Clinical trials
catalyst calendar across sponsors →Studies where LRMR is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
5 interventional · 2 observational
Held by 15 tracked-famous managers (AQR CAPITAL MANAGEMENT LLC, MARSHALL WACE, LLP, Qube Research & Technologies Ltd, CITADEL ADVISORS LLC, MILLENNIUM MANAGEMENT LLC, +10 more) · FINRA short interest 9.4 days to cover (settled 2026-07-31) · government filings 180d: none disclosed
| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| An Open-Label Study of CTI-1601 in Subjects With Friedreich's AtaxiaNCT06447025Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum SeverityOpen-label · Treatment | Phase 2 | Recruiting | Drug: CTI-1601 | Friedreich Ataxia | 85 | Jan 2027 | ≤ 165 d | 2026-05-13 |
| A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's AtaxiaNCT06681766Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEsRandomized · Quadruple-masked · Treatment | Phase 1 | Terminatedsponsor's stated reason: Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study. | Drug: Nomlabofusp, Placebo | Friedreich Ataxia | 18 | 2025-04-27actual | — | 2026-07-10 |
| A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's AtaxiaNCT05579691Number of Participants with Treatment Emergent Adverse EventsRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Biological: CTI-1601 · Other: Placebo | Friedreich Ataxia | 28 | 2023-12-04actual | — | 2024-11-29 |
| Determine Range of Tissue Frataxin Concentrations and Other Potential BiomarkersNCT05028764observationalExamine tissue frataxin concentrations in buccal cells, blood and skin cells | — | Completed | Diagnostic test: Buccal Swabs, Blood Draws and Skin Punch Biopsy | Friedreich Ataxia, Healthy Volunteers | 60 | 2022-02-04actual | — | 2022-02-24 |
| Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaNCT04519567Number of Participants with Treatment Emergent Adverse EventsRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Biological: CTI-1601, Placebo | Friedreich Ataxia | 27 | 2021-03-16actual | — | 2021-06-30 |
| Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaNCT04176991Number of Participants with Treatment-Emergent Adverse EventsRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Biological: CTI-1601, Placebo | Friedreich Ataxia | 28 | 2020-10-31actual | — | 2020-11-12 |
| A Study to Assess Variation in Potential Biomarkers in Friedreich AtaxiaNCT04255680observationalDifference in frataxin levels, specific RNAs and proteins | — | Completed | Diagnostic test: Buccal Swabs and Blood Draws | Friedreich Ataxia | 20 | 2020-06-30actual | — | 2020-08-18 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19