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Antisense Oligonucleotide

Belcesiran for Alpha-1 Antitrypsin Deficiency (ESTRELLA Trial)

Phase 2
Waitlist Available
Research Sponsored by Dicerna Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Documented diagnosis of PiZZ-type alpha-1 antitrypsin deficiency, confirmed by genotyping. Historical genotyping data may be used, if available
18 to 75 years, inclusive, at the time of consent
Timeline
Screening 3 weeks
Treatment Varies
Follow Up [cohort 1 + cohort 2: at predose, at 5 minutes postdose, at 15 minutes postdose, at 30 minutes postdose, at 60 minutes postdose, at 2 hours postdose, at 4 hours postdose, at 6 hours postdose, at 8 hours postdose, at 12 hours postdose, at 24 hours postdo
Awards & highlights

ESTRELLA Trial Summary

This trial will test the safety and effectiveness of a new drug, belcesiran, in adults with a specific liver disease. The trial will have two groups of patients, who will receive different doses of the drug or a placebo. The trial will last 24 or 48 weeks, depending on the group, and patients will have a liver biopsy at the end to check for improvement.

Who is the study for?
This trial is for adults aged 18-75 with Alpha-1 Antitrypsin Deficiency-associated liver disease, confirmed by genotyping and liver biopsy. Participants must have normal lung, kidney, and liver function tests and agree to undergo two liver biopsies. Excluded are those with severe lung disease exacerbations in the past year, prior RNAi drug use, significant respiratory infections within three months before screening, or advanced liver disease (Child-Pugh Score B or C).Check my eligibility
What is being tested?
The study tests Belcesiran's safety and effects on patients with AATLD compared to a placebo. It's randomized (participants are put into groups by chance), double-blind (neither participants nor researchers know who gets what treatment), involving multiple doses across three cohorts differing in treatment duration, dose count, and timing of a second biopsy.See study design
What are the potential side effects?
While specific side effects for Belcesiran aren't listed here, common ones for similar treatments include injection site reactions, fatigue, abdominal pain or discomfort; potential risks may also involve changes in liver enzymes indicating possible organ stress.

ESTRELLA Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My condition is confirmed PiZZ-type alpha-1 antitrypsin deficiency.
Select...
I am between 18 and 75 years old.
Select...
My liver disease linked to AATD was confirmed by a biopsy.

ESTRELLA Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~[cohort 1 + cohort 2: at predose, at 5 minutes postdose, at 15 minutes postdose, at 30 minutes postdose, at 60 minutes postdose, at 2 hours postdose, at 4 hours postdose, at 6 hours postdose, at 8 hours postdose, at 12 hours postdose, at 24 hours postdo
This trial's timeline: 3 weeks for screening, Varies for treatment, and [cohort 1 + cohort 2: at predose, at 5 minutes postdose, at 15 minutes postdose, at 30 minutes postdose, at 60 minutes postdose, at 2 hours postdose, at 4 hours postdose, at 6 hours postdose, at 8 hours postdose, at 12 hours postdose, at 24 hours postdo for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from Baseline in serum AAT concentration
Change from baseline in 12-lead ECGs: Heart Rate
Change from baseline in 12-lead ECGs: PR interval
+23 more
Secondary outcome measures
Disease
Plasma concentration of Belcesiran on Day 1
Plasma concentration of Belcesiran on Day 29
+1 more

ESTRELLA Trial Design

6Treatment groups
Experimental Treatment
Placebo Group
Group I: Belcesiran Cohort 3Experimental Treatment1 Intervention
Group II: Belcesiran Cohort 2Experimental Treatment1 Intervention
Group III: Belcesiran Cohort 1Experimental Treatment1 Intervention
Group IV: Placebo Cohort 1Placebo Group1 Intervention
Group V: Placebo Cohort 2Placebo Group1 Intervention
Group VI: Placebo Cohort 3Placebo Group1 Intervention

Find a Location

Who is running the clinical trial?

Dicerna Pharmaceuticals, Inc.Lead Sponsor
14 Previous Clinical Trials
471 Total Patients Enrolled
Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyLead Sponsor
17 Previous Clinical Trials
519 Total Patients Enrolled
Hardean Achneck, MDStudy DirectorDicerna Pharmaceuticals
2 Previous Clinical Trials
30 Total Patients Enrolled

Media Library

Belcesiran (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04764448 — Phase 2
Alpha-1 Antitrypsin Deficiency Research Study Groups: Belcesiran Cohort 1, Placebo Cohort 1, Belcesiran Cohort 2, Belcesiran Cohort 3, Placebo Cohort 2, Placebo Cohort 3
Alpha-1 Antitrypsin Deficiency Clinical Trial 2023: Belcesiran Highlights & Side Effects. Trial Name: NCT04764448 — Phase 2
Belcesiran (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04764448 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any participants needed for this research project?

"Yes, the data on clinicaltrials.gov suggests that this clinical trial is actively seeking patients. The trial was originally posted on 2/12/2021 and was most recently updated on 8/10/2022. The clinical trial is recruiting for 54 patients at 1 sites."

Answered by AI

If I wanted to be a subject in this experiment, what would I need to do?

"This trial is searching for 54 individuals that have alpha 1-antitrypsin deficiency and are between 18-70 years old."

Answered by AI

What is the status of belcesiran's drug approval process?

"Belcesiran's safety is yet to be confirmed by efficacy trials, but it has received a score of 2 due to the lack of evidence against its safety."

Answered by AI

Does this research involve any participants that are over the age of 35?

"The age range for patients that can enroll in this clinical trial is between 18-70 years old. If a patient does not meet this criteria, they may be able to participate in one of the 1 trials designed for minors or the 11 senior citizen friendly options."

Answered by AI

How many patients are participating in this clinical trial?

"That is correct. The clinical trial information available on clinicaltrials.gov reveals that the study is currently looking for 54 patients from 1 location. The study was originally posted on February 12th, 2021 and was last updated on August 10th, 2022."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
How old are they?
18 - 65
What site did they apply to?
Centre Hospitalier de l'Universite de Montreal (CHUM)
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Study of Belcesiran in Patients With AATLD
2021. "A Study of Belcesiran in Patients With AATLD". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04764448.
~11 spots leftby Apr 2025
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