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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT05146882
Other study ID # DCR-A1AT-202
Secondary ID STARLIGHT
Status Withdrawn
Phase Phase 2
First received
Last updated
Start date December 2021
Est. completion date May 23, 2022

Study information

Verified date June 2022
Source Dicerna Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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.


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date May 23, 2022
Est. primary completion date May 23, 2022
Accepts healthy volunteers No
Gender All
Age group 18 Years to 75 Years
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

Drug:
Belcesiran
Belcesiran will be administered subcutaneously (SC) in the treatment arm.

Locations

Country Name City State
New Zealand Auckland Clinical Studies Grafton Auckland

Sponsors (1)

Lead Sponsor Collaborator
Dicerna Pharmaceuticals, Inc.

Country where clinical trial is conducted

New Zealand, 

Outcome

Type Measure Description Time frame Safety issue
Primary The incidence of treatment-emergent adverse events up to 152 weeks
Primary The change from baseline in pulmonary function tests (PFTs) Forced expiratory volume in 1 second (FEV1) up to 152 weeks
Primary The change from baseline in PFTs Forced vital capacity (FVC) up to 152 weeks
Primary The change from baseline in PFTs FEV1/FVC up to 152 weeks
Primary The change from baseline in PFTs diffusing capacity for carbon monoxide (DLCO) up to 152 weeks
Primary The change from baseline in 12-lead electrocardiogram (ECG) heart rate up to 56 weeks
Primary The change from baseline in ECG ventricular rate up to 56 weeks
Primary The change from baseline in 12-lead ECG RR interval up to 56 weeks
Primary The change from baseline in 12-lead ECG PR interval up to 56 weeks
Primary The change from baseline in 12-lead ECG QRS duration up to 56 weeks
Primary The change from baseline in 12-lead ECG QT interval up to 56 weeks
Primary The change from baseline in 12-lead ECG corrected QT interval (QTcF, Fridericia correction) up to 56 weeks
Primary The change from baseline in physical examination (PE) findings body weight up to 56 weeks
Primary The change from baseline in PE findings body-mass index (BMI) (using height from DCR-A1AT-201 study) up to 56 weeks
Primary 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 up to 56 weeks
Primary The change from baseline in vital sign measurements blood pressure up to 56 weeks
Primary The change from baseline in vital sign measurements pulse rate up to 56 weeks
Primary The change from baseline in vital sign measurements respiratory rate up to 56 weeks
Primary The change from baseline in vital sign measurements oral temperature up to 56 weeks
Primary The change from baseline in clinical laboratory tests: Hematology Hematology is collected to evaluate the long-term safety of belcesiran up to 152 weeks
Primary The change from baseline in clinical laboratory tests: Clinical Chemistry Clinical Chemistry is collected to evaluate the long-term safety of belcesiran up to 152 weeks
Primary The change from baseline in clinical laboratory tests: Coagulation Coagulation is collected to evaluate the long-term safety of belcesiran up to 152 weeks
Primary The change from baseline in clinical laboratory tests: Urinalysis Urinalysis is collected to evaluate the long-term safety of belcesiran up to 152 weeks
Secondary Changes in serum AAT protein concentrations over time up to 152 weeks
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