Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05146882




Registration number
NCT05146882
Ethics application status
Date submitted
9/11/2021
Date registered
7/12/2021
Date last updated
11/07/2024

Titles & IDs
Public title
An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
Scientific 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
Secondary ID [1] 0 0
STARLIGHT
Secondary ID [2] 0 0
DCR-A1AT-202
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alpha 1-Antitrypsin Deficiency 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Cancer 0 0 0 0
Liver
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Belcesiran

Experimental: belcesiran - Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously

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)


Treatment: Drugs: Belcesiran
Belcesiran will be administered subcutaneously (SC) in the treatment arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The incidence of treatment-emergent adverse events
Timepoint [1] 0 0
up to 152 weeks
Primary outcome [2] 0 0
The change from baseline in pulmonary function tests (PFTs)
Timepoint [2] 0 0
up to 152 weeks
Primary outcome [3] 0 0
The change from baseline in PFTs
Timepoint [3] 0 0
up to 152 weeks
Primary outcome [4] 0 0
The change from baseline in PFTs
Timepoint [4] 0 0
up to 152 weeks
Primary outcome [5] 0 0
The change from baseline in PFTs
Timepoint [5] 0 0
up to 152 weeks
Primary outcome [6] 0 0
The change from baseline in 12-lead electrocardiogram (ECG)
Timepoint [6] 0 0
up to 56 weeks
Primary outcome [7] 0 0
The change from baseline in ECG
Timepoint [7] 0 0
up to 56 weeks
Primary outcome [8] 0 0
The change from baseline in 12-lead ECG
Timepoint [8] 0 0
up to 56 weeks
Primary outcome [9] 0 0
The change from baseline in 12-lead ECG
Timepoint [9] 0 0
up to 56 weeks
Primary outcome [10] 0 0
The change from baseline in 12-lead ECG
Timepoint [10] 0 0
up to 56 weeks
Primary outcome [11] 0 0
The change from baseline in 12-lead ECG
Timepoint [11] 0 0
up to 56 weeks
Primary outcome [12] 0 0
The change from baseline in 12-lead ECG
Timepoint [12] 0 0
up to 56 weeks
Primary outcome [13] 0 0
The change from baseline in physical examination (PE) findings
Timepoint [13] 0 0
up to 56 weeks
Primary outcome [14] 0 0
The change from baseline in PE findings
Timepoint [14] 0 0
up to 56 weeks
Primary outcome [15] 0 0
The change from baseline in PE findings
Timepoint [15] 0 0
up to 56 weeks
Primary outcome [16] 0 0
The change from baseline in vital sign measurements
Timepoint [16] 0 0
up to 56 weeks
Primary outcome [17] 0 0
The change from baseline in vital sign measurements
Timepoint [17] 0 0
up to 56 weeks
Primary outcome [18] 0 0
The change from baseline in vital sign measurements
Timepoint [18] 0 0
up to 56 weeks
Primary outcome [19] 0 0
The change from baseline in vital sign measurements
Timepoint [19] 0 0
up to 56 weeks
Primary outcome [20] 0 0
The change from baseline in clinical laboratory tests: Hematology
Timepoint [20] 0 0
up to 152 weeks
Primary outcome [21] 0 0
The change from baseline in clinical laboratory tests: Clinical Chemistry
Timepoint [21] 0 0
up to 152 weeks
Primary outcome [22] 0 0
The change from baseline in clinical laboratory tests: Coagulation
Timepoint [22] 0 0
up to 152 weeks
Primary outcome [23] 0 0
The change from baseline in clinical laboratory tests: Urinalysis
Timepoint [23] 0 0
up to 152 weeks
Secondary outcome [1] 0 0
Changes in serum AAT protein concentrations over time
Timepoint [1] 0 0
up to 152 weeks

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Other
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/12/2021
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/05/2022
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Anne-Sophie Sejling, MD
Address 0 0
Dicerna Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05146882