Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN).

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-511170-69-00

Acronym

ITL-2001-CL-001

Enrollment

Registered

2024-08-14

Start date

2021-08-17

Completion date

2025-04-09

Last updated

2025-07-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)

Brief summary

Safety assessments, immunogenicity assessments, pharmacokinetic assessments, pharmacodynamic assessments

Detailed description

Polyneuropathy assessments

Interventions

DRUGNTLA-2001

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Safety assessments, immunogenicity assessments, pharmacokinetic assessments, pharmacodynamic assessments	—

Secondary

Measure	Time frame
Polyneuropathy assessments	—

Countries

France, Sweden

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026