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Sikkerhed og effektivitet af Hectorol hos pædiatriske patienter med kronisk nyresygdom trin 3 og 4 med sekundær hyperparathyroidisme, der endnu ikke er i dialyse

8. juni 2026 opdateret af: Sanofi

Et åbent, randomiseret, parallelt gruppestudie for at vurdere sikkerheden og effektiviteten af ​​Hectorol® (Doxercalciferol-kapsler) hos pædiatriske patienter med kronisk nyresygdom, stadier 3 og 4 med sekundær hyperparathyroidisme, der endnu ikke er i dialyse

Primært mål:

Evaluer effekten af ​​Hectorol®-kapsler til at reducere forhøjede niveauer af intakt parathyroidhormon (iPTH).

Sekundære mål:

  • Evaluer sikkerhedsprofilen af ​​Hectorol® kapsler versus Rocaltrol® (calcitriol) kapsler.
  • Bestem den farmakokinetiske profil af 1,25-dihydroxyvitamin D2 efter administration af Hectorol®.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

Den samlede undersøgelsesvarighed pr. patient vil være ca. op til 28 uger.

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

21

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

1 år til 14 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

  • Mand eller kvinde i alderen 5 til 18 år.
  • Vægt ≥15 kg.
  • Kronisk nyresygdom (CKD) Stadium 3 eller 4 ikke i dialyse, defineret som glomerulær filtrationshastighed (GFR) mellem 15 og 59 ml/min/1,73 m^2 (etableret af Schwartz ligning) ved uge -2 besøg.
  • Værdi for intakt parathyreoideahormon (iPTH) >100 pg/mL for CKD Stadium 3 eller >160 pg/ml for CKD Stadium 4, ved uge -2 besøg.
  • Underskrevet informeret samtykke/samtykkeformular.

Ekskluderingskriterier:

  • Patienten har et serum 25-hydroxyvitamin D niveau
  • Patienten har en korrigeret calcium ≥10 mg/dL ved uge -2 besøg.
  • Patienten har et serumfosfor >4,5 mg/dL til børn i alderen 13 til 18 år; >5,8 mg/dL for børn i alderen 5 til 12 år ved uge -2 besøg.
  • Patienten forventes at have behov for vedligeholdelseshæmodialyse inden for 3 måneder.
  • Patienten brugte cinacalcet- eller vitamin D-sterolbehandlinger såsom calcitriol, doxercalciferol eller paricalcitol inden for 14 dage før baseline-besøget.
  • Patienten har en historie med, eller aktiv, symptomatisk hjertesygdom inden for 12 måneder før baseline-besøget (uge 0).
  • Patienten har i øjeblikket en kronisk gastrointestinal sygdom (dvs. malabsorption, svær kronisk diarré, kronisk colitis ulcerosa eller ileostomi).
  • Patienten har i øjeblikket primær hyperparathyroidisme eller har fået foretaget en total parathyreoidektomi.
  • Patienten har en aktiv malignitet.
  • Patienten er ude af stand til at sluge en kapsel i størrelse svarende til Hectorol®- og Rocaltrol®-kapslerne.
  • Patienten har en historie med følsomhed eller allergi over for doxercalciferol, calcitriol eller andre D-vitaminanaloger.
  • Patienten bruger i øjeblikket aluminium- eller magnesiumbaserede bindemidler.

Ovenstående information er ikke beregnet til at indeholde alle overvejelser, der er relevante for en patients potentielle deltagelse i et klinisk forsøg.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Medicin: Doxercalciferol (GZ427397)

Lægemiddelform: kapsel

Indgivelsesvej: oral

Andre navne:
  • Hectorol
Aktiv komparator: 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.
Medicin: Calcitriol

Lægemiddelform: kapsel

Indgivelsesvej: oral

Andre navne:
  • Rocaltrol

Hvad måler undersøgelsen?

Primære resultatmål

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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Percent Change in Intact Parathyroid Hormone From Baseline to Weeks 12 and 24
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Sanofi

Efterforskere

  • Studieleder: Clinical Sciences & Operations, Sanofi

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

19. januar 2017

Primær færdiggørelse (Faktiske)

11. juni 2025

Studieafslutning (Faktiske)

11. juni 2025

Datoer for studieregistrering

Først indsendt

4. august 2016

Først indsendt, der opfyldte QC-kriterier

4. august 2016

Først opslået (Anslået)

9. august 2016

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

2. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

8. juni 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • LPS14314
  • U1111-1178-4657 (Registry Identifier: ICTRP)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

Kvalificerede forskere kan anmode om adgang til data på patientniveau og relaterede undersøgelsesdokumenter, herunder den kliniske undersøgelsesrapport, undersøgelsesprotokol med eventuelle ændringer, blank case-rapportformular, statistisk analyseplan og datasætspecifikationer. Data på patientniveau vil blive anonymiseret, og undersøgelsesdokumenter vil blive redigeret for at beskytte forsøgsdeltagernes privatliv. Yderligere detaljer om Sanofis kriterier for datadeling, kvalificerede undersøgelser og proces for at anmode om adgang kan findes på: https://vivli.org

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Doxercalciferol (GZ427397)

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