Alpha 1-Antitrypsin DeficiencyPhase 2NCT05146882
An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
A Phase 2 study testing Belcesiran in people with Alpha 1-Antitrypsin Deficiency, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 1 site in New Zealand.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- Belcesiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteNew Zealand
- Study dates
- Dec 2021to 23 May 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be 18 to 75 years of age inclusive, at the time of signing the Informed Consent Form (ICF).
- Documented diagnosis of PiZZ-type Alpha-1 Antitrypsin deficiency (AATD), confirmed by genotyping.
- Lung, renal and liver function within acceptable limits.
- Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Who cannot join
- 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
- Routine use of acetaminophen/paracetamol
- Use of systemically acting steroids in the month prior to Screening and throughout the study period.
- Positive SARS-CoV-2 virus test at Screening
- Any other safety laboratory test result considered clinically significant and unacceptable by the Investigator
- Inability or unwillingness to comply with the specified study procedures, including lifestyle considerations
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- Belcesiran
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BelcesiranDrug
Belcesiran will be administered subcutaneously (SC) in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| belcesiran | Experimental | Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously Receives: Drug: Belcesiran |
| Observational | No intervention | 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 being measured
The primary outcome is the main question the study is designed to answer.
The incidence of treatment-emergent adverse events
Measured: up to 152 weeks
The change from baseline in pulmonary function tests (PFTs)
Forced expiratory volume in 1 second (FEV1)
Measured: up to 152 weeks
The change from baseline in PFTs
Forced vital capacity (FVC)
Measured: up to 152 weeks
The change from baseline in PFTs
FEV1/FVC
Measured: up to 152 weeks
The change from baseline in PFTs
diffusing capacity for carbon monoxide (DLCO)
Measured: up to 152 weeks
The change from baseline in 12-lead electrocardiogram (ECG)
heart rate
Measured: up to 56 weeks
The change from baseline in ECG
ventricular rate
Measured: up to 56 weeks
The change from baseline in 12-lead ECG
RR interval
Measured: up to 56 weeks
The change from baseline in 12-lead ECG
PR interval
Measured: up to 56 weeks
The change from baseline in 12-lead ECG
QRS duration
Measured: up to 56 weeks
The change from baseline in 12-lead ECG
QT interval
Measured: up to 56 weeks
The change from baseline in 12-lead ECG
corrected QT interval (QTcF, Fridericia correction)
Measured: up to 56 weeks
The change from baseline in physical examination (PE) findings
body weight
Measured: up to 56 weeks
The change from baseline in PE findings
body-mass index (BMI) (using height from DCR-A1AT-201 study)
Measured: up to 56 weeks
The change from baseline in PE findings
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
Measured: up to 56 weeks
The change from baseline in vital sign measurements
blood pressure
Measured: up to 56 weeks
The change from baseline in vital sign measurements
pulse rate
Measured: up to 56 weeks
The change from baseline in vital sign measurements
respiratory rate
Measured: up to 56 weeks
The change from baseline in vital sign measurements
oral temperature
Measured: up to 56 weeks
The change from baseline in clinical laboratory tests: Hematology
Hematology is collected to evaluate the long-term safety of belcesiran
Measured: up to 152 weeks
The change from baseline in clinical laboratory tests: Clinical Chemistry
Clinical Chemistry is collected to evaluate the long-term safety of belcesiran
Measured: up to 152 weeks
The change from baseline in clinical laboratory tests: Coagulation
Coagulation is collected to evaluate the long-term safety of belcesiran
Measured: up to 152 weeks
The change from baseline in clinical laboratory tests: Urinalysis
Urinalysis is collected to evaluate the long-term safety of belcesiran
Measured: up to 152 weeks
Secondary outcomes (1)
Changes in serum AAT protein concentrations over time
Measured: up to 152 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2021
- Study startDec 2021 (estimated)
- Primary completion23 May 2022
- Study completion23 May 2022
Why it stopped: Update to Program Design - No Participants Enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anne-Sophie Sejling, MD, Study director, Dicerna Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05146882
- Sponsor's protocol ID
- DCR-A1AT-202
- Other IDs
- STARLIGHT
- Status last verified
- Sep 2024
- First submitted
- 9 Nov 2021
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05146882
- Acronym / sponsor ID
- DCR-A1AT-202
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Update to Program Design - No Participants Enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyIndustry (pharma, biotech, devices)
- Study start
- Dec 2021 (estimated)
- Primary completion
- 23 May 2022 (actual)
- Study completion
- 23 May 2022 (actual)
- First posted
- 7 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| belcesiran | Experimental | Drug: BelcesiranParticipants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously |
| Observational | No intervention | –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) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Must be 18 to 75 years of age inclusive, at the time of signing the Informed Consent Form (ICF).
- Documented diagnosis of PiZZ-type Alpha-1 Antitrypsin deficiency (AATD), confirmed by genotyping.
- Lung, renal and liver function within acceptable limits.
- Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Key exclusion criteria (6 listed)
- 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
- Routine use of acetaminophen/paracetamol
- Use of systemically acting steroids in the month prior to Screening and throughout the study period.
- Positive SARS-CoV-2 virus test at Screening
- Any other safety laboratory test result considered clinically significant and unacceptable by the Investigator
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BelcesiranBelcesiran will be administered subcutaneously (SC) in the treatment arm. | Drug | belcesiran |
Primary outcome
The incidence of treatment-emergent adverse events
Time frame: 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)
The change from baseline in PFTs
Time frame: up to 152 weeks
Forced vital capacity (FVC)
The change from baseline in PFTs
Time frame: up to 152 weeks
FEV1/FVC
The change from baseline in PFTs
Time frame: up to 152 weeks
diffusing capacity for carbon monoxide (DLCO)
The change from baseline in 12-lead electrocardiogram (ECG)
Time frame: up to 56 weeks
heart rate
The change from baseline in ECG
Time frame: up to 56 weeks
ventricular rate
The change from baseline in 12-lead ECG
Time frame: up to 56 weeks
RR interval
The change from baseline in 12-lead ECG
Time frame: up to 56 weeks
PR interval
The change from baseline in 12-lead ECG
Time frame: up to 56 weeks
QRS duration
The change from baseline in 12-lead ECG
Time frame: up to 56 weeks
QT interval
The change from baseline in 12-lead ECG
Time frame: up to 56 weeks
corrected QT interval (QTcF, Fridericia correction)
The change from baseline in physical examination (PE) findings
Time frame: up to 56 weeks
body weight
The change from baseline in PE findings
Time frame: up to 56 weeks
body-mass index (BMI) (using height from DCR-A1AT-201 study)
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
The change from baseline in vital sign measurements
Time frame: up to 56 weeks
blood pressure
The change from baseline in vital sign measurements
Time frame: up to 56 weeks
pulse rate
The change from baseline in vital sign measurements
Time frame: up to 56 weeks
respiratory rate
The change from baseline in vital sign measurements
Time frame: up to 56 weeks
oral temperature
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
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
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
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
1 secondary outcome
Changes in serum AAT protein concentrations over time
Time frame: up to 152 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anne-Sophie Sejling, MD study director
Dicerna Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Belcesiran in people with Alpha 1-Antitrypsin Deficiency, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 1 site in New Zealand.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of treatment-emergent adverse events (time frame: up to 152 weeks). The study lists 23 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.