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

NCT02147886 | Completed Phase 2

• 1 other identifier: BB-MJD-201
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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).

Conditions

Machado-Joseph Disease / Spinocerebellar Ataxia 3

Keywords

Bioblast, Randomized, parallel-group, and dose-controlled

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (1)

• Disease markers

  27 weeks

Other Outcomes (1)

• Biochemical marker

  27 weeks

Study Arms (2)

Cabaletta 15gr

EXPERIMENTAL

Cabaletta 15gr

Drug: Cabaletta for IV infusion once weekly during 24 weeks

Cabaletta 30gr

EXPERIMENTAL

Cabaletta 30gr

Drug: Cabaletta for IV infusion once weekly during 24 weeks

Interventions

Cabaletta for IV infusion once weekly during 24 weeks DRUG

Cabaletta for IV infusion once weekly

Cabaletta 15gr

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Bioblast Pharma Ltd.: lead

Study Sites (1)

Meir Medical Center

Kfar Saba, Israel

Location

MeSH Terms

Conditions

Machado-Joseph Disease

Condition Hierarchy (Ancestors)

Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Carlos Gordon, Prof.

  Meir Medical Center Kfar Saba Israel

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2014
• First Posted: May 28, 2014
• Study Start: July 1, 2014
• Primary Completion: October 1, 2016
• Study Completion: November 1, 2016
• Last Updated: November 22, 2016

Record last verified: 2016-11

Locations

IL (1)