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

Study Overview
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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).

Official Title
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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

Conditions
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Machado-Joseph Disease / Spinocerebellar Ataxia 3

Intervention / Treatment
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* Drug: Cabaletta for IV infusion once weekly during 24 weeks
* Drug: Cabaletta for IV infusion once weekly during 24 weeks


Participation Criteria
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Eligibility Criteria
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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).

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cabaletta 15gr<br>Cabaletta 15gr | Drug: Cabaletta for IV infusion once weekly during 24 weeks<br>* Cabaletta for IV infusion once weekly<br>|
| Experimental: Cabaletta 30gr<br>Cabaletta 30gr | Drug: Cabaletta for IV infusion once weekly during 24 weeks<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 28 weeks | 
| 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 | 28 weeks | 
| 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 | 28weeks | 
| 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 | 28weeks | 
| 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 | 28weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 27 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Bioblast, Randomized, parallel-group, and dose-controlled