Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04174118
Registration number
NCT04174118
Ethics application status
Date submitted
6/11/2019
Date registered
22/11/2019
Date last updated
11/07/2024
Titles & IDs
Public title
Study of DCR-A1AT in Healthy Adult Volunteers
Query!
Scientific title
A Phase 1 Single Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered Belcesiran in Healthy Adult Volunteers
Query!
Secondary ID [1]
0
0
DCR-A1AT-101
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Alpha 1-Antitrypsin Deficiency
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - belcesiran
Treatment: Drugs - Placebo
Experimental: belcesiran - Healthy volunteers will be administered a single dose of belcesiran.
Placebo comparator: Placebo - Healthy volunteers will be administered a single dose of matching placebo.
Treatment: Drugs: belcesiran
belcesiran will be administered subcutaneously (SC) at dose levels planned.
Treatment: Drugs: Placebo
Sterile normal saline (0.9% NaCL) matching volume of belcesiran doses will be administered subcutaneously (SC).
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Safety and tolerability
Query!
Assessment method [1]
0
0
The incidence of adverse events (AE), serious adverse events (SAE), DLT, and AE leading to study drug discontinuation
Query!
Timepoint [1]
0
0
approximately up to 2 months
Query!
Primary outcome [2]
0
0
Evaluating safety and tolerability through physical exams
Query!
Assessment method [2]
0
0
The incidence of clinically significant physical examination (PE) findings
Query!
Timepoint [2]
0
0
approximately up to 2 months
Query!
Primary outcome [3]
0
0
Changes in 12-lead electrocardiograms (ECG)
Query!
Assessment method [3]
0
0
Absolute QTc \> 500 msec and/or QTc change of \> 60 msec from baseline will be evaluated
Query!
Timepoint [3]
0
0
approximately up to 2 months
Query!
Secondary outcome [1]
0
0
Urine pharmacokinetics (PK) of belcesiran
Query!
Assessment method [1]
0
0
Maximum observed concentration (Cmax)
Query!
Timepoint [1]
0
0
up to Day 3
Query!
Secondary outcome [2]
0
0
Plasma pharmacokinetics (PK) of belcesiran
Query!
Assessment method [2]
0
0
Maximum observed concentration (Cmax)
Query!
Timepoint [2]
0
0
up to 57 days
Query!
Secondary outcome [3]
0
0
Plasma pharmacokinetics (PK) of belcesiran
Query!
Assessment method [3]
0
0
Area under the curve (AUC)
Query!
Timepoint [3]
0
0
up to 57 days
Query!
Secondary outcome [4]
0
0
Urine pharmacokinetics (PK) of belcesiran
Query!
Assessment method [4]
0
0
Area under the curve (AUC)
Query!
Timepoint [4]
0
0
up to Day 3
Query!
Secondary outcome [5]
0
0
Urine pharmacokinetics (PK) of belcesiran
Query!
Assessment method [5]
0
0
Minimum observed concentration (Cmin)
Query!
Timepoint [5]
0
0
up to Day 3
Query!
Secondary outcome [6]
0
0
Plasma pharmacokinetics (PK) of belcesiran
Query!
Assessment method [6]
0
0
Minimum observed concentration (Cmin)
Query!
Timepoint [6]
0
0
up to 57 days
Query!
Secondary outcome [7]
0
0
Plasma pharmacokinetics (PK) of belcesiran
Query!
Assessment method [7]
0
0
Time to maximum concentration (Tmax)
Query!
Timepoint [7]
0
0
up to 57 days
Query!
Secondary outcome [8]
0
0
Urine pharmacokinetics (PK) of belcesiran
Query!
Assessment method [8]
0
0
Time to maximum concentration (Tmax)
Query!
Timepoint [8]
0
0
up to Day 3
Query!
Secondary outcome [9]
0
0
Urine pharmacokinetics (PK) of belcesiran
Query!
Assessment method [9]
0
0
Terminal elimination half-life (t1/2)
Query!
Timepoint [9]
0
0
up to Day 3
Query!
Secondary outcome [10]
0
0
Plama pharmacokinetics (PK) of belcesiran
Query!
Assessment method [10]
0
0
Terminal elimination half-life (t1/2)
Query!
Timepoint [10]
0
0
up to 57 days
Query!
Secondary outcome [11]
0
0
Change in protein concentration
Query!
Assessment method [11]
0
0
Changes in A1AT protein concentrations
Query!
Timepoint [11]
0
0
up to day 57
Query!
Eligibility
Key inclusion criteria
* Male or Female aged 18 to 55 years, inclusive. Female participants must be either surgically sterile or postmenopausal. No women of childbearing potential are eligible for enrollment.
* Overtly Healthy, as determined by the investigator.
* Serum A1AT protein concentration >100 mg/dL
* Adequate forced expiratory volume in one second (FEV1) and adequate FEV1/forced vital capacity (FVC) ratio
* Non-smokers with a <2 pack-year history and smoking cessation for at least 6 months with a negative urinary cotinine test a screening
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
55
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
Yes
Query!
Key exclusion criteria
* Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially affect participant safety
* Clinically significant abnormal laboratory tests
* Received an experimental drug within past 4 months
* Prior to use of RNAi drug or oligonucleotide-based therapy
* Known human immunodeficiency virus (HIV), hepatitis C virus (HCV), or Hepatitis B (HBV)
* Serum creatinine or estimated glomerular filtration rate (eGFR) outside normal reference ranges.
Query!
Study design
Purpose of the study
Other
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 1
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
24/10/2019
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
6/03/2023
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
30
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment outside Australia
Country [1]
0
0
New Zealand
Query!
State/province [1]
0
0
Auckland
Query!
Country [2]
0
0
Sweden
Query!
State/province [2]
0
0
Uppsala
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This is a research study to test an experimental study drug (belcesiran, also known as DCR-A1AT). This drug is being tested to see if it helps people with a rare condition known as Alpha-1 Antitrypsin Deficiency, or A1ATD. Prior to initiation of this study belcesiran had not yet been tested in humans. All study participants will be randomly assigned to either receive the study drug or a placebo. This will allow for the sponsor to compare the effects of the study drug with that of the placebo. A placebo looks like the study drug but does not contain any of the study drug. The main purpose of the first part of the study is to evaluate the safety profile of the study drug in people who do not have A1ATD. This part of the study will also help find the dose of the study drug that has an acceptable safety profile for testing.
Query!
Trial website
https://clinicaltrials.gov/study/NCT04174118
Query!
Trial related presentations / publications
Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Thomas Bowman, MD
Query!
Address
0
0
Dicerna Pharmaceuticals
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
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/NCT04174118
Download to PDF