Methylprednisolone Treatment of Friedreich Ataxia

Status Completed

Clinical Trial Summary

This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA). The study will also explore if methylprednisolone has any effects on biomarkers associated with FRDA. All subjects in the study will receive the same steroid treatment.

Clinical Trial Description

Context: Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy. One of the hallmarks of FRDA is a deficiency of frataxin protein, causing dysregulation of iron metabolism, lack of detoxification, and increased iron bioavailability. The accumulation of iron in mitochondria leads to increased sensitivity to oxidative stress. A secondary inflammatory response has also been proposed to be present in FRDA, as revealed in autopsy studies and in the alteration of immune pathways in microarray analysis. Inflammation in FRDA raises the possibility of a therapeutic benefit from anti-inflammatory steroid treatment, as inflammation may directly underlie multiple complications of FRDA including cardiomyopathy. In support of this theory are clinical observations and patient self-reports of improvement of ataxia symptoms following the prescription of steroids for indications other than the primary FRDA diagnosis. Objectives: Primary: To assess the effect of oral administration of methylprednisolone on the functional performance scores of patients with FRDA using the Timed 25-Foot Walk (T25FW). Secondary: To assess the effect of methylprednisolone on neurological performance measures (Friedreich Ataxia Rating Scale, 9-Hole Peg Test, 1-minute walk, home-based measures of gait, hand function and speech assessed through smartphone application) and to assess the safety and tolerability of methylprednisolone in the FRDA population. Study Design: This study is an open-label clinical trial of methylprednisolone in patients with FRDA. Setting/Participants: This study will take place at the Children's Hospital of Philadelphia as an outpatient trial in 5 children who are at least 5 years and less than 10 years of age, and in 5 adults ages 45 years and older, with genetically confirmed FRDA. See below for description of study intervention and measures. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02424435
Study type	Interventional
Source	Children's Hospital of Philadelphia
Contact
Status	Completed
Phase	Early Phase 1
Start date	June 2015
Completion date	April 2018

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT05573698` - Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia	Phase 1
Completed	`NCT00229632` - Idebenone to Treat Friedreich's Ataxia	Phase 2
Completed	`NCT05579691` - A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia	Phase 2
Completed	`NCT04273165` - Safety and Efficacy of Etravirine in Friedreich Ataxia Patients	Phase 2
Not yet recruiting	`NCT05874388` - Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia	N/A
Completed	`NCT02594917` - Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension
Completed	`NCT01716221` - An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia	Phase 4
Active, not recruiting	`NCT05485987` - A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	Phase 2
Completed	`NCT03214588` - Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia	Phase 2
Completed	`NCT03418740` - Neurology Measures in FA Children
Not yet recruiting	`NCT06054893` - A Study of Omaveloxolone in Children With Friedreich's Ataxia	Phase 1
Recruiting	`NCT04921930` - Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)	Phase 1/Phase 2
Completed	`NCT03933163` - Micronised Resveratrol as a Treatment for Friedreich Ataxia	Phase 2
Completed	`NCT02705547` - Rosuvastatin (Crestor) in Friedreich Ataxia	Early Phase 1
Completed	`NCT02035020` - A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients	Phase 2
Completed	`NCT00015808` - Safety Study of Idebenone to Treat Friedreich's Ataxia	Phase 1
Completed	`NCT04817111` - NAD+ Precursor Supplementation in Friedreich's Ataxia	Phase 2
Active, not recruiting	`NCT05168774` - FRDA Investigator Initiated Study (IIS) With Elamipretide	Phase 1/Phase 2
Completed	`NCT03917225` - A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients	Phase 2
Completed	`NCT00224640` - Iron-Chelating Therapy and Friedreich Ataxia	Phase 1/Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.