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| ID | Type | Description | Link |
|---|---|---|---|
| STARLIGHT | Other Identifier | Dicerna Pharmaceuticals |
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Update to Program Design - No Participants Enrolled
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| belcesiran | Experimental | Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously |
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| 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) |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Belcesiran | Drug | Belcesiran will be administered subcutaneously (SC) in the treatment arm. |
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| Measure | Description | Time Frame |
|---|---|---|
| The incidence of treatment-emergent adverse events | up to 152 weeks | |
| The change from baseline in pulmonary function tests (PFTs) | Forced expiratory volume in 1 second (FEV1) | up to 152 weeks |
| The change from baseline in PFTs | Forced vital capacity (FVC) | up to 152 weeks |
| The change from baseline in PFTs | FEV1/FVC | up to 152 weeks |
| The change from baseline in PFTs | diffusing capacity for carbon monoxide (DLCO) | up to 152 weeks |
| The change from baseline in 12-lead electrocardiogram (ECG) | heart rate | up to 56 weeks |
| The change from baseline in ECG | ventricular rate | up to 56 weeks |
| The change from baseline in 12-lead ECG | RR interval | up to 56 weeks |
| The change from baseline in 12-lead ECG | PR interval |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in serum AAT protein concentrations over time | up to 152 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Anne-Sophie Sejling, MD | Dicerna Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Auckland Clinical Studies | Grafton | Auckland | 1010 | New Zealand |
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| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| up to 56 weeks |
| The change from baseline in 12-lead ECG | QRS duration | up to 56 weeks |
| The change from baseline in 12-lead ECG | QT interval | up to 56 weeks |
| The change from baseline in 12-lead ECG | corrected QT interval (QTcF, Fridericia correction) | up to 56 weeks |
| The change from baseline in physical examination (PE) findings | body weight | up to 56 weeks |
| The change from baseline in PE findings | body-mass index (BMI) (using height from DCR-A1AT-201 study) | 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 | up to 56 weeks |
| The change from baseline in vital sign measurements | blood pressure | up to 56 weeks |
| The change from baseline in vital sign measurements | pulse rate | up to 56 weeks |
| The change from baseline in vital sign measurements | respiratory rate | up to 56 weeks |
| The change from baseline in vital sign measurements | oral temperature | up to 56 weeks |
| The change from baseline in clinical laboratory tests: Hematology | 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 | 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 | Coagulation is collected to evaluate the long-term safety of belcesiran | up to 152 weeks |
| The change from baseline in clinical laboratory tests: Urinalysis | Urinalysis is collected to evaluate the long-term safety of belcesiran | up to 152 weeks |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |