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NAD+ precursor supplementation in Friedreich ataxia (NCT04817111)

Research clinicians at the Children’s Hospital of Philadelphia (CHOP) are enrolling 10 participants in a study to evaluate safety, tolerability, and effects on cardiac and skeletal muscle bioenergetics of compound MIB-626, which is an NAD+ precursor. This Phase 2 study will be enrolling 10 participants and is open label, meaning that all participants will be taking the compound.

NAD+, or nicotinamide adenine dinucleotide, has two general functions in the human body: helping to generate energy in mitochondria and working as a helper molecule for proteins that regulate other cellular functions.

The study investigators plan to complete enrollment by April 10, 2022.

Eligibility criteria include:

Inclusion:
-  Genetically confirmed diagnosis of FA
-  Age 18-64 years old

Exclusion:
-  Concurrent use of Vit B3 supplements
-  Cardiac ejection fraction less than 40%
-  Contraindication to MRI (e.g., spinal rods)
-  Involvement in another investigational drug study (must be >4 weeks out from another trial)

Participation is this study involves completion of two (2) in-person clinic visits, at the beginning and end of the 14-day study. At the clinic visits, you will be asked to complete blood testing and MRI scans.

If you are interested in learning more about this study, please contact Anna DeDio at Children’s Hospital of Philadelphia through email dedioa@email.chop.edu or by phone (267)-425-1998.

 

TRACK-FA study: bioimaging of brain and spinal cord

TRACK-FA is a natural history study to investigate brain and spinal cord changes in individuals with Friedreich ataxia. If imaging differences in the central nervous system are correlated with progression of FA, these differences could be used in future clinical trials to monitor treatment outcome.

Study Website
TRACK-FA Informational Webinar


There are 3 study visits, each approximately 12 months apart. At each study visit, participants will be asked to complete -
  • Neurologic and functional testing
  • Blood draw
  • Brain and spinal cord MRI scan


Inclusion criteria (partial):
  • Age >5 years with genetically confirmed diagnosis of FA.
  • GAA repeat expansion >55 in length on both alleles of the FXN gene or GAA repeat expansion on one allele and another mutation type on the other allele (e.g., point mutation, gene deletion).
  • Age of disease onset less than 25 years.
  • Disease duration less than 25 years.
  • Able to read and converse in English.


Exclusion criteria (partial):
  • MRI contraindications such as pacemaker, other metallic surgical implants, metallic braces.
  • Pregnancy
  • Other ongoing medical conditions, including psychiatric and neurological diagnoses (speak with the site investigator).


Additional information can be found on the clinicaltrials.gov website (NCT04349514)

Compensation is provided for time and travel expenses.

Track-FA Newsletters
July 2022 Newsletter


Sites open for enrollment

University of Florida, Gainesville
Principal Investigators: Sub H Subramony, MD, Thomas Mareci, PhD, Manuela Corti, PhD
Study coordinator: Samantha Norman
Email: Samantha.Norman@peds.ufl.edu
Phone: (352) 273-8218

University of Minnesota, Minneapolis
Principal Investigators: Pierre-Gilles Henry, PhD, Christophe Lenglet, PhD
Study coordinator: Diane Hutter
Email: hutte019@umn.edu
Phone: (612) 625-2350
More information about this team

Children’s Hospital of Philadelphia
Principal investigators: William Gaetz, PhD, Timothy PL Robets, PhD, David Lynch, MD, PhD
Study coordinator: Syama Patel
Email: patels60@chop.edu
Phone: 267-425-1153

McGill University, Montréal, Canada
Principal Investigators: Massimo Pandolfo, MD; Alain Dagher, MD; David Rudko, PhD
Study coordinator: Martin Chevrier
Email: martin.chevrier2@mcgill.ca
Phone: +1 514-398-2538

Monash University, Clayton, Victoria, Australia
Principal Investigators: Nellie Georgiou-Karistianis, PhD, Ian Harding, PhD, Louise Corben, PhD, Martin Delatycki, PhD
Study coordinator: Helena Bujalka
Email: helena.bujalka@monash.edu
Phone: +61 3 9905 3487

