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New treatment option for Friedreich Ataxia
Official title An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
ClinicalTrials.gov ID: NCT06447025
What this study is testing
What is CTI-1601?
CTI-1601 is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for friedreich ataxia.
Also referred to as Nomlabofusp.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 60
You may be able to join if
- people with FRDA who have or have not previously completed participation in a study of CTI-1601 are eligible to participate in this study unless the...
- Subject has a HbA1c less than or equal to 7.0%.
- Subject must demonstrate sufficient dexterity and visual acuity to prepare and self-administer SC injections of CTI-1601 QD or is able to identify a...
You likely can't join if
- people are excluded from the study if any of the following exclusion criteria are met:
- people 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 PI, could confound the results...
- Subject used any investigational drug (other than CTI-1601) or device within 90 days prior to Screening.
- Subject requires use of amiodarone.
- Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to Screening.
See the full eligibility criteria
- people with FRDA who have or have not previously completed participation in a study of CTI-1601 are eligible to participate in this study unless the subject experienced one or more of the following in a previous...
- Subject has a HbA1c less than or equal to 7.0%.
- Subject must demonstrate sufficient dexterity and visual acuity to prepare and self-administer SC injections of CTI-1601 QD or is able to identify a caregiver who will be trained and committed to prepare and administer...
- people are excluded from the study if any of the following exclusion criteria are met:
- people 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 PI, could confound the results of the study or put the subject at undue risk, making participation...
- Subject used any investigational drug (other than CTI-1601) or device within 90 days prior to Screening.
- Subject requires use of amiodarone.
- Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to Screening.
- Subject use of biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug. Biotin supplementation ≤30 mcg/day is...
- Subject uses more than 3 grams of acetaminophen daily.
- Subject receives medication that requires SC injection in the abdomen or thigh.
- Subject is unable to discontinue medications that have not been at a stable dose and frequency for at least 28 days prior to Screening.
- Subject has a Screening echocardiogram (ECHO) LVEF \< 45%.
- Male subject has a QTcF \> 450 milliseconds or female subject has a QTcF \> 470 milliseconds on an ECG.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Gainesville, Florida, United States
- Tampa, Florida, United States
- Iowa City, Iowa, United States
- Chevy Chase, Maryland, United States
- Eatontown, New Jersey, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Gainesville, Florida, United States; Tampa, Florida, United States; Iowa City, Iowa, United States; Chevy Chase, Maryland, United States; Eatontown, New Jersey, United States and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.