Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

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/NCT05211037




Registration number
NCT05211037
Ethics application status
Date submitted
6/01/2022
Date registered
27/01/2022
Date last updated
1/10/2024

Titles & IDs
Public title
Screening for Renal Complications in Children and Young Adults With Major Sickle Cell Disease
Scientific title
Screening for Renal Complications in Children and Young Adults With Major Sickle Cell Disease
Secondary ID [1] 0 0
21-AOI-09
Universal Trial Number (UTN)
Trial acronym
NEPHRO-DREPA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Anemia 0 0
Sickle Cell Disease 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Diagnosis / Prognosis - Kidney function assessment

Experimental: Kidney function assessment -


Diagnosis / Prognosis: Kidney function assessment
kidney clearance measured by scintigraphy
Intervention code [1] 0 0
Diagnosis / Prognosis
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
renal clearance
Timepoint [1] 0 0
at baseline

Eligibility
Key inclusion criteria
- Patient over 1 year old followed up in the Competence Centre at the Nice University Hospital, treated for a major sickle cell disease during the annual check-up
Minimum age
1 Year
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Pregnant patients (positive urine pregnancy test)
* Patients with other chronic conditions
* Progressive cancer or kidney disease
* Patients who are breastfeeding

Study design
Purpose of the study
Diagnosis
Allocation to intervention
Not applicable
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
Single group
Other design features
Phase
Not applicable
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
15/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2024
Actual
Sample size
Target
40
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Nice

Funding & Sponsors
Primary sponsor type
Other
Name
Centre Hospitalier Universitaire de Nice
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Sickle cell disease is the subject of targeted neonatal screening (carried out when one of the two parents is from an endemic country - sub-Saharan Africa, South-East Asia, Central America, the Caribbean) during the Guthrie test. Haemolysis, which results from the abnormality of the haemoglobin, and the vascular activation it causes, are responsible for multiple organ damage. Major sickle cell syndromes (MSC), by several mechanisms, are responsible for a wide range of renal damage, culminating in end-stage renal failure at an average age of 45 years and with an average survival of 3 years beyond ESRD.

The various renal disorders are : glomerular hyperfiltration and then glomerulosclerosis; tubular dysfunction, especially proximal and distal hyposthenuria (a factor in enuresis); papillary necrosis, renal infarction, episodes of acute renal failure during vaso-occlusive crises; dysregulation of the renin-angiotensin system with early arterial hypertension and, more rarely, extra-membranous glomerulonephritis. In the early stages of these conditions, simple paraclinical tests can identify them before the appearance of specific clinical signs.

In patients suffering from MDS, the HAS (High Authority of Health) recommends an annual check-up carried out in a Competence Centre. According to the HAS recommendations for annual surveillance, in addition to the search for other organic complications, for renal pathology, only microalbuminuria and renal ultrasound are recommended. However, as the literature shows, microalbuminuria and ultrasound only detect some of these renal disorders and at a very late stage. A large number of publications in adults and, to a lesser degree, in children, demonstrate the correlation between the frequency of acute complications of sickle cell disease (episodes of haemolysis, etc.) and the occurrence of kidney damage.
Trial website
https://clinicaltrials.gov/study/NCT05211037
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Camille FAUDEUX, Dr
Address 0 0
Centre Hospitalier Universitaire de Nice
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Camille FAUDEUX, Dr
Address 0 0
Country 0 0
Phone 0 0
4.92.03.63.65
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05211037