NCT04174118

Study of DCR-A1AT in Healthy Adult Volunteers

Completed Phase 1 Last updated 6 November 2024

What this trial tests

Phase 1 trial testing belcesiran in Alpha 1-Antitrypsin Deficiency in 30 participants. Completed in 6 March 2023.

Timeline

24 October 2019

Primary endpoint
6 July 2021

6 March 2023

Quick facts

Lead sponsor	Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	other
Enrollment	30
Start date	24 October 2019
Primary completion	6 July 2021
Estimated completion	6 March 2023
Sites	2 locations across New Zealand, Sweden

Drugs / interventions tested

• belcesiran — full drug profile →
• Placebo

Conditions studied

• Alpha 1-Antitrypsin Deficiency — all drugs for Alpha 1-Antitrypsin Deficiency →

Sponsor

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company — full company profile →

Who can join

Adults 18 to 55, any sex, with Alpha 1-Antitrypsin Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a research study to test an experimental study drug (belcesiran, also known as DCR-A1AT). This drug is being tested to see if it helps people with a rare condition known as Alpha-1 Antitrypsin Deficiency, or A1ATD. Prior to initiation of this study belcesiran had not yet been tested in humans. All study participants will be randomly assigned to either receive the study drug or a placebo. This will allow for the sponsor to compare the effects of the study drug with that of the placebo. A placebo looks like the study drug but does not contain any of the study drug.

The main purpose of the first part of the study is to evaluate the safety profile of the study drug in people who do not have A1ATD. This part of the study will also help find the dose of the study drug that has an acceptable safety profile for testing.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Paving the Road for RNA Therapeutics.
   Dammes N, Peer D. · · 2020 · cited 159× · PMID 32893005 · DOI 10.1016/j.tips.2020.08.004
2. Therapeutic RNA Delivery for COVID and Other Diseases.
   Dobrowolski C, Paunovska K, Hatit MZC, Lokugamage MP, et al · · 2021 · cited 46× · PMID 33661555 · DOI 10.1002/adhm.202002022
3. Hepatocyte targeting <i>via</i> the asialoglycoprotein receptor.
   Ramírez-Cortés F, Ménová P. · · 2025 · cited 22× · PMID 39628900 · DOI 10.1039/d4md00652f
4. Development, opportunities, and challenges of siRNA nucleic acid drugs.
   Xiao B, Wang S, Pan Y, Zhi W, et al · · 2025 · cited 19× · PMID 39897581 · DOI 10.1016/j.omtn.2024.102437
5. Targeting the Liver with Nucleic Acid Therapeutics for the Treatment of Systemic Diseases of Liver Origin.
   Gogate A, Belcourt J, Shah M, Wang AZ, et al · · 2023 · cited 17× · PMID 37696583 · DOI 10.1124/pharmrev.123.000815
6. Alpha-1 antitrypsin Pi∗Z allele is an independent risk factor for liver transplantation and death in patients with advanced chronic liver disease.
   Balcar L, Scheiner B, Urheu M, Weinberger P, et al · · 2022 · cited 14× · PMID 36176936 · DOI 10.1016/j.jhepr.2022.100562
7. Whole-Body Physiologically Based Pharmacokinetic-Pharmacodynamic Modeling for Interspecies Translation and Mechanistic Characterization of Plasma and Tissue Disposition of GalNAc-siRNAs.
   Salim EL, Kristensen K, Chopda G, Sjögren E. · · 2025 · cited 1× · PMID 41012491 · DOI 10.3390/pharmaceutics17091154