An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
1 września 2020 zaktualizowane przez: Novartis Pharmaceuticals
A Multicenter, Randomized, Double-blind, Placebo Controlled Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly Compared to Placebo in Osteoporotic Children Treated With Glucocorticoids.
This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids
Przegląd badań
Szczegółowy opis
In March 2017, Novartis stopped enrollment as the study was not feasible to be conducted due to low enrollment and other recruitment challenges.
Patients receiving the treatment continued to receive the treatment per protocol.
Typ studiów
Interwencyjne
Zapisy (Rzeczywisty)
34
Faza
- Faza 3
Kontakty i lokalizacje
Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.
Lokalizacje studiów
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Gauteng
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Soweto, Gauteng, Afryka Południowa, 2013
- Novartis Investigative Site
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New South Wales
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Westmead, New South Wales, Australia, 2145
- Novartis Investigative Site
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Moscow, Federacja Rosyjska, 119991
- Novartis Investigative Site
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Saint Petersburg, Federacja Rosyjska, 195067
- Novartis Investigative Site
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British Columbia
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Vancouver, British Columbia, Kanada, V6H 3V4
- Novartis Investigative Site
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Manitoba
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Winnipeg, Manitoba, Kanada, R3E 0Z2
- Novartis Investigative Site
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Ontario
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Ottawa, Ontario, Kanada, K1H 8L1
- Novartis Investigative Site
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Quebec
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Montreal, Quebec, Kanada, H3T 1C5
- Novartis Investigative Site
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Montreal, Quebec, Kanada, H3H 1P3
- Novartis Investigative Site
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Budapest, Węgry, 1085
- Novartis Investigative Site
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Manchester, Zjednoczone Królestwo, M14 0JH
- Novartis Investigative Site
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Birmingham
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West Midlands, Birmingham, Zjednoczone Królestwo, B4 6NH
- Novartis Investigative Site
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Kryteria uczestnictwa
Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.
Kryteria kwalifikacji
Wiek uprawniający do nauki
5 lat do 17 lat (Dziecko)
Akceptuje zdrowych ochotników
Nie
Płeć kwalifikująca się do nauki
Wszystko
Opis
Key Inclusion Criteria:
- A diagnosis of chronic rheumatologic conditions or inflammatory bowel disease or Duchenne muscular dystrophy requiring systemic glucocorticoids (i.v. or oral) within 12 months prior to screening
- Lumbar Spine BMDZ-score of -0.5 or worse
- Evidence of at least at least 1 vertebral compression fracture of Genant Grade 1 or higher (or radiographic signs of vertebral fracture) within 1 month from Screening visit OR One or more, low-trauma, lower extremity long-bone fracture which occurred sometime within the 2 years PRECEDING enrollment in the study OR Two or more, low-trauma, upper extremity long-bone fractures which occurred sometime within the 2 years PRECEDING enrollment in the study
- Consent/assent to study participation
Key Exclusion Criteria:
- History of primary bone disease (OI, Idiopathic Juvenile Osteoporosis, Rickets/Osteomalacia)
- Any medical condition that might have interfered with the evaluation of lumbar spine BMD, such as severe scoliosis or spinal fusion. Patients with less than 3 evaluable vertebrae by Dual Energy X-ray Absorptiometry (DXA) evaluation in the region of interest lumbar 1 (L1) to lumbar 4 (L4),
- Hypocalcemia and hypophosphatemia
- Serum 25-hydroxy vitamin D concentrations of <20 ng/mL or <50 nmol/L
- estimated glomerular filtration rate (GFR) <60 mL/min/1.73 m2
- serum creatinine increase between Visit 1 and Visit 2 >0.5 mg/dL (44.2 μmol/L)
- Uncontrolled symptoms of cardiac failure or arrhythmia
- Any prior use of bisphosphonates, or high dose sodium fluoride
Plan studiów
Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Randomizowane
- Model interwencyjny: Przydział równoległy
- Maskowanie: Poczwórny
Broń i interwencje
Grupa uczestników / Arm |
Interwencja / Leczenie |
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Eksperymentalny: Kwas zoledronowy
Dwa razy w roku 0,05 mg/kg mc. (maks. 5 mg) w infuzji dożylnej (co najmniej 30 minut) kwasu zoledronowego
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infuzja dożylna
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Komparator placebo: Placebo
Twice yearly i.v of infusion of Placebo (similar dosing as active drug)
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infuzja dożylna
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Co mierzy badanie?
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
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Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12
Ramy czasowe: Month 12
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Lumbar Spine Bone Mineral Density (BMD) Z-score was determined by the central imaging vendor before first treatment and at Month 12.
The methods to be used to measure Lumbar Spine BMD Z-score were described in the respective DXA Manuals provided by central imaging vendor.
Positive changes from baseline indicated an improvement in condition.
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Month 12
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Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
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Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6
Ramy czasowe: Month 6
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Lumbar Spine Bone Mineral Density (BMD) Z-score was determined by the central imaging vendor before first treatment and at Month 6.
The methods to be used to measure Lumbar Spine BMD Z-score were described in the respective DXA Manuals provided by central imaging vendor.
Positive changes from baseline indicated an improvement in condition.
