Hadassah medical center

Jerusalem, , Israel

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Canada Israel

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

BBCO-001

Identifier Type: -

Identifier Source: org_study_id