An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
ConditionFriedreich Ataxia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age2-60
SponsorLarimar Therapeutics, Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Subjects 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 CTI-1601 study: a) serious adverse event (SAE) related to study drug; b) significant AE, defined as Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), related to study drug; c) some other event, related to participation in a previous study with CTI-1601, that supports the exclusion of the subject from participating in this study as determined by the Sponsor (i.e., an AE considered clinically significant by the Sponsor regardless of whether it met SAE criteria and regardless of CTCAE grade); d) Withdraw from participation in a previous study of CTI-1601 for any reason.
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 the daily injections.
Disqualifiers
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 PI, could confound the results of the study or put the subject at undue risk, making participation inadvisable.
Subject used any investigational drug (other than CTI-1601) or device within 90 days prior to Screening.
Subject requires use of amiodarone.
Trial design
Design model
Single group
Treatments tested in this trial
CTI-1601
DrugCTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia
Treatment groups
85 Participants
are divided into 1 treatment groupGroup A: CTI-1601Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity
Number of subjects
Time frame
Up to 24 months2
Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval
Number change in ECG parameters
Time frame
Up to 24 months3
Change from baseline in left ventricular ejection fraction (LVEF)
LVEF indicates the percentage of change in LV volume from diastole to systole that measures how well the left ventricle of the heart pumps blood.
Time frame
Up to 24 months4
Change from baseline in left ventricular end-diastolic volume (LVEDV)
LVEDV is the amount of blood, measured in milliliters (mL), in the heart's left ventricle just before the heart contracts.
Time frame
Up to 24 monthsSponsors and contacts
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Source ClinicalTrials.gov