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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04189601




Registration number
NCT04189601
Ethics application status
Date submitted
1/12/2019
Date registered
6/12/2019

Titles & IDs
Public title
Complement Activation in the Lysosomal Storage Disorders
Scientific title
Complement Activation in the Lysosomal Storage Disorders
Secondary ID [1] 0 0
Royal_Melbourne
Universal Trial Number (UTN)
Trial acronym
CATALYST
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Gaucher Disease 0 0
Niemann-Pick Disease, Type C 0 0
Lysosomal Storage Diseases 0 0
Condition category
Condition code
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
Neurological 0 0 0 0
Neurodegenerative diseases
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Diagnosis / Prognosis - Complement measurements

Study subjects - Patients with Fabry disease, Gaucher disease, or Niemann-Pick disease, type D

Controls - Age- and sex-matched to Study subjects


Diagnosis / Prognosis: Complement measurements
Blood and urine tests to assess the complement activation state
Intervention code [1] 0 0
Diagnosis / Prognosis
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in soluble C5b-9
Timepoint [1] 0 0
At baseline
Secondary outcome [1] 0 0
Other complement biomarkers
Timepoint [1] 0 0
Difference in C3a and C5a between Subjects and Controls at a single timepoint up to 8 months

Eligibility
Key inclusion criteria
* All consenting patients with a prior diagnosis of FD, GD or NPC will be included in the study. Control participants will be healthy volunteers.
Minimum age
17 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
* Patients who are unable to provide consent or to perform a blood or urine test will be excluded.

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
30/09/2020
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/04/2021
Actual
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3050 - Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
Melbourne Health
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Sanofi
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Thomas D Barbour, MBBS
Address 0 0
Melbourne Health
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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
No IPD sharing


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/NCT04189601