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 Results FDA Drug

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

Brief Summary

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®.

Conditions

Secondary Hyperparathyroidism-Chronic Kidney Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline (Day 1) up to Week 12

Secondary Outcomes (7)

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

  Baseline (Day 1) to Weeks 12 and 24

• Number of Hypercalcemia Events up to Weeks 12 and 24

  Up to Weeks 12 and 24

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

  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

  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

  Pre-dose, 1, 4, 7, and 24 hours post-dose at Week 8 or 10

Study Arms (2)

Hectorol

EXPERIMENTAL

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)

Rocaltrol

ACTIVE COMPARATOR

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

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 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (23)

Children's 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

University of California Davis Health- Site Number : 8400005

Sacramento, California, 95817, United States

Location

Yale University School of Medicine- Site Number : 8400029

New Haven, Connecticut, 06510, United States

Location

University of Miami Hospital- Site Number : 8400006

Miami, Florida, 33136, United States

Location

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

Miami, Florida, 33155, United States

Location

Rush University Medical Center- Site Number : 8400020

Chicago, Illinois, 60612, United States

Location

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

Minneapolis, Minnesota, 55454, United States

Location

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

Hackensack, New Jersey, 07601, United States

Location

Goryeb Chidlren's Hospital- Site Number : 8400016

Morristown, New Jersey, 07962, United States

Location

Cohen Children's Medical Center- Site Number : 8400017

New Hyde Park, New York, 11040, United States

Location

The Mount Sinai Hospital- Site Number : 8400007

New York, New York, 10029, United States

Location

Duke University Medical Center- Site Number : 8400034

Durham, North Carolina, 27710, United States

Location

East Carolina University- Site Number : 8400025

Greenville, North Carolina, 27858, United States

Location

UPMC Children's Hospital of Pittsburgh- Site Number : 8400028

Pittsburgh, Pennsylvania, 15224, United States

Location

Greenville Memorial Hospital- 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 Health Hospital- Site Number : 8400026

Salt Lake City, Utah, 84132, United States

Location

Virginia Commonwealth University Medical Center- Site Number : 8400009

Richmond, Virginia, 23219, United States

Location

Marshfield Medical Center - Marshfield- Site Number : 8400001

Marshfield, Wisconsin, 54449, United States

Location

Investigational Site Number : 1520004

Concepción, Biobio, 4070038, Chile

Location

Investigational Site Number : 1520003

Santiago, Reg Metropolitana de Santiago, 7500539, 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

Limitations and Caveats

The study was conducted to fulfill a PMC. The study was terminated as FDA acknowledged the fulfillment of this PMC on 11 June 2025.

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

800-633-1610

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

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 2, 2026
• Results First Posted: July 2, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share

Locations

US (21); CL (2)