- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT01549314
Cystic Fibrosis Related Bone Disease: the Role of CFTR
Visão geral do estudo
Status
Descrição detalhada
Tipo de estudo
Inscrição (Real)
Contactos e Locais
Locais de estudo
-
-
Massachusetts
-
Boston, Massachusetts, Estados Unidos, 02114
- Massachusetts General Hospital
-
-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
Descrição
COHORT 1
Inclusion Criteria:
- Age 6 to 75 years old
- Established diagnosis of CF with at least one abnormal G551D-CFTR allele
- Eligibility for and intent to start treatment with ivacaftor or started treatment with ivacaftor within previous 6 months
Exclusion Criteria:
- Psychiatric or mental incapacity that would preclude subject from assenting to study participation
- Current pregnancy
- History of organ transplantation
- History of Burkholderia dolosa infection
COHORT 2:
Subjects will be grouped by gender, age and race to match subjects in Cohort 1 within two years. Pubertal subjects will be matched by Tanner stage.
Inclusion Criteria:
- Age 6 to 75 years old
- Established diagnosis of CF
Exclusion Criteria:
- Psychiatric or mental incapacity that would preclude subject from assenting to study participation
- Current pregnancy
- History of organ transplantation
- History of Burkholderia dolosa infection
COHORT 3:
Subjects will be grouped by gender, age and race to match subjects in Cohort 2 within two years. Pubertal subjects will be matched by Tanner stage.
Inclusion criteria:
- Age 6 to 75 years old
- Clinically stable, deemed able to complete the screening, baseline, and scheduled study visits.
Exclusion criteria:
- History of significant cardiac, renal, pulmonary, hepatic, or malignant disease, current alcohol or illicit drug abuse, or major psychiatric disorder
- Current diagnoses known to affect bone metabolism, including cystic fibrosis, osteoporosis, amenorrhea >3 months (in menstruating women who are not taking oral contraceptives or have an IUD), hyperthyroidism, diabetes, hyperparathyroidism, Paget's disease, kidney stones, chronic inflammatory diseases, malabsorptive disorders, malnutrition, prolonged immobility, and skeletal dysplasias
- History of a non-digital fracture in the previous 6 months, history of one pathologic fracture, or greater than four total lifetime non-digital fractures
- Cumulative lifetime use of oral glucocorticoids for greater than 2 months
- Current or prior use of medications known to affect bone metabolism including hormone replacement therapy, anti-estrogens, bisphosphonates, calcitonin, fluoride, lithium, suppressive doses of levothyroxine, or anticonvulsants.
- Pregnancy
- BMI less than 18.5 or greater than 30 kg/m2 in subjects 18 years and older, or BMI less than 5th or greater than 95th percentile in subjects under the age of 18 years.
- Any medical or psychiatric condition or situation that would compromise subject safety, informed consent/assent, treatment compliance, follow-up measurements, or data quality
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
Coortes e Intervenções
Grupo / Coorte |
---|
Subjects with CF taking ivacaftor
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects with CF not taking ivacaftor
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy subjects
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
---|---|---|
Bone Microarchitecture and Strength Measures of the Radius and Tibia
Prazo: Baseline and 24 months
|
Change in cortical volumetric bone mineral density at the radius
|
Baseline and 24 months
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
---|---|---|
Areal Bone Mineral Density as Measured by DXA
Prazo: Baseline and 24 months
|
Change in PA spine bone mineral density
|
Baseline and 24 months
|
Bone Turnover Markers
Prazo: Baseline and 24 months
|
Change in osteocalcin
|
Baseline and 24 months
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Joel Finkelstein, MD, Massachusetts General Hospital
Publicações e links úteis
Publicações Gerais
- Aris RM, Merkel PA, Bachrach LK, Borowitz DS, Boyle MP, Elkin SL, Guise TA, Hardin DS, Haworth CS, Holick MF, Joseph PM, O'Brien K, Tullis E, Watts NB, White TB. Guide to bone health and disease in cystic fibrosis. J Clin Endocrinol Metab. 2005 Mar;90(3):1888-96. doi: 10.1210/jc.2004-1629. Epub 2004 Dec 21.
- Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.
- Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordonez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.
- MacNeil JA, Boyd SK. Accuracy of high-resolution peripheral quantitative computed tomography for measurement of bone quality. Med Eng Phys. 2007 Dec;29(10):1096-105. doi: 10.1016/j.medengphy.2006.11.002. Epub 2007 Jan 16.
- Shead EF, Haworth CS, Condliffe AM, McKeon DJ, Scott MA, Compston JE. Cystic fibrosis transmembrane conductance regulator (CFTR) is expressed in human bone. Thorax. 2007 Jul;62(7):650-1. doi: 10.1136/thx.2006.075887. No abstract available.
- Putman MS, Greenblatt LB, Bruce M, Joseph T, Lee H, Sawicki G, Uluer A, Sicilian L, Neuringer I, Gordon CM, Bouxsein ML, Finkelstein JS. The Effects of Ivacaftor on Bone Density and Microarchitecture in Children and Adults with Cystic Fibrosis. J Clin Endocrinol Metab. 2021 Mar 8;106(3):e1248-e1261. doi: 10.1210/clinem/dgaa890.
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Real)
Conclusão do estudo (Real)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Estimativa)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- MGH 2012P000269
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .