An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)

July 10, 2024 updated by: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease

This is a Phase 2, multicenter, open-label extension of Study DCR-A1AT-201, designed to evaluate the long-term safety and further characterize the pharmacodynamics (PD) of belcesiran in adult patients with PiZZ AATLD.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • New Zealand
    • Auckland
      • Grafton, Auckland, New Zealand, 1010
        • Auckland Clinical Studies

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Must be 18 to 75 years of age inclusive, at the time of signing the Informed Consent Form (ICF).
  2. Documented diagnosis of PiZZ-type Alpha-1 Antitrypsin deficiency (AATD), confirmed by genotyping.
  3. Lung, renal and liver function within acceptable limits.
  4. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.

Exclusion Criteria:

  1. Any condition that, in the opinion of the Investigator, would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study
  2. Routine use of acetaminophen/paracetamol
  3. Use of systemically acting steroids in the month prior to Screening and throughout the study period.
  4. Positive SARS-CoV-2 virus test at Screening
  5. Any other safety laboratory test result considered clinically significant and unacceptable by the Investigator
  6. Inability or unwillingness to comply with the specified study procedures, including lifestyle considerations

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: belcesiran
Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously
Drug: Belcesiran
Belcesiran will be administered subcutaneously (SC) in the treatment arm.
No Intervention: Observational
Participants who completed the DCR-A1AT-201 Conditional Follow-up period will enter DCR-A1AT-202 for continued follow-up (will not receive open-label belcesiran)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
The incidence of treatment-emergent adverse events
Time Frame: up to 152 weeks
up to 152 weeks
The change from baseline in pulmonary function tests (PFTs)
Time Frame: up to 152 weeks
Forced expiratory volume in 1 second (FEV1)
up to 152 weeks
The change from baseline in PFTs
Time Frame: up to 152 weeks
Forced vital capacity (FVC)
up to 152 weeks
The change from baseline in PFTs
Time Frame: up to 152 weeks
FEV1/FVC
up to 152 weeks
The change from baseline in PFTs
Time Frame: up to 152 weeks
diffusing capacity for carbon monoxide (DLCO)
up to 152 weeks
The change from baseline in 12-lead electrocardiogram (ECG)
Time Frame: up to 56 weeks
heart rate
up to 56 weeks
The change from baseline in ECG
Time Frame: up to 56 weeks
ventricular rate
up to 56 weeks
The change from baseline in 12-lead ECG
Time Frame: up to 56 weeks
RR interval
up to 56 weeks
The change from baseline in 12-lead ECG
Time Frame: up to 56 weeks
PR interval
up to 56 weeks
The change from baseline in 12-lead ECG
Time Frame: up to 56 weeks
QRS duration
up to 56 weeks
The change from baseline in 12-lead ECG
Time Frame: up to 56 weeks
QT interval
up to 56 weeks
The change from baseline in 12-lead ECG
Time Frame: up to 56 weeks
corrected QT interval (QTcF, Fridericia correction)
up to 56 weeks
The change from baseline in physical examination (PE) findings
Time Frame: up to 56 weeks
body weight
up to 56 weeks
The change from baseline in PE findings
Time Frame: up to 56 weeks
body-mass index (BMI) (using height from DCR-A1AT-201 study)
up to 56 weeks
The change from baseline in PE findings
Time Frame: up to 56 weeks
physical examination to assess skin, lungs, cardiovascular system, and abdomen (liver and spleen) based on the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 5.0) grading scale
up to 56 weeks
The change from baseline in vital sign measurements
Time Frame: up to 56 weeks
blood pressure
up to 56 weeks
The change from baseline in vital sign measurements
Time Frame: up to 56 weeks
pulse rate
up to 56 weeks
The change from baseline in vital sign measurements
Time Frame: up to 56 weeks
respiratory rate
up to 56 weeks
The change from baseline in vital sign measurements
Time Frame: up to 56 weeks
oral temperature
up to 56 weeks
The change from baseline in clinical laboratory tests: Hematology
Time Frame: up to 152 weeks
Hematology is collected to evaluate the long-term safety of belcesiran
up to 152 weeks
The change from baseline in clinical laboratory tests: Clinical Chemistry
Time Frame: up to 152 weeks
Clinical Chemistry is collected to evaluate the long-term safety of belcesiran
up to 152 weeks
The change from baseline in clinical laboratory tests: Coagulation
Time Frame: up to 152 weeks
Coagulation is collected to evaluate the long-term safety of belcesiran
up to 152 weeks
The change from baseline in clinical laboratory tests: Urinalysis
Time Frame: up to 152 weeks
Urinalysis is collected to evaluate the long-term safety of belcesiran
up to 152 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Changes in serum AAT protein concentrations over time
Time Frame: up to 152 weeks
up to 152 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Anne-Sophie Sejling, MD, Dicerna Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 23, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 23, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 9, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 23, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 7, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 11, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 10, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • DCR-A1AT-202
  • STARLIGHT (Other Identifier: Dicerna Pharmaceuticals)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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