NTLA-2001 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
28全身性アミロイドーシス1

28. 全身性アミロイドーシス


臨床試験数 : 267 薬物数 : 241 - (DrugBank : 77) / 標的遺伝子数 : 68 - 標的パスウェイ数 : 180
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04601051
(ClinicalTrials.gov)
November 5, 202019/10/2020Study 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 AmyloidosisBiological: NTLA-2001Intellia TherapeuticsNULLRecruiting18 Years90 YearsAll72Phase 1France;New Zealand;Sweden;United Kingdom