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

Bioblast Pharma

Status and phase: Completed; Phase 2

Conditions: Machado-Joseph Disease / Spinocerebellar Ataxia 3

Treatments: Drug: Cabaletta for IV infusion once weekly during 24 weeks

Study type: Interventional

Funder types: Industry

Identifiers

NCT02147886

BB-MJD-201

Details and patient eligibility

About

• 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).

Enrollment: 15 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No 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).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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15 participants in 2 patient groups

Cabaletta 15gr

Experimental group

Description:

Cabaletta 15gr

Treatment:

Drug: Cabaletta for IV infusion once weekly during 24 weeks

Drug: Cabaletta for IV infusion once weekly during 24 weeks

Cabaletta 30gr

Experimental group

Description:

Cabaletta 30gr

Treatment:

Drug: Cabaletta for IV infusion once weekly during 24 weeks

Drug: Cabaletta for IV infusion once weekly during 24 weeks