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

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ClinicalTrials.gov Identifier: NCT02147886
Recruitment Status : Completed
First Posted : May 28, 2014
Last Update Posted : November 22, 2016
Sponsor:
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Machado-Joseph Disease / Spinocerebellar Ataxia 3 Drug: Cabaletta for IV infusion once weekly during 24 weeks Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Study Start Date : July 2014
Actual Primary Completion Date : October 2016
Actual Study Completion Date : November 2016


Arm Intervention/treatment
Experimental: Cabaletta 15gr
Cabaletta 15gr
Drug: Cabaletta for IV infusion once weekly during 24 weeks
Cabaletta for IV infusion once weekly

Experimental: Cabaletta 30gr
Cabaletta 30gr
Drug: Cabaletta for IV infusion once weekly during 24 weeks



Primary Outcome Measures :
  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 Outcome Measures :
  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 Outcome Measures:
  1. Biochemical marker [ Time Frame: 27 weeks ]
    Assessment of disease biochemical marker neuron specific enolase (NSE) and protein S 100 B (S100B)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02147886


Locations
Israel
Meir Medical Center
Kfar Saba, Israel
Sponsors and Collaborators
Bioblast Pharma Ltd.
Investigators
Principal Investigator: Carlos Gordon, Prof. Meir Medical Center Kfar Saba Israel

Responsible Party: Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier: NCT02147886     History of Changes
Other Study ID Numbers: BB-MJD-201
First Posted: May 28, 2014    Key Record Dates
Last Update Posted: November 22, 2016
Last Verified: November 2016

Keywords provided by Bioblast Pharma Ltd.:
Bioblast, Randomized, parallel-group, and dose-controlled

Additional relevant MeSH terms:
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Cerebellar Ataxia
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Ataxia
Dyskinesias
Neurologic Manifestations
Genetic Diseases, Inborn