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

Sponsor

Sanofi (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02859896

Collaborator

(none)

Enrollment

Locations

Arms

105.8

Anticipated Duration (Months)

2.8

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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®.

Condition or Disease	Intervention/Treatment	Phase
Secondary Hyperparathyroidism-Chronic Kidney Disease	Drug: Doxercalciferol (GZ427397) Drug: Calcitriol	Phase 3

Detailed Description

The total study duration per patient will be approximately up to 28 weeks.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

84 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jan 19, 2017

Anticipated Primary Completion Date :

Nov 12, 2025

Anticipated Study Completion Date :

Nov 12, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Hectorol 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) Pharmaceutical form: capsule Route of administration: oral Other Names: Hectorol
Active Comparator: Rocaltrol 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 Pharmaceutical form: capsule Route of administration: oral Other Names: Rocaltrol

Arm

Intervention/Treatment

Experimental: Hectorol

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)

Pharmaceutical form: capsule Route of administration: oral

Other Names:

Hectorol

Active Comparator: Rocaltrol

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

Pharmaceutical form: capsule Route of administration: oral

Other Names:

Rocaltrol

Outcome Measures

Primary Outcome Measures

Percentage of participants achieving two consecutive ≥30% reductions in iPTH [Baseline up to Week 12]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigational Site Number :8400022	Birmingham	Alabama	United States	35233
2	Investigational Site Number :8400023	Los Angeles	California	United States	90027
3	Investigational Site Number :8400033	Los Angeles	California	United States	90048
4	Investigational Site Number :8400005	Sacramento	California	United States	95817
5	Investigational Site Number :8400029	New Haven	Connecticut	United States	06520-8017
6	Investigational Site Number :8400006	Miami	Florida	United States	33101
7	Investigational Site Number :8400008	Miami	Florida	United States	33155
8	Investigational Site Number :8400020	Chicago	Illinois	United States	60612
9	Investigational Site Number :8400036	Indianapolis	Indiana	United States	46202
10	Investigational Site Number :8400014	Minneapolis	Minnesota	United States	55454
11	Investigational Site Number :8400015	Jackson	Mississippi	United States	39216
12	Investigational Site Number :8400010	Hackensack	New Jersey	United States	07601
13	Investigational Site Number :8400016	Morristown	New Jersey	United States	07962
14	Investigational Site Number :8400017	New Hyde Park	New York	United States	11040
15	Investigational Site Number :8400007	New York	New York	United States	10021
16	Investigational Site Number :8400021	Syracuse	New York	United States	13210
17	Investigational Site Number :8400034	Durham	North Carolina	United States	27710
18	Investigational Site Number :8400025	Greenville	North Carolina	United States	27834
19	Investigational Site Number :8400004	Portland	Oregon	United States	97227
20	Investigational Site Number :8400035	Portland	Oregon	United States	97239-3098
21	Investigational Site Number :8400028	Pittsburgh	Pennsylvania	United States	15224
22	Investigational Site Number :8400027	Greenville	South Carolina	United States	29605
23	Investigational Site Number :8400024	Nashville	Tennessee	United States	37292
24	Investigational Site Number :8400013	Houston	Texas	United States	77030
25	Investigational Site Number :8400019	Houston	Texas	United States	77030
26	Investigational Site Number :8400026	Salt Lake City	Utah	United States	84113
27	Investigational Site Number :8400009	Richmond	Virginia	United States	23298
28	Investigational Site Number :8400001	Marshfield	Wisconsin	United States	54449
29	Investigational Site Number :1520004	Concepción	Biobío	Chile
30	Investigational Site Number :1520003	Santiago	Reg Metropolitana De Santiago	Chile