Department of Neurology, RWTH Aachen University
Aachen, Germany
Principal Investigators: Kathrin Reetz, MD, Imis Dogan, PhD, Sandro Romanzetti, PhD, Jörg B Schulz, MD
Study coordinator: Dr. Imis Dogan
Email: idogan@ukaachen.de
Phone: +49-241-80 80719

University of Campinas, Brazil
Principal investigators: Marcondes C. França, MD, PhD and Thiago JR Rezende, PhD
Study coordinator: Dr. Fernanda Bittar
Email: bittarfernanda1@gmail.com
Phone: +55 19 98128004

Exercise/NAD+ study at CHOP & Penn

Exercise/NAD+ study

Research clinicians at the Children’s Hospital of Philadelphia and University of Pennsylvania are enrolling participants in a study to evaluate the effects of a dietary supplement combined with exercise. The primary outcome measure is changes in aerobic capacity, which is the body’s ability to take in, transport and use oxygen during exercise.

This study will be enrolling over 4 years, starting in 2020, with a target enrollment of 72 participants. Compensation is provided for study time.

Eligibility criteria include:

  • Genetically-confirmed diagnosis of FA
  • Age 10-40 years old
  • Able to complete exercise testing
Participation is this study involves completion of two (2) in-person clinic visits, each lasting 2 days, over a 12-week period. During the study period, you will be asked to complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.

If you are interested in learning more about this study, please contact Anna DeDio through email dedioa@email.chop.edu or by phone (267)-425-1998.

Instrumented Data Exchange for Ataxia (IDEA study)

Participants are needed for a research study to test body-worn sensors to measure movement during simple tests of coordination. The goal of this study is to evaluate progression and severity of ataxia.The outcomes of this study are critical for allowing remote data collection during future clinical trials.

Eligibility criteria include:

  • Early-onset participants - 12-30 years of age with FA-onset between age 5-25
  • Late-onset participants - 26-60 years of age with FA-onset between age 25-40
  • Be able to walk 10-yards independently, without an assistive device.
  • Be able to sit and stand unassisted for at least 30 seconds.
  • Participants can be involved in an investigational drug trial and still be eligible for the IDEA study.
Participants can be involved in an investigational drug trial and still be eligible for the IDEA study.

The study involves visits every 6 months over 2 years at a clinic site. Involvement in the study provides an Apple watch.

Compensation is available for travel to study sites.

Participants are needed at all study sites in Baltimore (Johns Hopkins), Boston (MGH), Chicago (University of Chicago), Los Angeles (UCLA) or Portland (Oregon Health & Science University).

For additional eligibility criteria, please contact the site study coordinator:

University of Chicago
Study coordinator: Hannah Casey
Email: hannahcasey@uchicago.edu
Phone: (773) 702-4610

Oregon Health & Sciences University
Study coordinator: Austin Prewitt
Email: prewitta@ohsu.edu

Johns Hopkins Medical Institute
Study coordinator: Michelle Joyce
Email: mjoyce14@jhmi.edu

Massachusetts General Hospital
Study coordinator: Brigitte Jacoby
Email: BJACOBY@mgh.harvard.edu

UCLA
Study coordinator: Aaron Fisher
Email: ADFisher@mednet.ucla.edu

Burke Institute - Seated Exercise Study

Home Based Tele-Exercise for People with Chronic Neurological Impairments
The seated exercise study will include:

  • Adaptive Boxing
  • Adaptive Power Posture
  • Adaptive High-Intensity Interval Training (HIIT)
Who can participate?
  • 18-75 years of age
  • Diagnosis of a chronic neurological impairment (at least 6 months post injury)
  • Ability to commit to 12-week program (3 times a week)
  • Medically stable, no contraindications to exercise
  • Access to high speed internet on a device with webcam
  • Currently exercising two (2) days or less per week
  • Can follow instructions in English
More information is available below.


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