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

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

• 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).

Study Overview

• Status: Completed
• Conditions: Machado-Joseph Disease / Spinocerebellar Ataxia 3
• Intervention / Treatment: Drug: Cabaletta for IV infusion once weekly during 24 weeks; Drug: Cabaletta for IV infusion once weekly during 24 weeks

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• Israel
  • Kfar Saba, Israel
    • Meir Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 73 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cabaletta 15gr	Drug: Cabaletta for IV infusion once weekly during 24 weeks; Cabaletta for IV infusion once weekly; Drug: Cabaletta for IV infusion once weekly during 24 weeks
Experimental: Cabaletta 30gr	Drug: Cabaletta for IV infusion once weekly during 24 weeks; Cabaletta for IV infusion once weekly; Drug: Cabaletta for IV infusion once weekly during 24 weeks

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Biochemical marker	Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B)	27 weeks

Collaborators and Investigators

• Sponsor: Bioblast Pharma Ltd.
• Investigators: Principal Investigator: Carlos Gordon, Prof., Meir Medical Center Kfar Saba Israel

Study record dates

Study Major Dates

• Study Start: July 1, 2014
• Primary Completion (Actual): October 1, 2016
• Study Completion (Actual): November 1, 2016

Study Registration Dates

• First Submitted: May 21, 2014
• First Submitted That Met QC Criteria: May 23, 2014
• First Posted (Estimate): May 28, 2014

Study Record Updates

• Last Update Posted (Estimate): November 22, 2016
• Last Update Submitted That Met QC Criteria: November 21, 2016
• Last Verified: November 1, 2016

More Information

Terms related to this study

• Keywords: Bioblast, Randomized, parallel-group, and dose-controlled
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Machado-Joseph Disease
• Other Study ID Numbers: BB-MJD-201