Pharmacoepidemiological Study of Secondary Hyperparathyroidism in Lorraine

ESRD Clinical Trial

— EPHEYL  

Official title:

EPHEYL: Étude PHarmacoÉpidémiologique de l'hYperparathyroïdie Secondaire en Lorraine

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02888639
• Other study ID #: EPHEYL
• Status: Completed
• Phase: N/A
• First received: August 25, 2016
• Last updated: August 30, 2016
• Start date: December 2009
• Est. completion date: May 2012

Study information

• Verified date: August 2016
• Source: Central Hospital, Nancy, France
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Comité consultatif sur le traitement de l'information en matière de recherche dans le domaine de la santé
• Study type: Observational

Clinical Trial Summary

The pharmacoepidemiological EPHEYL (Étude PHarmacoÉpidémiologique de l'hYperparathyroïdie secondaire en Lorraine) study is a 2-year, open-cohort, prospective, observational study on incident SHPT, i.e. newly diagnosed, with a 2 year follow-up, set in the 12 dialysis units located in the French region of Lorraine (public or private).

Description:

In the early stages of the disease, 90% of patients with chronic renal failure present evidence of bone remodeling due to secondary hyperparathyroidism (HPT). As the disease progresses and patients require dialysis for end-stage renal failure (ESRF), the incidence of secondary HPT declines to 25-40% with reduced bone remodeling as shown by osteomalacia observed in 4-12% of patients and adynamic bone in 20-40%. Observational studies have described an association between serum phosphorus level, phosphorus-calcium product, parathormone (PTH) level, and mortality. Since there is no real consensus, treatments, basically diet and drugs, are tailored to each individual. The therapeutic range is wide and interactions are frequent. Therapeutic efficacy is compromised by the variability of PTH assay results, the ambulatory nature of interventions, and poor patient adherence. The guidelines issued in 2003 stipulate that corrective measures, i.e. dietetic counseling with or without drug therapy, are needed when serum PTH exceeds 300 pg/ml. When the PTH level exceeds 500, or even more so 800 pg/ml, HPT can constitute an indication for parathyroid surgery or calcimimetic treatment.

Data from a randomized comparison between cinacalcet and surgery are lacking. The evidence-based decision for surgery is made conjointly by the nephrologist, the surgeon and the patient, limiting the feasibility of randomization. Parathyroidectomy can be achieved by three techniques whose efficacy has been studied in historical observational studies. Cinacalcet received marketing approval in 2004 for "the treatment of secondary HPT in patients with ESRF treated by dialysis. Mimpara can be used in the context of a regimen including phosphate chelators and/or vitamin D analogs, as needed". Four double-blind studies have demonstrated the efficacy of cinacalcet versus placebo in decreasing the PTH level, but there is no proof of the efficacy of cinacalcet in terms of cardiovascular events and mortality. One cost-effectiveness study showed that parathyroidectomy was superior to cinacalcet, except if the patient was on dialysis for a short period before renal transplantation or presented contraindications for surgery.

While randomized trials are the gold standard, such trials include a limited number of patients for a limited period of time. This does not enable an observation of long-term practices and adverse effects. Moreover, such trials do not concern patients with other co-morbid conditions taking several drugs, in particular, elderly subjects. The concept of the pharmacoepidemiological study is to describe real-life populations taking a given drug, using both quantitative and qualitative parameters to determine efficacy, risk, and drug use in real-life conditions free of the constraints of a clinical trial.

ÉPHÉYL is an observational pharmacoepidemiological study concerning the prescription of calcimimetic agents and parathyroidectomy in patients with ESRF on dialysis who present severe secondary HPT (PTH ≥ 500 pg/ml). The aim is to describe indications and efficacy: control of PTH, calcium and phosphorus serum levels within their recommended ranges, patient tolerance of the therapeutic interventions, and the cost of such interventions, as well as patients' adhesion to treatment and their self-assessed quality-of-life. This three-year inclusion study, based on the Lorraine REIN registry, will involve all participants in the Néphrolor network. The study will begin on April 1st, 2009.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 305
• Est. completion date: May 2012
• Est. primary completion date: May 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• age >= 18 years

• Patient on hemodialysis or peritoneal dialysis for 3 months or more in one of the 12 dialysis units in the study.

• Patient included in the REIN registry in the Lorraine region.

• Parathormone greater than or equal to 500 pg / ml for the first time after 01/04/2009 (patient incident for secondary hyperparathyroidism).

Exclusion Criteria:

• age < 18 years.

• Patient living in Lorraine but dialysed in a unit outside Lorraine.

• Diagnosis of secondary hyperparathyroidism with higher parathormone or equal to 500 pg / ml placed before 1/4/2009.

• Patient who have received medical treatment with cinacalcet or by surgical parathyroidectomy for secondary hyperparathyroidism.

• Patient expressing opposition to the collection of information.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• ESRD
• Hyperparathyroidism, Secondary

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Central Hospital, Nancy, France

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Parathormone level	Obtaining the therapeutic target according to the K/DOQI	2 years	No
Secondary	Phosphocalcium product level		2 years	No
Secondary	Serum calcium level		2 years	No
Secondary	Serum phosphorus level		2 years	No
Secondary	Symptoms and / or clinical events caused by hyperparathyroidism	using clinical and paraclinical examinations	2 years	No
Secondary	Tolerance of therapeutic interventions: nausea, vomiting, diarrhea, hypocalcemia	using clinical and paraclinical examinations	2 years	No
Secondary	The quality of life	using the KDQOL questionnaire	2 years	No
Secondary	Satisfaction of professionals	Interview of professionals on patient care	2 years	No
Secondary	The direct costs of treatment	Cost related to medical treatment, parathyroidectomy, hospitalisation, clinical and paraclinical examination	2 years	No
Secondary	Patient compliance	using the CET (Compliance Evaluation Test ) Questionnaire	2 years	No