Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Amgen
With
Friedreich's Ataxia Research Alliance
Study with completed results acquired from Horizon in 2024.
Age
10–25
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Written informed consent and child assent, if applicable.
FA confirmed by genetic testing with two expanded guanine-adenine-adenine (GAA) repeats.
FA functional stage of \>1 to \<5 and ability to walk 25 feet with or without an assistive device.
Male or female subject between the ages of 10 and 25 years, inclusive.
If female, the subject is not pregnant or lactating or intending to become pregnant during the study, or within 30 days after the last dose of study drug. Female subjects of child-bearing potential must have a negative serum pregnancy test result at Screening, a negative urine pregnancy test result at Baseline, and agree to use a reliable method of contraception throughout the study and for 30 days after the last dose of study drug.
History of hypersensitivity to interferon (IFN)-ɣ or E. coli-derived products.
Presence of moderate or severe renal disease (estimated creatinine clearance \<50 mL/min) or hepatic disease (aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] \>2x the upper limit of normal) as evidenced by laboratory results at Screening.
Clinically significant abnormal white blood cell count, hemoglobin, or platelet count as evidenced by laboratory test results at Screening.
You may not be if
Any unstable illness that in the investigator's opinion precludes participation in the study.
Use of any investigational product within 30 days prior to randomization.
A history of substance abuse.
Clareo Health | Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
