NCT04176991

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

A Phase 1 Single Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Larimar Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia

Detailed description

Single Ascending Dose (SAD), Double-Blind, Placebo Controlled Study. To evaluate the safety and tolerability of single ascending doses of CTI-1601 in subjects with Friedreich's ataxia. Secondary Objectives: 1. To evaluate the pharmacokinetics (PK) of CTI-1601 following increasing single doses of subcutaneously (SC) administered CTI-1601. 2. To evaluate the pharmacodynamics (PD) of CTI-1601 following increasing single doses of SC administered CTI-1601. CTI-1601 or Placebo - Dose/Mode of Administration: Single Dose/Subcutaneous

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
BIOLOGICAL	CTI-1601	CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
BIOLOGICAL	Placebo	Placebo Comparator

Timeline

• Start date: 2019-12-11
• Primary completion: 2020-10-31
• Completion: 2020-10-31
• First posted: 2019-11-26
• Last updated: 2020-11-12

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study