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

NCT ID: NCT02147886

Last Updated: 2016-11-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-07-31

Study Completion Date

2016-11-30

Brief Summary

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* 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).

Detailed Description

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Conditions

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Machado-Joseph Disease / Spinocerebellar Ataxia 3

Keywords

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Bioblast, Randomized, parallel-group, and dose-controlled

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Cabaletta 15gr

Cabaletta 15gr

Group Type EXPERIMENTAL

Cabaletta for IV infusion once weekly during 24 weeks

Intervention Type DRUG

Cabaletta for IV infusion once weekly

Cabaletta 30gr

Cabaletta 30gr

Group Type EXPERIMENTAL

Cabaletta for IV infusion once weekly during 24 weeks

Intervention Type DRUG

Interventions

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Cabaletta for IV infusion once weekly during 24 weeks

Cabaletta for IV infusion once weekly

Intervention Type DRUG

Cabaletta for IV infusion once weekly during 24 weeks

Intervention Type DRUG

Eligibility Criteria

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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).
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bioblast Pharma Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Carlos Gordon, Prof.

Role: PRINCIPAL_INVESTIGATOR

Meir Medical Center Kfar Saba Israel

Locations

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Meir Medical Center

Kfar Saba, , Israel

Site Status

Countries

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Israel

Other Identifiers

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BB-MJD-201

Identifier Type: -

Identifier Source: org_study_id