Methylprednisolone Treatment of Friedreich Ataxia

• Status: Completed
• Trial end date: 2018-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Philadelphia
• Collaborator: Friedreich's Ataxia Research Alliance
• Treatments: Methylprednisolone; Methylprednisolone Acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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