NCT04126954

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
25

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2020

Longer than P75 for all trials

Geographic Reach
1 country

10 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 11, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 15, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

January 1, 2020

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2021

Completed
2.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2023

Completed
Last Updated

October 15, 2019

Status Verified

October 1, 2019

Enrollment Period

1 year

First QC Date

October 11, 2019

Last Update Submit

October 11, 2019

Conditions

Keywords

Hyperparathyroidismpathology phosphocalcic

Outcome Measures

Primary Outcomes (1)

  • Serum PTH concentration

    3 months after treatment initiation

Secondary Outcomes (4)

  • 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

Study Arms (1)

Cinacalcet

Patients with primary or secondary hyperPTH resulting from phosphocalcic pathology treated by cinacalcet

Other: Serum PTH concentrations results

Interventions

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.

Cinacalcet

Eligibility Criteria

AgeUp to 99 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Endocrinologie Diabète et Maladies Métaboliques - Hôpital Gabriel Montpied

Clermont-Ferrand, France

Location

Service de Néphrologie Pédiatrique -Hôpital Jeanne de Flandre

Lille, 59037, France

Location

Service d'Endocrinologie Pédiatrique - Hôpital de la mère et de l'Enfant

Limoges, 87042, France

Location

Pole Femme Mère Enfant - Pédiatrie spécialisée - Centre Hospitalier Universitaire

Montpellier, 34090, France

Location

Service de Néphrologie pédiatrique - Clinique Médicale Pédiatrique

Nantes, 44093, France

Location

Service d'endocrinologie et Diabétologie Pédiatrique-Hôpital Robert Debré

Paris, 75019, France

Location

Service de Diabétologie et endocrinologie pédiatriques - Centre Hospitalier Universitaire

Reims, 51100, France

Location

Pôle Néphrologie-Urologie-Diabétologie-Endocrinologie

Strasbourg, 67091, France

Location

Service d'Endocrinologie, Maladies Osseuses, Gynécologie, Génétique

Toulouse, 31059, France

Location

Unité Endocrinologie, Nutrition, Diabétologie -Hôpital Bretonneau

Tours, 37044, France

Location

MeSH Terms

Conditions

Hyperparathyroidism, SecondaryHyperparathyroidism, PrimaryHyperparathyroidism

Condition Hierarchy (Ancestors)

Parathyroid DiseasesEndocrine System Diseases

Study Officials

  • Justine BACCHETTA, MD

    Service de Néphrologie, Rhumatologie et Dermatologie Pédiatriques

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Justine BACCHETTA, MD

CONTACT

Sacha FLAMMIER, PHD

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 11, 2019

First Posted

October 15, 2019

Study Start

January 1, 2020

Primary Completion

January 1, 2021

Study Completion

December 1, 2023

Last Updated

October 15, 2019

Record last verified: 2019-10

Locations