CTI-160l for Friedreich Ataxia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Friedreich AtaxiaCTI-1601 - Biological
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Eligible Conditions
  • Friedreich Ataxia

Treatment Effectiveness

Study Objectives

1 Primary · 6 Secondary · Reporting Duration: Through study completion, an average of 86 days

Day 29
Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point
Cells
Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses
Time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses
Plasma
Day 58
Changes from baseline in frataxin levels in buccal cells
Day 86
Therapeutic procedure

Trial Safety

Trial Design

2 Treatment Groups

CTI-160l
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

15 Total Participants · 2 Treatment Groups

Primary Treatment: CTI-160l · Has Placebo Group · Phase 2

CTI-160l
Biological
Experimental Group · 1 Intervention: CTI-1601 · Intervention Types: Biological
Placebo
Other
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Other
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CTI-1601
2020
Completed Phase 1
~60

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: through study completion, an average of 86 days

Who is running the clinical trial?

Larimar Therapeutics, Inc.Lead Sponsor
4 Previous Clinical Trials
135 Total Patients Enrolled
4 Trials studying Friedreich Ataxia
135 Patients Enrolled for Friedreich Ataxia
Magdy Shenouda, M.D.Principal InvestigatorClinilabs, Inc.
2 Previous Clinical Trials
87 Total Patients Enrolled
2 Trials studying Friedreich Ataxia
87 Patients Enrolled for Friedreich Ataxia

Eligibility Criteria

Age 18+ · All Participants · 4 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a genetically confirmed diagnosis of Friedreich's ataxia.
You are biologically male or female, 18 years of age or older.
References