New Biomarker-based Strategy to Screen and Monitor for Activated Phosphoinositide 3-kinase Δ Syndrome
NCT ID: NCT06694363
Last Updated: 2024-11-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
14 participants
OBSERVATIONAL
2024-12-31
2029-05-31
Brief Summary
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Collection of blood samples of APDS patients undergoing PI3K inhibitor treatment will be collected when feasible according to the standard of care planning (a blood test is supposed to be performed for these patients at M0-M3-M6-M12 then each 6 months for a total period of 2 years from the beginning of the PI3K inhibitor treatment).
The whole blood will be processed in order to isolate the peripheral blood mononuclear cells (PBMC) and the plasma. Serum, RNA and DNA extraction will be performed on a separate sample.
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Detailed Description
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In this context, it will be interesting to evaluate the clinical and biological profile of these patients before and after leniolisib treatment in order to identify useful biomarkers for the follow up of the disease. In addition, carful long-term monitoring of patients under PI3Kδ inhibitors is mandatory to detect adverse effects of iatrogenic overinhibition of the PI3K pathway.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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1 - initiating treatment
10 patients with a genetic diagnosis of APDS type 1 or type 2 who are to be treated with a selective PI3Kδ inhibitor, Lenolisib.
Blood samples
A maximum of 27 ml of blood collected at each visit for metabolic markers analysis
Urine samples
One urine sample collected at each visit for enteric virus infection research
Stool samples
One stool sample collected at each visit for enteric virus infection research
2 - already on treatment
4 patients with a genetic diagnosis of APDS type 1 or type 2 already treated with a selective PI3Kδ inhibitor, Lenolisib.
Blood samples
A maximum of 27 ml of blood collected at each visit for metabolic markers analysis
Urine samples
One urine sample collected at each visit for enteric virus infection research
Stool samples
One stool sample collected at each visit for enteric virus infection research
Interventions
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Blood samples
A maximum of 27 ml of blood collected at each visit for metabolic markers analysis
Urine samples
One urine sample collected at each visit for enteric virus infection research
Stool samples
One stool sample collected at each visit for enteric virus infection research
Eligibility Criteria
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Inclusion Criteria
* Patients with genetic diagnosis of APDS type 1 or type 2 and planned to be treated by PI3Kδ selective inhibitor leniolisib
* Primary immunodeficient patients with new disease-causing variants in the PIK3CD gene or PIK3R1 gene
* Minimum age 12 years old
* Patients or holders of parental authority do not oppose participation in this research.
* Patients affiliated to a Health Insurance scheme or beneficiaries
Group 2 :
* Patients with genetic diagnosis of APDS type 1 or type 2 already treated by PI3Kδ selective inhibitor leniolisib in the last 2 years
* Patients whose pre-treatment samples are available/analyzable
* Minimum age 12 years old
* Patients or holders of parental authority do not oppose participation in this research.
* patients affiliated to a Health Insurance scheme or beneficiaries
Exclusion Criteria
* Refusal to participate to the study.
12 Years
ALL
No
Sponsors
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Assistance Publique - Hôpitaux de Paris
OTHER
Responsible Party
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Principal Investigators
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Sven Kracker, PHD
Role: STUDY_DIRECTOR
Institut National de la Santé Et de la Recherche Médicale, France
Locations
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Hôpital Haut Levêque - BORDEAUX
Bordeaux, , France
Hôpital Pellerin Enfants - BORDEAUX
Bordeaux, , France
Hôpital Jeanne de Flandres - LILLE
Lille, , France
Hôpital La Timone adulte - MARSEILLE
Marseille, , France
Hôpital Necker Enfants Malades - PARIS
Paris, , France
CHU IUCT Oncopole - TOULOUSE
Toulouse, , France
Hôpital des enfants - TOULOUSE
Toulouse, , France
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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APHP240329
Identifier Type: -
Identifier Source: org_study_id
2024-A01816-41
Identifier Type: REGISTRY
Identifier Source: secondary_id
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