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

• Status: Completed
• Trial end date: 2016-11-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioblast Pharma Ltd.

Criteria

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).