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

Phase 2

Completed

• Conditions: Machado-Joseph Disease / Spinocerebellar Ataxia 3
• Interventions: Drug: Cabaletta for IV infusion once weekly during 24 weeks

• Registration Number: NCT02147886
• Lead Sponsor: Bioblast Pharma Ltd.

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-05-21
• Study First Submitted QC: 2014-05-23
• Study First Posted: 2014-05-28
• Study Start: 2014-07
• Primary Completion: 2016-10
• Status Verified: 2016-11
• Study Completion: 2016-11
• Last Update Submitted: 2016-11-21
• Last Update Posted: 2016-11-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cabaletta 15gr	Cabaletta for IV infusion once weekly during 24 weeks	Cabaletta 15gr
Cabaletta 30gr	Cabaletta for IV infusion once weekly during 24 weeks	Cabaletta 30gr

Primary Outcome Measures

Name	Time	Method
Safety laboratory tests	28weeks	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	28weeks	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	28weeks	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	28 weeks	Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Adverse events	28 weeks	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

Name	Time	Method
Disease markers	27 weeks	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

Trial Locations

Locations (1)

Meir Medical Center; 🇮🇱 Kfar Saba, Israel