Methylprednisolone Treatment of Friedreich Ataxia

Completed · Early Phase 1

Conditions studied: Friedreich Ataxia

In brief

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.

Key facts

Study ID

NCT02424435

Run by

Children's Hospital of Philadelphia

People needed

11

Starts

2015-06-01

Expected to finish

2018-04-01

Last updated by the study team

2021-03-25

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects with FRDA confirmed by genetic testing who are able to walk 25 feet (assistive devices allowed).
• Children between ages 5 and less than 10 years or adults ages 45 years and older at screening.
• Stable doses of all medications, vitamins and supplements for 30 days prior to study entry and for the duration of the study. Throughout the study, all possible efforts will be made to maintain stable doses of concomitant medications.
• Females who are not pregnant or breast feeding, and who do not intend to become pregnant. Females of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at screening.
• Informed consent for adult participants, parent/guardian permission (informed consent) and child assent for pediatric participants.

You may not qualify if…

• Patients unable to walk 25 feet.
• Treatment with methylprednisolone or cyclic methylprednisolone during the 3 previous months before inclusion.
• Treatment with gamma interferon, immunoglobulin G or other immunomodulating treatment the 3 previous month before inclusion
• Immunosuppressive treatment within 6 month of inclusion
• Prior history of a disease associated with immune dysfunction
• Poorly controlled Diabetes Mellitus (HbA1C > 9.0)
• History of untreated or uncontrolled hypertension
• Presence of infectious disease or other active infections which the treating physician finds relevant
• Active or previous history of liver or renal failure
• Known history of renal insufficiency or creatinine > 2 x upper limit of normal (ULN)
• Active infection at time of screening
• History of known osteoporosis

Where it is running

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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