Long-Term Follow-Up of Subjects Treated With NTLA-2001

Status

Recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-507385-11-00

Acronym

ITL-2001-CL-999

Enrollment

Registered

2024-04-16

Start date

2024-05-06

Completion date

Unknown

Last updated

2025-10-21

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) -Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Brief summary

For ATTR-CM and ATTRv-PN participants, incidence of: • Treatment-related SAEs • Protocol-specified AESIs

Detailed description

PD biomarkers for ATTR over time: • Serum TTR • Serum prealbumin

Interventions

DRUGNTLA-2001

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
For ATTR-CM and ATTRv-PN participants, incidence of: • Treatment-related SAEs • Protocol-specified AESIs	—

Secondary

Measure	Time frame
PD biomarkers for ATTR over time: • Serum TTR • Serum prealbumin	—

Countries

France, Sweden

Outcome results

None listed

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