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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05146882
Recruitment Status : Withdrawn (Update to Program Design - No Participants Enrolled)
First Posted : December 7, 2021
Last Update Posted : June 27, 2022
Sponsor:
Information provided by (Responsible Party):
Dicerna Pharmaceuticals, Inc.

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Alpha 1-Antitrypsin Deficiency Drug: Belcesiran Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
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
Estimated Study Start Date : December 2021
Actual Primary Completion Date : May 23, 2022
Actual Study Completion Date : May 23, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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)


Outcome Measures
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Primary Outcome Measures :
  1. The incidence of treatment-emergent adverse events [ Time Frame: up to 152 weeks ]
  2. The change from baseline in pulmonary function tests (PFTs) [ Time Frame: up to 152 weeks ]
    Forced expiratory volume in 1 second (FEV1)

  3. The change from baseline in PFTs [ Time Frame: up to 152 weeks ]
    Forced vital capacity (FVC)

  4. The change from baseline in PFTs [ Time Frame: up to 152 weeks ]
    FEV1/FVC

  5. The change from baseline in PFTs [ Time Frame: up to 152 weeks ]
    diffusing capacity for carbon monoxide (DLCO)

  6. The change from baseline in 12-lead electrocardiogram (ECG) [ Time Frame: up to 56 weeks ]
    heart rate

  7. The change from baseline in ECG [ Time Frame: up to 56 weeks ]
    ventricular rate

  8. The change from baseline in 12-lead ECG [ Time Frame: up to 56 weeks ]
    RR interval

  9. The change from baseline in 12-lead ECG [ Time Frame: up to 56 weeks ]
    PR interval

  10. The change from baseline in 12-lead ECG [ Time Frame: up to 56 weeks ]
    QRS duration

  11. The change from baseline in 12-lead ECG [ Time Frame: up to 56 weeks ]
    QT interval

  12. The change from baseline in 12-lead ECG [ Time Frame: up to 56 weeks ]
    corrected QT interval (QTcF, Fridericia correction)

  13. The change from baseline in physical examination (PE) findings [ Time Frame: up to 56 weeks ]
    body weight

  14. The change from baseline in PE findings [ Time Frame: up to 56 weeks ]
    body-mass index (BMI) (using height from DCR-A1AT-201 study)

  15. 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

  16. The change from baseline in vital sign measurements [ Time Frame: up to 56 weeks ]
    blood pressure

  17. The change from baseline in vital sign measurements [ Time Frame: up to 56 weeks ]
    pulse rate

  18. The change from baseline in vital sign measurements [ Time Frame: up to 56 weeks ]
    respiratory rate

  19. The change from baseline in vital sign measurements [ Time Frame: up to 56 weeks ]
    oral temperature

  20. 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

  21. 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

  22. 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

  23. 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


Secondary Outcome Measures :
  1. Changes in serum AAT protein concentrations over time [ Time Frame: up to 152 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05146882


Locations
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New Zealand
Auckland Clinical Studies
Grafton, Auckland, New Zealand, 1010
Sponsors and Collaborators
Dicerna Pharmaceuticals, Inc.
Investigators
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Study Director: Anne-Sophie Sejling, MD Dicerna Pharmaceuticals
More Information
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Responsible Party: Dicerna Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT05146882    
Other Study ID Numbers: DCR-A1AT-202
STARLIGHT ( Other Identifier: Dicerna Pharmaceuticals )
First Posted: December 7, 2021    Key Record Dates
Last Update Posted: June 27, 2022
Last Verified: June 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Liver Diseases
Alpha 1-Antitrypsin Deficiency
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes