Safety and Efficacy of Hectorol in Pediatric Patients With Chronic Kidney Disease Stage 3 and 4 With Secondary Hyperparathyroidism Not Yet on Dialysis

Phase 3TerminatedNCT02859896

Sanofi21 enrolled

Overview

Primary Objective: Evaluated the effect of Hectorol® capsules in reducing elevated levels of intact parathyroid hormone (iPTH). Secondary Objectives: * Evaluated the safety profile of Hectorol® capsules versus Rocaltrol® (calcitriol) capsules. * Determined the pharmacokinetic profile of 1,25-dihydroxyvitamin D2 after administration of Hectorol®.

The total study duration per participant was approximately up to 28 weeks.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Secondary Hyperparathyroidism-Chronic Kidney Disease

Interventions

Doxercalciferol (GZ427397)DRUG

Pharmaceutical form: capsule Route of administration: oral

CalcitriolDRUG

Pharmaceutical form: capsule Route of administration: oral

Eligibility

Sex: ALLMin age: 5 YearsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion criteria : * Male or female aged 5 to 18 years old. * Weight ≥15 kg. * Chronic kidney disease (CKD) Stage 3 or 4 not on dialysis, defined as glomerular filtration rate (GFR) between 15 and 59 mL/min/1.73m\^2 (established by Schwartz equation) at Week -2 visit. * Intact parathyroid hormone (iPTH) value \>100 pg/mL for CKD Stage 3 or \>160 pg/mL for CKD Stage 4, at Week -2 visit. * Signed informed consent/assent form. Exclusion criteria: * The participant had a serum 25-hydroxyvitamin D level \<30 ng/mL at screening. * The participant had a corrected calcium ≥10 mg/dL at the Week -2 visit. * The participant had a serum phosphorus \>4.5 mg/dL for children 13 to 18 years of age; \>5.8 mg/dL for children 5 to 12 years of age at the Week -2 visit. * The participant was anticipated to require maintenance hemodialysis within 3 months. * The participant used cinacalcet or vitamin D sterol therapies such as calcitriol, doxercalciferol, or paricalcitol within 14 days prior to the baseline visit. * The participant had a history of, or active, symptomatic heart disease within 12 months prior to the baseline (Week 0) visit. * The participant had a chronic gastrointestinal disease (ie, malabsorption, severe chronic diarrhea, chronic ulcerative colitis, or ileostomy). * The participant had primary hyperparathyroidism or has had a total parathyroidectomy. * The participant had an active malignancy. * The participant was unable to swallow a capsule in size similar to the Hectorol® and Rocaltrol® capsules. * The participant had a history of sensitivity or allergy to doxercalciferol, calcitriol or other vitamin D analogs. * The participant used aluminum or magnesium-based binders. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Locations (23)

Outcomes

Primary Outcomes

Percentage of Participants Who Achieved 2 Consecutive >=30% Reductions in Intact Parathyroid Hormone From Baseline up to Week 12

Blood samples were collected for assessment of iPTH levels. The percentage of participants meeting the iPTH \>=30% reduction from baseline at 2 consecutive study visits up to Week 12 was calculated. Two consecutive \>=30% reductions in iPTH from baseline up to Week 12 was defined as two consecutive 30% or greater reductions at any two consecutive measurements from baseline up to Week 12 with on-treatment strategy applied. The confidence interval (CI) was estimated using Clopper-Pearson method. The baseline value is defined as the last available value before the first dose of study treatment. Percentages are rounded off to the tenth decimal place.

Time frame: Baseline (Day 1) up to Week 12

Secondary Outcomes

Percent Change in Intact Parathyroid Hormone From Baseline to Weeks 12 and 24

Blood samples were collected for assessment of iPTH levels. The percentage changes from baseline iPTH, the effects over the treatment period time was explored using a mixed model for repeated measures approach (MMRM) as appropriate. The baseline value is defined as the last available value before the first dose of study treatment.

Time frame: Baseline (Day 1) to Weeks 12 and 24

Number of Hypercalcemia Events up to Weeks 12 and 24

Hypercalcemia was defined as albumin corrected serum calcium \>10.2 milligrams per deciliter (mg/dL). Here, data for number of hypercalcemia events are reported.

Time frame: Up to Weeks 12 and 24

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant who was administered a pharmaceutical product, and which did not necessarily have a causal relationship with this treatment. An AE occurred or was detected from the date the participant signed the informed consent form, irrespective of study periods without administration of the study treatment. TEAEs were defined as the AEs that developed, worsened or became serious during the treatment-emergent period (defined as time from administration of study treatment \[Day 1\] to last administration of study treatment + 4 days). SAE: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.

Data from ClinicalTrials.gov

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Children's of Alabama- Site Number : 8400022

Birmingham, Alabama, United States

Cedars-Sinai Medical Center- Site Number : 8400033

Los Angeles, California, United States

University of California Davis Health- Site Number : 8400005

Sacramento, California, United States

Yale University School of Medicine- Site Number : 8400029

New Haven, Connecticut, United States

University of Miami Hospital- Site Number : 8400006

Miami, Florida, United States

Nicklaus Children's Hospital - Miami - Southwest 62nd Avenue- Site Number : 8400008

Miami, Florida, United States

Rush University Medical Center- Site Number : 8400020

Chicago, Illinois, United States

M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400014

Minneapolis, Minnesota, United States

Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400010

Hackensack, New Jersey, United States

Goryeb Chidlren's Hospital- Site Number : 8400016

Morristown, New Jersey, United States

...and 13 more locations