Study type

Study topic

Disease /health condition
Human medicinal product

Study type

Non-interventional study

Scope of the study

Safety study (incl. comparative)

Data collection methods

Combined primary data collection and secondary use of data
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

Prospective and retrospective observational study
Study drug and medical condition

Name of medicine

SKYCLARYS

Name of medicine, other

Omaveloxolone (SKYCLARYS)

Study drug International non-proprietary name (INN) or common name

OMAVELOXOLONE

Anatomical Therapeutic Chemical (ATC) code

(N07XX25) omaveloxolone
omaveloxolone

Medical condition to be studied

Friedreich's ataxia
Population studied

Short description of the study population

All participants for this study will be identified and enrolled via the FA-Global Clinical Consortium (FA-GCC) UNIFIED Natural History Study (UNIFAI study).

Age groups

Adult and elderly population (≥18 years)
Adults (18 to < 65 years)
Adults (18 to < 46 years)
Adults (46 to < 65 years)
Elderly (≥ 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Special population of interest

Other

Special population of interest, other

Friedreich’s ataxia patients

Estimated number of subjects

300
Study design details

Study design

Patient registry study

Main study objective

The primary objective of this study is to assess the long-term safety of omaveloxolone as prescribed to participants with FA in the real-world setting, including characterization of all drug-induced liver injury (DILI) and congestive heart failure (CHF) AEs.
The secondary objective of this study is to capture the reasons and timing of omaveloxolone treatment interruptions, discontinuations, and drug overdose.

Outcomes

Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs), Number of Participants With DILI and CHF AEs, Time to Omaveloxolone Treatment Interruption, Time to Omaveloxolone Treatment Discontinuation, Time to Omaveloxolone Drug Overdose, Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose

Data analysis plan

Data analyses will be presented separately for participants in the omaveloxolone-naive cohort and the non-naive cohort. An overall summary of TESAEs will be presented, consisting of the number and percentage of participants experiencing the total number of TESAEs. TESAEs will also be summarized by system organ class (SOC) and Preferred Term (PT).
Incidence rates of all DILI and CHF AEs will be estimated, with two-sided 95% CIs. Time to treatment interruption, time to discontinuation and time to omaveloxolone drug overdose will be analyzed using the Kaplan-Meier method and competing risk-based survival analysis if needed.
Reasons for treatment interruption, treatment discontinuation and drug overdose will be summarized descriptively.