Methylprednisolone Treatment of Friedreich Ataxia

NCT02424435 | Completed Early Phase 1 DMC

• 1 other identifier: 15-011801
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Friedreich Ataxia

Keywords

Friedreich Ataxia; FRDA

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change in the Friedreich Ataxia Rating Scale (FARS) Score

  This outcome will be measured at baseline and 26 weeks

• Change in The 1-Minute Walk Distance

  This outcome will be measured at baseline and 26 weeks

• Change in the Change in the 9-Hole Peg Test (9HPT) Time

  This outcome will be measured at baseline and 26 weeks

Study Arms (1)

Methylprednisolone

EXPERIMENTAL

This is an open-label study of methylprednisolone in patients with FRDA. Subjects will begin oral administration of 48 mg methylprednisolone at day 1 and will decrease their administered dose by 8 mg per day. After 6 days, subjects will spend 22 days off medication before repeating the same treatment cycle. Last dosing cycle of methylprednisolone will be administered at 24 weeks after baseline. Visits will occur at weeks 2, 6, 14, 26, and 30 following baseline.

Drug: Methylprednisolone

Interventions

Methylprednisolone DRUG

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.

Methylprednisolone

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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.
• 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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Friedreich's Ataxia Research Alliance: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Methylprednisolone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• David R Lynch, MD PhD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2015
• First Posted: April 23, 2015
• Study Start: June 1, 2015
• Primary Completion: December 1, 2017
• Study Completion: April 1, 2018
• Last Updated: March 25, 2021

Record last verified: 2021-03

Locations

US (1)