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Completed

NCT Number: NCT02147886

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).

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Key information

Conditions

Machado-Joseph Disease / Spinocerebellar Ataxia 3 Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Machado-Joseph Disease Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Ataxias Spinocerebellar Degenerations

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Meir Medical Center

Kfar Saba, Israel

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

Cabaletta for IV infusion once weekly during 24 weeks

Drug

Cabaletta for IV infusion once weekly

Primary outcomes

  1. Adverse events

    Time frame: 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

  2. Physical examination

    Time frame: 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

  3. Vital signs

    Time frame: 28weeks

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

  4. 12-lead ECG

    Time frame: 28weeks

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

  5. Safety laboratory tests

    Time frame: 28weeks

    Safety will be evaluated on the basis of the following assessments:

    Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations

Secondary outcomes

  1. Disease markers

    Time frame: 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

Other outcomes

  1. Biochemical marker

    Time frame: 27 weeks

    Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B)

Sponsors and collaborators

Lead sponsor

Bioblast Pharma Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2016
Study completion
2016
First posted
May 28, 2014
Registry last updated
Nov 22, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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