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

NCT02859896 | Terminated Phase 3 FDA Drug

• 2 other identifiers: LPS14314U1111-1178-4657
• Study Type: interventional
• Target: 21
• Locations: 2 countries
• Sites: 23

Brief Summary

Primary Objective: Evaluate the effect of Hectorol® capsules in reducing elevated levels of intact parathyroid hormone (iPTH). Secondary Objectives:

• Evaluate the safety profile of Hectorol® capsules versus Rocaltrol® (calcitriol) capsules.
• Determine the pharmacokinetic profile of 1,25-dihydroxyvitamin D2 after administration of Hectorol®.

Conditions

Secondary Hyperparathyroidism-Chronic Kidney Disease

Outcome Measures

Primary Outcomes (1)

• Percentage of participants achieving two consecutive ≥30% reductions in iPTH

  Baseline up to Week 12

Secondary Outcomes (4)

• Percentage change from baseline in iPTH

  Baseline, Week 12, Week 24

• Number of hypercalcemia events (albumin corrected serum calcium >10.2 mg/dL)

  Up to Weeks 12 and 24

• Number of participants with adverse events

  Baseline up to Week 24

• Assessment of pharmacokinetic (PK) parameter: Serum 1,25-dihydroxyvitamin D2 concentration-time data

  At Week -2, Baseline, and then within 24 hours of the most recent dose of Hectorol® at Weeks 2, 4, 6, 8 or 10 (whichever week is not used for serial PK assessment), and 12

Study Arms (2)

Hectorol

EXPERIMENTAL

Hectorol (Doxercalciferol) will be administered orally two to three times weekly dependent on patient age. A dose titration scheme is used to individualize the dose to the patient's iPTH management.

Drug: Doxercalciferol (GZ427397)

Rocaltrol

ACTIVE COMPARATOR

Rocaltrol (Calcitriol) will be administered orally seven days/week. A dose titration scheme is used to individualize the dose to the patient's iPTH management.

Drug: Calcitriol

Interventions

Doxercalciferol (GZ427397) DRUG

Pharmaceutical form: capsule Route of administration: oral

Also known as: Hectorol

Hectorol

Calcitriol DRUG

Pharmaceutical form: capsule Route of administration: oral

Also known as: Rocaltrol

Rocaltrol

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• The patient has a serum 25-hydroxyvitamin D level \<30 ng/mL at screening.
• The patient has a corrected calcium ≥10 mg/dL at the Week -2 visit.
• The patient has 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 patient is anticipated to require maintenance hemodialysis within 3 months.
• The patient used cinacalcet or vitamin D sterol therapies such as calcitriol, doxercalciferol, or paricalcitol within 14 days prior to the baseline visit.
• The patient has a history of, or active, symptomatic heart disease within 12 months prior to the baseline (Week 0) visit.
• The patient currently has a chronic gastrointestinal disease (ie, malabsorption, severe chronic diarrhea, chronic ulcerative colitis, or ileostomy).
• The patient currently has primary hyperparathyroidism or has had a total parathyroidectomy.
• The patient has an active malignancy.
• The patient is unable to swallow a capsule in size similar to the Hectorol® and Rocaltrol® capsules.
• The patient has a history of sensitivity or allergy to doxercalciferol, calcitriol or other vitamin D analogs.
• The patient currently uses aluminum or magnesium-based binders.
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (23)

Children's Hospital Of Alabama Site Number : 8400022

Birmingham, Alabama, 35233, United States

Location

Cedars Sinai Medical Center Site Number : 8400033

Los Angeles, California, 90048, United States

Location

UC DAVIS Medical Center Site Number : 8400005

Sacramento, California, 95817, United States

Location

Yale University School of Medicine Site Number : 8400029

New Haven, Connecticut, 06520-8017, United States

Location

University of Miami Site Number : 8400006

Miami, Florida, 33101, United States

Location

Nicklaus Children's Hospital Site Number : 8400008

Miami, Florida, 33155, United States

Location

Rush University Medical Center Site Number : 8400020

Chicago, Illinois, 60612, United States

Location

University of Minnesota Site Number : 8400014

Minneapolis, Minnesota, 55454, United States

Location

Hackensack University Site Number : 8400010

Hackensack, New Jersey, 07601, United States

Location

Goryeb Children's Hospital Site Number : 8400016

Morristown, New Jersey, 07962, United States

Location

Cohen Children's Medical Center of NY Site Number : 8400017

New Hyde Park, New York, 11040, United States

Location

Mount Sinai Medical Center Site Number : 8400007

New York, New York, 10021, United States

Location

Duke University Medical Center Site Number : 8400034

Durham, North Carolina, 27710-4000, United States

Location

East Carolina University, Brody School of Medicine Site Number : 8400025

Greenville, North Carolina, 27834, United States

Location

Children's Hospital of Pittsburgh Site Number : 8400028

Pittsburgh, Pennsylvania, 15224, United States

Location

Greenville Hospital System Site Number : 8400027

Greenville, South Carolina, 29605, United States

Location

Vanderbilt University Medical Center Site Number : 8400024

Nashville, Tennessee, 37292, United States

Location

Texas Children's Hospital Site Number : 8400013

Houston, Texas, 77030, United States

Location

University of Utah Site Number : 8400026

Salt Lake City, Utah, 84113, United States

Location

Virginia Commonwealth University Site Number : 8400009

Richmond, Virginia, 23298, United States

Location

Marshfield Clinic Site Number : 8400001

Marshfield, Wisconsin, 54449, United States

Location

Investigational Site Number : 1520003

Santiago, Reg Metropolitana de Santiago, Chile

Location

Investigational Site Number : 1520004

Concepción, Región del Biobío, Chile

Location

MeSH Terms

Interventions

1 alpha-hydroxyergocalciferol; Calcitriol

Intervention Hierarchy (Ancestors)

Dihydroxycholecalciferols; Hydroxycholecalciferols; Cholecalciferol; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 4, 2016
• First Posted: August 9, 2016
• Study Start: January 19, 2017
• Primary Completion: June 11, 2025
• Study Completion: June 11, 2025
• Last Updated: July 29, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

US (21); CL (2)