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

June 8, 2026 updated by: Sanofi

An Open-Label, Randomized, Parallel Group Study to Assess the Safety and Efficacy of Hectorol® (Doxercalciferol Capsules) in Pediatric Patients With Chronic Kidney Disease Stages 3 and 4 With Secondary Hyperparathyroidism Not Yet on Dialysis

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®.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Chile
    • Biobio
      • Concepción, Biobio, Chile, 4070038
        • Investigational Site Number : 1520004
    • Reg Metropolitana de Santiago
      • Santiago, Reg Metropolitana de Santiago, Chile, 7500539
        • Investigational Site Number : 1520003
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Children's of Alabama- Site Number : 8400022
    • California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center- Site Number : 8400033
      • Sacramento, California, United States, 95817
        • University of California Davis Health- Site Number : 8400005
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale University School of Medicine- Site Number : 8400029
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami Hospital- Site Number : 8400006
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital - Miami - Southwest 62nd Avenue- Site Number : 8400008
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center- Site Number : 8400020
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400014
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400010
      • Morristown, New Jersey, United States, 07962
        • Goryeb Chidlren's Hospital- Site Number : 8400016
    • New York
      • New Hyde Park, New York, United States, 11040
        • Cohen Children's Medical Center- Site Number : 8400017
      • New York, New York, United States, 10029
        • The Mount Sinai Hospital- Site Number : 8400007
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center- Site Number : 8400034
      • Greenville, North Carolina, United States, 27858
        • East Carolina University- Site Number : 8400025
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • UPMC Children's Hospital of Pittsburgh- Site Number : 8400028
    • South Carolina
      • Greenville, South Carolina, United States, 29605
        • Greenville Memorial Hospital- Site Number : 8400027
    • Tennessee
      • Nashville, Tennessee, United States, 37292
        • Vanderbilt University Medical Center- Site Number : 8400024
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital- Site Number : 8400013
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Health Hospital- Site Number : 8400026
    • Virginia
      • Richmond, Virginia, United States, 23219
        • Virginia Commonwealth University Medical Center- Site Number : 8400009
    • Wisconsin
      • Marshfield, Wisconsin, United States, 54449
        • Marshfield Medical Center - Marshfield- Site Number : 8400001

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 14 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Hectorol
Hectorol (Doxercalciferol) was administered orally two to three times weekly dependent on participant age. A dose titration scheme was used to individualize the dose to the participant's iPTH management.
Drug: Doxercalciferol (GZ427397)

Pharmaceutical form: capsule

Route of administration: oral

Other Names:
  • Hectorol
Active Comparator: Rocaltrol
Rocaltrol (Calcitriol) was administered orally seven days/week. A dose titration scheme was used to individualize the dose to the participant's iPTH management.
Drug: Calcitriol

Pharmaceutical form: capsule

Route of administration: oral

Other Names:
  • Rocaltrol

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants Who Achieved 2 Consecutive >=30% Reductions in Intact Parathyroid Hormone From Baseline up to Week 12
Time Frame: Baseline (Day 1) 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.
Baseline (Day 1) up to Week 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change in Intact Parathyroid Hormone From Baseline to Weeks 12 and 24
Time Frame: Baseline (Day 1) 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.
Baseline (Day 1) to Weeks 12 and 24
Number of Hypercalcemia Events up to Weeks 12 and 24
Time Frame: 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.
Up to Weeks 12 and 24
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
Time Frame: From first dose of study treatment (Day 1) up to 4 days after the last dose of study treatment; approximately 36 weeks
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.
From first dose of study treatment (Day 1) up to 4 days after the last dose of study treatment; approximately 36 weeks
Maximum Observed Plasma Concentration (Cmax) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10
Time Frame: Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine Cmax. Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods. As pre-specified in protocol pharmacokinetic (PK) parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Time to Maximum Plasma Concentration (Tmax) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10
Time Frame: Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine tmax. Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods. As pre-specified in protocol PK parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24h) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10
Time Frame: Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine AUC0-24h. Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods. As pre-specified in protocol PK parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Trough Plasma Concentration (Ctrough) of 1,25-Dihydroxyvitamin D2 at Week 8 or 10
Time Frame: Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10
Blood samples were collected at specified timepoints after administration of doxercalciferol (Hectorol®) to determine Ctrough. Evaluation of the 1, 25-Dihydroxyvitamin D2 concentration-time data was obtained using non-compartmental methods. As pre-specified in protocol PK parameters were assessed at Week 8 or Week 10 choice was as per the schedule availability of the site and the participants.
Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sanofi

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 19, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 11, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 11, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 4, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 4, 2016

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 9, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 2, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 8, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • LPS14314
  • U1111-1178-4657 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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