This is an open-label study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of long-term daily subcutaneous (SC) administration of nomlabofusp, also known as CTI-1601, in subjects with Friedreich’s ataxia (FRDA).
This open label study is recruiting both individuals who have previously participated in a nomlabofusp study and individuals who have not previously participated in a nomlabofusp study. If you or your child have not previously participated in a nomlabofusp study and are interested in joining this open label study, register through this pre-screening questionnaire to determine if you may be eligible: https://www.larimarolestudy.com/OLE
Pre-screening registration will be limited to a set number of respondents so you are encouraged to complete the questionnaire as soon as possible.
The objectives of this open label study are:
- To evaluate the safety of long-term subcutaneous (SC) administration of nomlabofusp in subjects with FRDA
- To evaluate the PK of long-term subcutaneous (SC) administration of nomlabofusp in subjects with FRDA
- To evaluate the effect of long-term subcutaneous (SC) administration of nomlabofusp in subjects with FRDA on:
- Tissue FXN concentrations
- Clinical evaluations of FRDA
- Gene expression and select lipids
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
Key Inclusion Criteria:
- Subjects with FRDA over the age of 12 years who previously completed participation in a study of nomlabofusp who have not experienced a serious or significant adverse event during a previous nomlabofusp study or an adverse event considered clinically significant by the sponsor, or withdrawn from participation in a previous nomlabofusp for any reason
-OR- Subjects with FRDA between the ages of 2-60 years who have not previously participated in a nomlabofusp study. - Subject has an HbA1c less than or equal to 7.0%.
- Subject must demonstrate sufficient dexterity and visual acuity to prepare and self-administer daily subcutaneous injections of nomlabofusp or be able to identify a caregiver who will be trained and committed to prepare and administer the daily injections.
- Subject resides in the United States.
- For subjects taking or considering taking commercially available omaveloxolone (ages 16 and up):
- Subjects currently taking omaveloxolone must have been on a stable dose for 3 months.
- Subjects who intend to take omaveloxolone in the future must first be on a stable dose of nomlabofusp for 3 months before starting omaveloxolone.
Key Exclusion Criteria:
- Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
- Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the investigator, could confound the results of the study or put the subject at undue risk, making participation inadvisable.
- Subject used any investigational drug or device within 90 days prior to screening.
- Subject has a screen echocardiogram left ventricular ejection fraction (LVEF) less than 45%.
Additional inclusion and exclusion criteria apply and will be evaluated through the pre-screening questionnaire and by a study doctor.
To pre-screen for the study, interested individuals who have not previously participated in a nomlabofusp study should fill out this pre-screening questionnaire: https://www.larimarolestudy.com/OLE
Interested individuals who have previously participated in nomlabofusp study should contact their study coordinator.