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Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease

Primary Purpose

Machado-Joseph Disease / Spinocerebellar Ataxia 3

Status
Completed
Phase
Phase 2
Locations
Israel
Study Type
Interventional
Intervention
Cabaletta for IV infusion once weekly during 24 weeks
Cabaletta for IV infusion once weekly during 24 weeks
Sponsored by
Bioblast Pharma Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Machado-Joseph Disease / Spinocerebellar Ataxia 3 focused on measuring Bioblast, Randomized, parallel-group, and dose-controlled

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Men and women, 18 - 75 years
  2. Clinically diagnosed as Machado-Joseph disease/Spinocerebellar ataxia 3 confirmed by genetic testing
  3. With disease stage 2 or less
  4. Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
  5. Body Mass Index (BMI) ≤32 kg/m2.
  6. Ability to ambulate with or without assistance

Exclusion Criteria:

  1. Diabetes mellitus type 1 or 2
  2. Other major diseases
  3. Uncontrolled heart disease, chronic heart failure (CHF).
  4. Other neurological diseases.
  5. Ataxia derived from any other cause than genetically-confirmed spinocerebellar ataxia
  6. Presence of psychosis, bipolar disorder, untreated depression
  7. History of malignancy (except non-invasive skin malignancy).

Sites / Locations

  • Meir Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Cabaletta 15gr

Cabaletta 30gr

Arm Description

Cabaletta 15gr

Cabaletta 30gr

Outcomes

Primary Outcome Measures

Adverse events
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Physical examination
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Vital signs
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
12-lead ECG
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Safety laboratory tests
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

Secondary Outcome Measures

Disease markers
Changes in disease markers will be assessed based on the following assessments: Scale for the Assessment and Rating of Ataxia (SARA); Neurological Examination Score for Spinocerebellar Ataxia (NESSCA); Change in BMI - screening, spinocerebellar Ataxia Functional Tests; quality of life

Full Information

First Posted
May 21, 2014
Last Updated
November 21, 2016
Sponsor
Bioblast Pharma Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT02147886
Brief Title
Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
Official Title
A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease
Study Type
Interventional

2. Study Status

Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
July 2014 (undefined)
Primary Completion Date
October 2016 (Actual)
Study Completion Date
November 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioblast Pharma Ltd.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Machado-Joseph Disease / Spinocerebellar Ataxia 3
Keywords
Bioblast, Randomized, parallel-group, and dose-controlled

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cabaletta 15gr
Arm Type
Experimental
Arm Description
Cabaletta 15gr
Arm Title
Cabaletta 30gr
Arm Type
Experimental
Arm Description
Cabaletta 30gr
Intervention Type
Drug
Intervention Name(s)
Cabaletta for IV infusion once weekly during 24 weeks
Intervention Description
Cabaletta for IV infusion once weekly
Intervention Type
Drug
Intervention Name(s)
Cabaletta for IV infusion once weekly during 24 weeks
Primary Outcome Measure Information:
Title
Adverse events
Description
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time Frame
28 weeks
Title
Physical examination
Description
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time Frame
28 weeks
Title
Vital signs
Description
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time Frame
28weeks
Title
12-lead ECG
Description
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time Frame
28weeks
Title
Safety laboratory tests
Description
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time Frame
28weeks
Secondary Outcome Measure Information:
Title
Disease markers
Description
Changes in disease markers will be assessed based on the following assessments: Scale for the Assessment and Rating of Ataxia (SARA); Neurological Examination Score for Spinocerebellar Ataxia (NESSCA); Change in BMI - screening, spinocerebellar Ataxia Functional Tests; quality of life
Time Frame
27 weeks
Other Pre-specified Outcome Measures:
Title
Biochemical marker
Description
Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B)
Time Frame
27 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Men and women, 18 - 75 years Clinically diagnosed as Machado-Joseph disease/Spinocerebellar ataxia 3 confirmed by genetic testing With disease stage 2 or less Stable doses of all medications for 30 days prior to study entry and for the duration of the study. Body Mass Index (BMI) ≤32 kg/m2. Ability to ambulate with or without assistance Exclusion Criteria: Diabetes mellitus type 1 or 2 Other major diseases Uncontrolled heart disease, chronic heart failure (CHF). Other neurological diseases. Ataxia derived from any other cause than genetically-confirmed spinocerebellar ataxia Presence of psychosis, bipolar disorder, untreated depression History of malignancy (except non-invasive skin malignancy).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carlos Gordon, Prof.
Organizational Affiliation
Meir Medical Center Kfar Saba Israel
Official's Role
Principal Investigator
Facility Information:
Facility Name
Meir Medical Center
City
Kfar Saba
Country
Israel

12. IPD Sharing Statement

Learn more about this trial

Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease

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