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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02424435
Other study ID # 15-011801
Secondary ID
Status Completed
Phase Early Phase 1
First received
Last updated
Start date June 2015
Est. completion date April 2018

Study information

Verified date March 2021
Source Children's Hospital of Philadelphia
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

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.


Description:

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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Methylprednisolone
Oral tablets of methylprednisolone 8 mg. Subjects will receive a monthly prescription bottle of 25 tablets (standard quantity) and will self-administer 21 tablets over a 28-day dosing cycle.

Locations

Country Name City State
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania

Sponsors (2)

Lead Sponsor Collaborator
Children's Hospital of Philadelphia Friedreich's Ataxia Research Alliance

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in the Timed 25 Foot Walk (T25FW) Score The T25FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk. The subject is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible. The time is calculated from the initiation of the instruction to start and ends when the subject has reached the 25-foot mark. The task is immediately administered again by having the subject walk back the same distance. Subjects may use assistive devices when doing this task. This outcome will be measured at baseline and 26 weeks
Secondary Change in the Friedreich Ataxia Rating Scale (FARS) Score The Friedreich Ataxia Rating Scale (FARS) is neurological rating scale specifically developed and validated for FRDA. The FARS includes assessments of stance, gait, upper and lower limb coordination, speech, proprioception and strength. In addition to the standard neurological examination, the FARS contains three quantitative performance measures and a component that assesses activities of daily living (ADL). Quantitative performance measures include the nine-hole peg test, and a timed 25-foot walk. FARS scores correlate significantly with functional disability, activities of daily living scores and disease duration. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability. This outcome will be measured at baseline and 26 weeks
Secondary Change in The 1-Minute Walk Distance The 1-Minute Walk is a quantitative mobility and leg function performance test based on distance traveled in 1 minute. The subject is directed to one end of a clearly marked 25-foot course and instructed to walk back and forth as quickly as possible for 1 minute. The distance is calculated by measuring how far the subject travels along the 25-foot course. Subjects may use assistive devices during this task. This outcome will be measured at baseline and 26 weeks
Secondary Change in the Change in the 9-Hole Peg Test (9HPT) Time The 9HPT is a brief, standardized, quantitative test of upper extremity function. Both the dominant and non-dominant hands are tested twice. The subject is seated at a table with a small, shallow container holding nine pegs and a wood or plastic block containing nine empty holes. The total time to complete each of four trials is recorded. Two consecutive trials with the dominant hand are immediately followed by two consecutive trials with the non-dominant hand. This outcome will be measured at baseline and 26 weeks
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