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Month 6
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Mean Change From Baseline in Lumbar Spine BMC at Month 6 and 12
Ramy czasowe: Month 6, Month 12
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Lumbar Spine BMC was determined by the central imaging vendor before first treatment and at Months 6 and 12.
The methods to be used to measure BMC were described in the respective DXA Manuals.
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Month 6, Month 12
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Mean Change From Baseline in Total Body BMC at Month 6 and 12
Ramy czasowe: Month 6, Month 12
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Total body BMC was all determined by the central imaging vendor before first treatment and at Months 6 and 12.
The methods to be used to measure BMC were described in the respective DXA Manuals.
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Month 6, Month 12
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Mean Change From Baseline in Serum P1NP at Months 6 and 12
Ramy czasowe: Month 6, Month 12
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Serum Procollagen type 1 amino-terminal propeptide (P1NP) was collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Mean Change From Baseline in BSAP at Months 6 and 12
Ramy czasowe: Month 6, Month 12
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Bone specific alkaline phosphatase (BSAP) were collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Mean Change From Baseline in Serum NTX at Months 6 and 12
Ramy czasowe: Month 6, Month 12
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Serum Cross linked N-telopeptide (NTX) were collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Mean Change From Baseline in Serum TRAP-5b at Months 6 and 12
Ramy czasowe: Month 6, Month 12
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Serum Tartrate-resistant acid phosphatase isoform 5b (TRAP 5b) was collected before first treatment (baseline) and at Months 6 and Month 12 according to the instructions provided in the Laboratory Manual.
The samples were analyzed in batches at the laboratory.
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Month 6, Month 12
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Number of Participants With New Vertebral Fractures at Month 12
Ramy czasowe: Month 12
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New vertebral fractures were defined as fractures of Genant Grade 1 or higher that occurred at lumbar or thoracic spine from first dose infusion to the end of the study.
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Month 12
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Mean Change From Baseline in Vertebral Morphometry at Month 12
Ramy czasowe: Month 12
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Vertebral morphometry (or concave index) was calculated using the average ratio between mid-height and posterior height from L1 to L4 and performed by a central reader.
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Month 12
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Percentage of Patients With Reduction in Pain at Months 3, 6, 9 and 12
Ramy czasowe: Month 3, Month 6, Month 9 and Month 12
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Pain was evaluated at each visit (in office and telephone visit) at randomization, Months 3, 6, 9 and 12 using the Faces Pain Scale-Revised (FPS-R).
Children were selecting the face that best fits their pain.
The pain score ranged from 0 (No Pain) to 10 (Very Much Pain).
The reduction in pain from baseline by visit was evaluated based on whether or not patients had a decrease in their FPS-R from baseline.
If pain remained the same or worsened from baseline a patient was classified as '0' and if the pain scale decreased then the patient was classified as '1'.
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Month 3, Month 6, Month 9 and Month 12
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Mean Change From Baseline in 2nd Metacarpal Cortical Width at Month 12
Ramy czasowe: Month 12
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Left posteroanterior (PA) hand/wrist X-ray were taken at Visit 1 and at the Month 12 visit to assess bone age and the between-treatment differences for change in 2nd metacarpal cortical width at Month 12 relative to baseline.
If a fracture of the left upper extremity precluded radiographic imaging, then the right hand was evaluated for this purpose.
In this case, the right hand was be imaged at both Visit 1 and at Month 12.
The information was used in the assessment of bone density.
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Month 12
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Urinary Concentration of Zoledronic Acid at Month 12
Ramy czasowe: Month 12
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Urine was collected overnight or for at least 4 waking hours from all patients able to provide specimens, to measure urinary concentration of zoledronic acid at Month 12.
Only descriptive analysis done.
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Month 12
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Safety of Zoledronic Acid for the Treatment of Osteoporotic Children Treated With Glucocorticoids
Ramy czasowe: Baseline through Month 12
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Analysis of absolute and relative frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC) to demonstrate that zoledronic acid is safe for the treatment of osteoporotic children treated with glucocorticoids through the monitoring of relevant clinical and laboratory safety parameters.
Only descriptive analysis done.
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Baseline through Month 12
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Współpracownicy i badacze
Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.
Sponsor
Publikacje i pomocne linki
Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.
Daty zapisu na studia
Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.
Główne daty studiów
Rozpoczęcie studiów (Rzeczywisty)
4 grudnia 2008
Zakończenie podstawowe (Rzeczywisty)
5 marca 2018
Ukończenie studiów (Rzeczywisty)
5 marca 2018
Daty rejestracji na studia
Pierwszy przesłany
26 listopada 2008
Pierwszy przesłany, który spełnia kryteria kontroli jakości
26 listopada 2008
Pierwszy wysłany (Oszacować)
27 listopada 2008
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
2 września 2020
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
1 września 2020
Ostatnia weryfikacja
1 września 2020
Więcej informacji
Terminy związane z tym badaniem
Dodatkowe istotne warunki MeSH
Inne numery identyfikacyjne badania
- CZOL446H2337
- 2008-001252-52 (Numer EudraCT)
Plan dla danych uczestnika indywidualnego (IPD)
Planujesz udostępniać dane poszczególnych uczestników (IPD)?
NIEZDECYDOWANY
Opis planu IPD
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .