Register a trial

Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements. New passwords must include at least one lowercase letter, one uppercase letter, one number and one special character (e.g. !#$%&@).
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

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
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06393712




Registration number
NCT06393712
Ethics application status
Date submitted
19/04/2024
Date registered
1/05/2024

Titles & IDs
Public title
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
Scientific title
A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)
Secondary ID [1] 0 0
2023-510137-29-00
Secondary ID [2] 0 0
ALN-APP-002
Universal Trial Number (UTN)
Trial acronym
cAPPricorn-1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cerebral Amyloid Angiopathy 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - ALN-APP

Experimental: ALN-APP - Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period.

Placebo comparator: Placebo/ALN-APP - Participants will be administered multiple doses of placebo during the double-blind treatment period. Participants who continue into the optional open-label extension period will be administered multiple doses of ALN-APP.


Treatment: Drugs: Placebo
Placebo will be administered intrathecally

Treatment: Drugs: ALN-APP
ALN-APP will be administered intrathecally
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Double-blind Treatment Period: Annualized Rate of New Lobar Cerebral Microbleeds (CMBs) Assessed on Magnetic Resonance Imaging (MRI) of Brain in Patients with Sporadic Cerebral Amyloid Angiopathy (sCAA)
Timepoint [1] 0 0
Up to 24 months
Secondary outcome [1] 0 0
Double-blind Treatment Period: Global Rank Based on Severity, Count, Symptom Burden, and Timing of New Clinical Hemorrhagic Events and Hemorrhagic Lesions Assessed on MRI of Brain
Timepoint [1] 0 0
Up to 24 months
Secondary outcome [2] 0 0
Double-blind Treatment Period: Change from Baseline Over Time in Cerebrovascular Vasoreactivity Measured by Blood Oxygenation Level Dependent (BOLD) Parameters with Visual-evoked Functional MRI
Timepoint [2] 0 0
Up to 24 months
Secondary outcome [3] 0 0
Double-blind Treatment Period: Incidence of New Cerebral Hemorrhagic Lesions and White Matter Hyperintensities Assessed on MRI of Brain
Timepoint [3] 0 0
Up to 24 months
Secondary outcome [4] 0 0
Double-blind Treatment Period: Change from Baseline Over Time in the Total Cerebral Amyloid Angiopathy (CAA) Small Vessel Disease Score Assessed on MRI of Brain
Timepoint [4] 0 0
Up to 24 months
Secondary outcome [5] 0 0
Double-blind Treatment Period: Change from Baseline in Soluble Amyloid Precursor Protein Alpha (sAPPa) Concentration in Cerebrospinal Fluid (CSF)
Timepoint [5] 0 0
Up to 24 months
Secondary outcome [6] 0 0
Double-blind Treatment Period: Change from Baseline in Soluble Amyloid Precursor Protein Beta (sAPPß) Concentration in CSF
Timepoint [6] 0 0
Up to 24 months
Secondary outcome [7] 0 0
Double-blind Treatment Period and Open-label Extension (OLE) Period: Frequency of Adverse Events
Timepoint [7] 0 0
Up to 48 months

Eligibility
Key inclusion criteria
Inclusion Criteria (sporadic CAA patients):

* Is 50 years or older
* Has probable CAA per the Boston Criteria Version 2.0

Inclusion Criteria (Dutch-type CAA patients):

* Is 30 years or older
* Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA
Minimum age
30 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Moderate or severe stage Alzheimer's disease (AD) or significant cognitive impairment (CI)
* Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less than 90 days prior to anticipated randomization in the study
* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3×upper limit of normal (ULN) at Screening
* Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2 at Screening
* Has recently received an investigational agent
* Has had treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
17/05/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2029
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Heidelberg
Recruitment hospital [2] 0 0
Clinical Trial Site - Herston
Recruitment hospital [3] 0 0
Clinical Trial Site - Nedlands
Recruitment hospital [4] 0 0
Clinical Trial Site - Parkville
Recruitment postcode(s) [1] 0 0
- Heidelberg
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment postcode(s) [4] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Kentucky
Country [4] 0 0
United States of America
State/province [4] 0 0
Louisiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
Canada
State/province [12] 0 0
Alberta
Country [13] 0 0
Canada
State/province [13] 0 0
British Columbia
Country [14] 0 0
Canada
State/province [14] 0 0
New Brunswick
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
Canada
State/province [16] 0 0
Richmond
Country [17] 0 0
Switzerland
State/province [17] 0 0
Bern
Country [18] 0 0
Switzerland
State/province [18] 0 0
Geneve
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Denmark Hill
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Glasgow
Country [21] 0 0
United Kingdom
State/province [21] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alnylam Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Alnylam Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alnylam Clinical Trial Information Line
Address 0 0
Country 0 0
Phone 0 0
1-877-ALNYLAM
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


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/NCT06393712