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Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia

Primary Purpose

Ataxia, Chorea, Dystonia

Status
Completed
Phase
Phase 4
Locations
Israel
Study Type
Interventional
Intervention
amantadine sulphate
Sponsored by
Sheba Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Ataxia

Eligibility Criteria

4 Years - 50 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • diagnosis of A-T
  • significant functional disability
  • age over 4 years

Exclusion Criteria:

  • major comorbidity: active malignancy requiring chemotherapy, kidney or liver failure
  • sexually active
  • known hypersensitivity to amantadine
  • previous treatment with amantadine in the 2 months preceding the study

Sites / Locations

  • Sheba Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

amantadine (PKMERZ)

Arm Description

Outcomes

Primary Outcome Measures

improvement in ataxia

Secondary Outcome Measures

improvement in extrapyramidal movement disorder

Full Information

First Posted
July 30, 2009
Last Updated
June 26, 2011
Sponsor
Sheba Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00950196
Brief Title
Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia
Official Title
The Effect of Amantadine on Movement Disorder in Ataxia-Telangiectasia
Study Type
Interventional

2. Study Status

Record Verification Date
June 2011
Overall Recruitment Status
Completed
Study Start Date
November 2008 (undefined)
Primary Completion Date
November 2009 (Actual)
Study Completion Date
November 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Sheba Medical Center

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Ataxia-Telangiectasia A-T is a neurodegenerative disorder of the cerebellum, manifesting with ataxia, as well as extrapyramidal features. Treatment of A-T is discouraging, since no treatment seems to change the course of disease, but improvement can be achieved by symptomatic treatment of the bothersome movement disorder . While various dopaminergic agents are occasionally used, reports of benefit are rather sparse and anecdotal. Amantadine, a well known drug used in influenza as well as movement disorder of Parkinson, has been proved to improve various other types of movement disorder as ataxia, chorea, dystonia, akinesia and attention span. The purpose of this study is to investigate weather amantadine sulphate improves ataxia and the movement disorder (bradykinesia, parkinsonism, dystonia, chorea), as well as the general well being in patients with A-T.
Detailed Description
Ataxia-Telangiectasia (A-T) is a complex multisystem disorder with neurodegenerative course, immune deficiency, and tendency to develop malignancies .The clinical picture includes progressive cerebellar ataxia along with a movement disorder (chorea, dystonia or bradykinesia) that may be even more disabling than the ataxia . Fatigability, drooling and reduced stamina are other major concerns. Disease course is devastating: towards the second decade of life the affected children are usually bound to wheelchair and survival beyond the second decade of life is rare. Treatment of A-T is discouraging, since no treatment seems to change the course of disease, but improvement can be achieved by symptomatic treatment of the bothersome movement disorder . While various dopaminergic agents are occasionally used, reports of benefit are rather sparse and anecdotal. Amantadine is a dopaminergic agent approved for prophylaxis of influenza (in children over 1 year of age and adults) as well as for extrapyramidal disorders in adults: Parkinson disease and drug induced dyskinesias . Amantadine increases dopaminergic transmission by inhibiting its synaptic uptake, as well as an antagonizing the striatal NMDA receptors). Additional conditions found to be improved with amantadine are: cerebellar ataxia, vigilance after brain trauma in adults and children ,attention deficit disorder in children, chorea and akinesia in Huntington's disease . Amantadine is an FDA approved drug for treatment and prevention of influenza, Parkinson disease and drug induced dyskinesia; it is approved for use in adults and children over 1 year of age. Dosage in children: 5 mg/kg body weight up to 8.8mg/kg/. Dosage in adults: 200 to 300 mg/day. The daily dosage should be divided into 2 to 3 daily portions. Amantadine is a safe drug with mild side effects: headache, decreased appetite, sedation, fatigue, abdominal pain, vomiting, insomnia, pedal edema and rash (4-10). Studies in children proved amantadine to be a safe and tolerable drug. Amantadine was administered to 24 healthy children with ADHD, aged 5-13 year old . Side effects were present in 13/24 and were usually mild: decreased appetite, headache, sedation, mild insomnia, vomiting, fatigue, abdominal pain. One subject dropped out because of headache. Six low response children after traumatic brain injury were treated with amantadine (concurrently with other medication) The drug was safe with relatively mild side effects: sedation, intermittent tremor, dizziness, but no serious side effects requiring discontinuation of the protocol. Study purpose The purpose of this study is to investigate weather amantadine sulphate improves ataxia and the movement disorder (bradykinesia, parkinsonism, dystonia, chorea), as well as the general well being in patients with A-T.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ataxia, Chorea, Dystonia, Parkinsonism, Fatigue

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
amantadine (PKMERZ)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
amantadine sulphate
Other Intervention Name(s)
PKMERZ
Intervention Description
amantadine 5/mg/kg for 2 month- tapered up during 2 weeks at 1 month possibility to increase dosage to 8 mg/kg or reduce it if there are side effects
Primary Outcome Measure Information:
Title
improvement in ataxia
Time Frame
2 month
Secondary Outcome Measure Information:
Title
improvement in extrapyramidal movement disorder
Time Frame
2 month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
50 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: diagnosis of A-T significant functional disability age over 4 years Exclusion Criteria: major comorbidity: active malignancy requiring chemotherapy, kidney or liver failure sexually active known hypersensitivity to amantadine previous treatment with amantadine in the 2 months preceding the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Andreea Nissenkorn, MD
Organizational Affiliation
Sheba Medical Center, Pediatric Neurology and National A-T Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Sheba Medical Center
City
Ramat Gan
Country
Israel

12. IPD Sharing Statement

Citations:
PubMed Identifier
22550086
Citation
Nissenkorn A, Hassin-Baer S, Lerman SF, Levi YB, Tzadok M, Ben-Zeev B. Movement disorder in ataxia-telangiectasia: treatment with amantadine sulfate. J Child Neurol. 2013 Feb;28(2):155-60. doi: 10.1177/0883073812441999. Epub 2012 May 1.
Results Reference
derived

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Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia

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