Anybody knows anything about this clinical trial?, Is the first news I got from the use of this medicine to the FA.
It seems that FARA is the sponsor along with Pfizer. I have looked on the website of FARA but I have not been able to find any information.
What mechanism of action is? Prevents the disease or just improving symptoms?
Greetings
 
Juan Carlos
 
°°°°°
 
Added by Gian Piero:
 
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

This study is not yet open for participant recruitment.
Verified by University of South Florida, December 2008

Sponsors and Collaborators:

University of South Florida
Friedreich's Ataxia Research Alliance


Pfizer

Information provided by:

University of South Florida

ClinicalTrials.gov Identifier:

NCT00803868

  Purpose

The purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia.

 


Condition

Intervention

Phase

Friedreich's Ataxia

Drug: varenicline
Drug: placebo

Phase II
Phase III

 


Genetics Home Reference related topics: 

familial encephalopathy with neuroserpin inclusion bodies    Friedreich ataxia    mitochondrial neurogastrointestinal encephalopathy disease  

 


MedlinePlus related topics: 

Friedreich's Ataxia  

 


Drug Information available for: 

Varenicline  


U.S. FDA Resources

Study Type: 

Interventional

Study Design: 

Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study

 

 

 

Further study details as provided by University of South Florida:

 

Primary Outcome Measures:

Friedreich Ataxia Rating Scale (FARS) [ Time Frame: 9 weeks ] [ Designated as safety issue: No ]
 

Estimated Enrollment: 

32

Study Start Date: 

January 2009

Estimated Primary Completion Date: 

June 2009 (Final data collection date for primary outcome measure)

 


Arms

Assigned Interventions

1: Experimental

Varenicline

Drug: varenicline

up to 1mg po bid for 9 weeks

2: Placebo Comparator

Placebo

Drug: placebo

placebo matching study drug up to 1mg po bid

 


  Eligibility

Ages Eligible for Study: 

18 Years to 80 Years

Genders Eligible for Study: 

Both

Accepts Healthy Volunteers: 

No


Criteria

Inclusion Criteria:

Outpatients with FA diagnosed by confirmed by genetic testing.
Age 18 years to 80 years.
Women who are not pregnant or breast feeding, and who do not intend to become pregnant. Women of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at entry into the study.
4.      CBC, CMP, and diabetes lab results not indicative of clinically relevant abnormalities (results within the past 6 months prior to screening). These would include but are not limited to:

Electrolytes (Chloride, Sodium, Potassium) within laboratory defined normal limits.

Hemoglobin, white cell count, platelet count and fasting glucose within laboratory defined normal limits. Creatinine must be (?1.5 mg/dl).

ALT (6-40 u/l) and AST (10-30 u/l) must be less than 2 times normal limit

Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
Patient permission (informed consent).
Ambulatory status: Half of the enrolled patients must be able to ambulate with or without assistance; half of the enrolled patients must be non-ambulatory.
Exclusion Criteria:

Any unstable illness that in the investigator's opinion preclude participation in this study.
Use of another investigational product within the past 28 days.
Patients with a history of substance abuse.
Presence of preexisting psychiatric illness (specifically schizophrenia, bipolar disorder, or history of suicide attempt).
Presence of diabetes (as determined by fasting blood glucose labs within the past 6 months).
Presence of clinically significant cardiac disease (as determined by the investigator based on EKG and echocardiogram results within the past 6 months). Specifically, patients with an ejection fraction <40% or a prolonged QT interval (>50% of cycle duration) will be excluded. If abnormalities are noted on the EKG or echocardiogram, the patient will be eligible IF they provide clearance from a cardiologist.
Presence of current uncontrolled depression as measured by PHQ9 (criteria for depression include all of the following to be present: At least one of the first two questions on PHQ9 endorsed as positive (little pleasure, feeling depressed) indicating the symptom has been present more than half the time in the past two weeks; Question 10 about difficulty at work or home or getting along with others should be answered at least "somewhat difficult."; and the total score ? 10.
Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
Patients who currently smoke or have smoked within the past 12 months (Smoking will be defined as having smoked any substance even on a single occasion).
Dementia or other psychiatric illness that prevents the patient from giving informed consent (MMSE less than 25).
Legal incapacity or limited legal capacity.
Presence of severe renal disease (creatinine >1.6) or hepatic disease (AST or ALT>2x times normal) (as evidenced by labs reported within the past 6 months).
Abnormal WBC hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00803868

Contacts

 

Contact: Kelly Sullivan, MSPH   

813-974-5909   

kbarber@...   


Locations

 

United States, Florida

 

University of South Florida   

 

 

 

      Tampa, Florida, United States, 33612

 

 

 

 

 

 

United States, Pennsylvania

 

 

The Children's Hospital of Philadelphia   

 

 

 

 

      Philadelphia, Pennsylvania, United States, 19104


Sponsors and Collaborators

 

University of South Florida

 

Friedreich's Ataxia Research Alliance

 

Pfizer

  More Information

 

Responsible Party: 

University of South Florida ( Theresa Zesiewicz, MD )

Study ID Numbers: 

6

First Received: 

December 4, 2008

Last Updated: 

December 5, 2008

ClinicalTrials.gov Identifier: 

NCT00803868

Health Authority: 

United States: Food and Drug Administration

 

Study placed in the following topic categories:

Metabolic Diseases

Spinal Cord Diseases

Central Nervous System Diseases

Mitochondrial Diseases

Brain Diseases

Neurodegenerative Diseases

Dyskinesias

Signs and Symptoms

Heredodegenerative Disorders, Nervous System


 

Friedreich Ataxia

Genetic Diseases, Inborn

Friedreich ataxia

Ataxia

Neurologic Manifestations

Metabolic disorder

Cerebellar Diseases

Spinocerebellar Degenerations


 

Additional relevant MeSH terms:

Nervous System Diseases

 

ClinicalTrials.gov processed this record on December 08, 2008

 

 

 

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.

Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!

https://www.amazon.com/Legacy-Marie-Schlau-collective-Friedreichs-ebook/dp/B01N28AFWZ

 

Research projects currently being financed by BabelFAmily

Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:

1) Gene Therapy for Friedreich's Ataxia research project:

https://www.irbbarcelona.org/en/news/international-patient-advocates-partner-to-fund-spanish-gene-therapy-project-to-treat

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

2) Frataxin delivery research project:

https://www.irbbarcelona.org/en/news/new-research-front-to-tackle-friedreichs-ataxia
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.

The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.

 

 

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