NTLA-2001 ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 28 | Systemic amyloidosis | 1 |
28. Systemic amyloidosis
Clinical trials : 267 / Drugs : 241 - (DrugBank : 77) / Drug target genes : 68 - Drug target pathways : 180
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04601051 (ClinicalTrials.gov) | November 5, 2020 | 19/10/2020 | Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) | 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) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy;Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy;Wild-Type Transthyretin Cardiac Amyloidosis | Biological: NTLA-2001 | Intellia Therapeutics | NULL | Recruiting | 18 Years | 90 Years | All | 72 | Phase 1 | France;New Zealand;Sweden;United Kingdom |