Study on the Use of Cinacalcet in Phosphocalcic Context. (CALCI-CINA)

Multicentric Retrospective Study on the Use of Cinacalcet in an Off-label Phosphocalcic Context

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Hyperparathyroidism, Primary; Hyperparathyroidism, Secondary
• Intervention / Treatment: Other: Serum PTH concentrations results

• Study Type: Observational
• Enrollment (Anticipated): 25

Contacts and Locations

• Study Contact: Name: Justine BACCHETTA, MD; Phone Number: +33 4 27 85 61 30; Email: justine.bacchetta@chu-lyon.fr
• Study Contact Backup: Name: Sacha FLAMMIER, PHD; Phone Number: +33 4 27 85 66 69; Email: sacha.flammier@chu-lyon.fr

Study Locations

• France
  • Clermont-Ferrand, France
    • Endocrinologie Diabète et Maladies Métaboliques - Hôpital Gabriel Montpied
    • Contact: Igor TAUVERON, PR; Phone Number: +33 4 73 75 15 33; Email: itauveron@chu-clermontferrand.fr
  • Lille, France, 59037
    • Service de Néphrologie Pédiatrique -Hôpital Jeanne de Flandre
    • Contact: Robert NOVO, MD; Phone Number: +33 03.20.44.46.95; Email: robert.novo@chru-lille.fr
  • Limoges, France, 87042
    • Service d'Endocrinologie Pédiatrique - Hôpital de la mère et de l'Enfant
    • Contact: Anne LIENHARDT-ROUSSIE, MD; Phone Number: +33 5.55.05.63.58; Email: anne.lienhardt@chu-limoges.fr
  • Montpellier, France, 34090
    • Pole Femme Mère Enfant - Pédiatrie spécialisée - Centre Hospitalier Universitaire
    • Contact: Cyril AMOUROUX, MD; Phone Number: +33 4 67 33 81 12; Email: cyril-amouroux@chu-montpellier.fr
  • Nantes, France, 44093
    • Service de Néphrologie pédiatrique - Clinique Médicale Pédiatrique
    • Contact: Emma ALLAIN-LAUNAY, MD; Phone Number: +33 240083660; Email: emma.allainlaunay@chu-nantes.fr
  • Paris, France, 75019
    • Service d'endocrinologie et Diabétologie Pédiatrique-Hôpital Robert Debré
    • Contact: Laeticia MARTINERIE, MD; Phone Number: +33 1.40.03.53.03; Email: laetitia.martinerie@aphp.fr
  • Reims, France, 51100
    • Service de Diabétologie et endocrinologie pédiatriques - Centre Hospitalier Universitaire
    • Contact: Pierre-François SOUCHON, MD; Email: pfsouchon@chu-reims.fr
  • Strasbourg, France, 67091
    • Pôle Néphrologie-Urologie-Diabétologie-Endocrinologie
    • Contact: Nathalie JEANDIDIER, PR; Phone Number: +33 3 88 11 66 03; Email: nathalie.jeandidier@chru-strasbourg.fr
  • Toulouse, France, 31059
    • Service d'Endocrinologie, Maladies Osseuses, Gynécologie, Génétique
    • Contact: Jean Pierre SALLES, MD; Phone Number: +33 534558555; Email: salles.jp@chu-toulouse.fr
  • Tours, France, 37044
    • Unité Endocrinologie, Nutrition, Diabétologie -Hôpital Bretonneau
    • Contact: Lise CRINIERE, MD; Email: lise.criniere@univ-tours.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 99 years (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with phosphocalcic diseases suffering from hyperPTH without end-stage renal failure.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cinacalcet; Patients with primary or secondary hyperPTH resulting from phosphocalcic pathology treated by cinacalcet	Other: Serum PTH concentrations results; To compare serum PTH concentrations results in patients with primary or secondary hyperPTH resulting from phosphocalcic pathology before and three months after initiation of cinacalcet therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Serum PTH concentration	3 months after treatment initiation

Secondary Outcome Measures

Outcome Measure	Time Frame
Serum PTH concentration	1 month after treatment initiation
Serum PTH concentration	6 months after treatment initiation
Serum PTH concentration	12 months after treatment initiation
Serum PTH concentration	3 years after treatment initiation

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon
• Investigators: Principal Investigator: Justine BACCHETTA, MD, Service de Néphrologie, Rhumatologie et Dermatologie Pédiatriques

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): January 1, 2020
• Primary Completion (ANTICIPATED): January 1, 2021
• Study Completion (ANTICIPATED): December 1, 2023

Study Registration Dates

• First Submitted: October 11, 2019
• First Submitted That Met QC Criteria: October 11, 2019
• First Posted (ACTUAL): October 15, 2019

Study Record Updates

• Last Update Posted (ACTUAL): October 15, 2019
• Last Update Submitted That Met QC Criteria: October 11, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Keywords: Hyperparathyroidism; pathology phosphocalcic
• Additional Relevant MeSH Terms: Endocrine System Diseases; Parathyroid Diseases; Hyperparathyroidism; Hyperparathyroidism, Primary; Hyperparathyroidism, Secondary
• Other Study ID Numbers: 2019_CALCI-CINA

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No