Ignite Creation Date:
2024-05-06 @ 3:49 PM
Last Modification Date:
2024-10-26 @ 1:57 PM
Study NCT ID:
NCT04764448
Status:
TERMINATED
Last Update Posted:
2024-07-12
First Post:
2021-01-27
Brief Title:
A Study of Belcesiran in Patients With AATLD
Sponsor:
Dicerna Pharmaceuticals Inc a Novo Nordisk company
Organization:
Novo Nordisk AS
Study Overview
Official Title:
A Phase 2 Randomized Double-blind Placebo-Controlled Study Investigating Safety Tolerability Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
Status:
TERMINATED
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Development project was discontinued
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ESTRELLA
Brief Summary:
This is a multiple dose randomized placebo-controlled double-blind study of belcesiran to evaluate the safety tolerability PK and PD in adult patients with PiZZ AATD-associated liver disease AATLD
The study will be conducted in 3 separate cohorts A total of up to 16 participants may be enrolled in Cohort 1 and 2 A total number of 30 subjects will be enrolled in cohort 3 The 3 cohorts are differentiated by the duration of the treatment period the number of doses administered and the timing of the second liver biopsy
The study will be conducted in 3 separate cohorts A total of up to 16 participants may be enrolled in Cohort 1 and 2 A total number of 30 subjects will be enrolled in cohort 3 The 3 cohorts are differentiated by the duration of the treatment period the number of doses administered and the timing of the second liver biopsy
Detailed Description:
AATD-associated liver disease is a progressive condition resulting in liver fibrosis cirrhosis and in some cases hepatocellular carcinoma The lack of functional alpha-1 antitrypsin AAT in individuals with the PiZZ genotype in conjunction with other precipitating factors can lead to unchecked activity of neutrophil elastases in the alveoli causing emphysema and chronic obstructive pulmonary disease COPD This loss-of-function mechanism may be addressed by use of intravenous augmentation therapy which aims to substitute the missing AAT by infusing alpha-1 proteinase inhibitor A1PI purified from pooled human plasma
While augmentation therapy can address the loss of AAT in the lung no treatment exists for the associated liver disease
Given the severity of the disease with approximately 10 of affected patients developing liver cirrhosis and a subgroup of those patients in need of liver transplantation and the lack of an effective treatment that addresses the toxic hepatic gain-of-function mechanism there is an urgent unmet medical need to develop a therapy that can help this particular patient population
While augmentation therapy can address the loss of AAT in the lung no treatment exists for the associated liver disease
Given the severity of the disease with approximately 10 of affected patients developing liver cirrhosis and a subgroup of those patients in need of liver transplantation and the lack of an effective treatment that addresses the toxic hepatic gain-of-function mechanism there is an urgent unmet medical need to develop a therapy that can help this particular patient population
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None