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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06405633




Registration number
NCT06405633
Ethics application status
Date submitted
3/05/2024
Date registered
8/05/2024

Titles & IDs
Public title
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants with Pi*ZZ AATD
Scientific title
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants with AATD Pi*ZZ on WVE-006 (RestorAATion-2)
Secondary ID [1] 0 0
WVE-006-002
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
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 - WVE-006

Experimental: WVE-006 (Dose 1) -

Experimental: WVE-006 (Dose 2) -

Experimental: WVE-006 (Dose 3) -


Treatment: Drugs: WVE-006
RNA editing oligonucelotide
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The proportion of participants with adverse events
Timepoint [1] 0 0
Up to 36 Weeks
Secondary outcome [1] 0 0
Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.
Timepoint [1] 0 0
Up to 12 Weeks
Secondary outcome [2] 0 0
Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein
Timepoint [2] 0 0
Up to 24 Weeks
Secondary outcome [3] 0 0
Single Ascending Dose - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)
Timepoint [3] 0 0
Up to 12 Weeks
Secondary outcome [4] 0 0
Single Ascending Dose - Maximum concentration of WVE-006 in plasma
Timepoint [4] 0 0
Up to 12 Weeks
Secondary outcome [5] 0 0
Multiple Ascending Doses - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)
Timepoint [5] 0 0
Up to 24 Weeks
Secondary outcome [6] 0 0
Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)
Timepoint [6] 0 0
Up to 24 Weeks

Eligibility
Key inclusion criteria
* Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 =50%) and/or stable mild AATD-induced liver disease (=F2 (=10 kPa) on FibroScan.
* Genetic testing confirming Pi*ZZ.
* Participant has been a non-smoker for at least 1 year prior to screening.
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc).

* Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections.
* Any ongoing or recent infections.
* Any recent or planned vaccinations during the study.
* Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week.
* Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits.
* Any recent or planned major surgery during the study.
* Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study.
* Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit.
* Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study.
* Participant has received an investigational agent within 3 months of the Screening Visit.

Study design
Purpose of the study
Treatment
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
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/07/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2025
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
St. Vincent's Hospital, Melbourne - Fitzroy
Recruitment postcode(s) [1] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Nova Scotia
Country [2] 0 0
Canada
State/province [2] 0 0
Ontario
Country [3] 0 0
Finland
State/province [3] 0 0
Turku
Country [4] 0 0
New Zealand
State/province [4] 0 0
Hamilton
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Wave Life Sciences Ltd.
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Cynthia Caracta, MD
Address 0 0
Wave Life Sciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Operations
Address 0 0
Country 0 0
Phone 0 0
855-215-4687
Fax 0 0
Email 0 0
Clinicaltrials@wavelifesci.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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/NCT06405633