New Biomarker-based Strategy to Screen and Monitor for Activated Phosphoinositide 3-kinase δ Syndrome
Recruiting
Observational Study
Activated PI3K Delta Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 12 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 14 (estimated)
- Sponsor
- Assistance Publique - Hôpitaux de Paris · Other
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About This Trial
The study would like to compare patient samples at different time points using state-of the art-phenotyping tools.
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 fr…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
Group 1:
* 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 …
Contacts