Study type

Study topic

Human medicinal product
Disease /health condition

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness
Drug utilisation
Effectiveness study (incl. comparative)
Safety study (incl. comparative)

Data collection methods

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

Non-interventional study design

Other

Non-interventional study design, other

Multicentre, prospective, non-interventional post-authorisation safety study (PASS)
Study drug and medical condition

Name of medicine

RAXONE

Medical condition to be studied

Hereditary optic atrophy
Population studied

Short description of the study population

Patients with Leber’s hereditary optic neuropathy prescribed treatment with Raxone® under routine clinical practice.

Age groups

Children (2 to < 12 years)
Adolescents (12 to < 18 years)
Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Special population of interest

Renal impaired
Hepatic impaired
Pregnant women
Other

Special population of interest, other

Patients with Leber’s hereditary optic neuropathy

Estimated number of subjects

250
Study design details

Main study objective

To further evaluate the long-term safety profile of Raxone® in the treatment of patients with LHON when used under conditions of routine clinical care

Outcomes

• Frequency of adverse events of special interest (AESIs) • Frequency and nature of AEs and serious adverse events (SAEs), • Frequency and nature of adverse drug reactions (ADRs) and serious adverse drug reactions (SADRs), • Assessment of long term outcomes when Raxone® is used according to the SmPC

Data analysis plan

Collection of safety data, responder analysis