Clinical trial NCT04126954: Study on the Use of Cinacalcet in Phosphocalcic Context.
Official title: Multicentric Retrospective Study on the Use of Cinacalcet in an Off-label Phosphocalcic Context
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Hyperparathyroidism, Secondary
Hyperparathyroidism, Primary
Keywords: Hyperparathyroidism
pathology phosphocalcic
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2019-10-15
Enrollment: 25
Sex: ALL
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with phosphocalcic diseases suffering from hyperPTH without end-stage renal failure.

Brief summary: Currently, the indications used for MA (Marketing Authorization) Cinacalcet in France are hyperparathyroidism (hyperPTH) in adults, whether primary (for patients in whom parathyroidectomy is theoretically indicated but in whom it is contraindicated or not is not clinically appropriate) or secondary to a chronic kidney disease, and parathyroid carcinomas.

In pediatric patients, data on its use are restricted due to its recent marketing authorization (2017) and limited to dialysis patients suffering from secondary hyperPTH.

Nevertheless, some patients with phosphocalcic pathologies without renal insufficiency must be treated off-label by cinacalcet in the presence of severe hyperPTH, without any other chronic treatment available to date.

The objective of this study is therefore to evaluate the use in France of cinacalcet in phosphocalcic pathologies without renal insufficiency, in order to obtain efficacy and safety data in order to improve our knowledge on the management of these orphan diseases.


Eligibility criteria: Inclusion Criteria:

* Patients with phosphocalcic pathology without end-stage renal failure
* Patients followed in one of the hospitals of the centers of reference and competence of the diseases of the metabolism of calcium and phosphate
* For children under 18: patient and parent (s) / parent having been informed of the study and having expressed their on-opposition
* For adults: patient / legal guardian of the patient under guardianship having been informed of the study and expressing his or her non opposition

Exclusion Criteria:

* Patient suffering from parathyroid cancer,
* Patient suffering from primary hyperPTH in whom parathyroidectomy would theoretically be indicated but contraindicated or clinically inappropriate,
* Patient suffering from hyperPTH secondary to end-stage renal failure
* No social security support

Primary outcomes:
measure: Serum PTH concentration
timeFrame: 3 months after treatment initiation
Secondary outcomes:
measure: Serum PTH concentration
timeFrame: 1 month after treatment initiation
measure: Serum PTH concentration
timeFrame: 6 months after treatment initiation
measure: Serum PTH concentration
timeFrame: 12 months after treatment initiation
measure: Serum PTH concentration
timeFrame: 3 years after treatment initiation
Other outcomes:
Locations:
city: Clermont-Ferrand
country: France
facility: Endocrinologie Diabète et Maladies Métaboliques - Hôpital Gabriel Montpied
city: Lille
country: France
facility: Service de Néphrologie Pédiatrique -Hôpital Jeanne de Flandre
city: Limoges
country: France
facility: Service d'Endocrinologie Pédiatrique - Hôpital de la mère et de l'Enfant
city: Montpellier
country: France
facility: Pole Femme Mère Enfant - Pédiatrie spécialisée - Centre Hospitalier Universitaire
city: Nantes
country: France
facility: Service de Néphrologie pédiatrique - Clinique Médicale Pédiatrique
city: Paris
country: France
facility: Service d'endocrinologie et Diabétologie Pédiatrique-Hôpital Robert Debré
city: Reims
country: France
facility: Service de Diabétologie et endocrinologie pédiatriques - Centre Hospitalier Universitaire
city: Strasbourg
country: France
facility: Pôle Néphrologie-Urologie-Diabétologie-Endocrinologie
city: Toulouse
country: France
facility: Service d'Endocrinologie, Maladies Osseuses, Gynécologie, Génétique
city: Tours
country: France
facility: Unité Endocrinologie, Nutrition, Diabétologie -Hôpital Bretonneau
Study officials:
affiliation: Service de Néphrologie, Rhumatologie et Dermatologie Pédiatriques
name: Justine BACCHETTA, MD
role: PRINCIPAL_INVESTIGATOR
References: