A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

Launched by BIOGEN · Sep 19, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a medication called omaveloxolone to see how it works and if it is safe for children with Friedreich's Ataxia, a genetic condition that affects movement and coordination. The trial is divided into two parts. In the first part, researchers will give a single dose of the medication to children in three different age groups (from 2 to 16 years old) to learn how the body processes the drug and to check for any side effects. The second part of the study will look at the long-term safety of omaveloxolone after participants have taken it for a while.

To be eligible for this trial, children must have a confirmed genetic diagnosis of Friedreich's Ataxia and meet specific health criteria, like having a certain heart function. They also need to agree to use birth control during the study if they are capable of becoming pregnant. Participants can expect to have regular check-ups and monitoring throughout the trial to ensure their safety and well-being. It's important to know that the trial is currently recruiting participants, and the researchers will carefully assess each child's health status to determine if they can join the study.

Gender

ALL

Eligibility criteria

• Part 1:
• Inclusion Criteria:
• • Have genetically confirmed FA.
• • Have a left ventricular ejection fraction ≥ 40% (based on ECHO performed at Screening Visit).
• • During screening, during the treatment period, and until 28 days following administration of the last dose of omaveloxolone, females of childbearing potential must practice at least 1 of the acceptable methods of birth control.
• • During screening, during the treatment period, and until 28 days after the last dose of omaveloxolone, fertile males who have female partners of childbearing potential must practice one of the acceptable methods of birth control.
• Exclusion Criteria:
• • Have uncontrolled diabetes (haemoglobin A1c \[HbA1c\] \>11.0%).
• • Have B-type natriuretic peptide (BNP) level \>200 picograms per milliliter (pg/mL) at screening.
• • Have a history of clinically significant (CS) left-sided heart disease and/or CS cardiac disease, with the exception of mild to moderate cardiomyopathy associated with FA.
• • Presence of outflow tract obstruction defined as a peak instantaneous gradient \>50 mmHg (based on ECHO performed at screening).
• • Have taken any moderate or strong inhibitors and/or inducers of cytochrome P450 3A4 within the 7 days prior to Day 1 or plan to take during study participation (eg, itraconazole, carbamazepine, phenytoin, ciprofloxacin, grapefruit juice, cannabidiol, fluconazole, fluvoxamine, verapamil, diltiazem).
• • Have a history of CS liver disease (eg, fibrosis, cirrhosis, hepatitis), or have clinically relevant deviations in laboratory tests at screening
• • Plan to or have participated in any other interventional clinical study within the 30 days prior to Day 1.
• • Have a cognitive impairment that may preclude ability to comply with study procedures, in the opinion of the investigator.
• • Be unable to comply with the requirements of the study protocol or be unsuitable for the study for any reason, in the opinion of the investigator.
• • Have previously documented mitochondrial respiratory chain disease.
• • Have a history of thromboembolic events within the past 5 years.
• • Plan to or have taken anticoagulant therapy within 30 days prior to Day 1 with the exception of a daily low dose aspirin (up to 81 mg).
• • Plan to or have scheduled surgical treatment for scoliosis or foot deformity during the study.
• • Have had significant suicidal ideation within 30 days prior to Screening Visit, as per investigator judgment, or any history of suicide attempt.
• • For females, be pregnant or breastfeeding.
• Part 2:
• • No discontinuation criteria have been met.
• • Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.
• • In the event of intercurrent illness or other change in health status of the participant, additional Part 1 Screening assessments may be repeated prior to initiation of Part 2, based on the judgement of the Investigator in consultation with the Medical Monitor.
• • NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.