Cystic Fibrosis Related Bone DiseaseNCT01549314
Cystic Fibrosis Related Bone Disease: the Role of CFTR
An observational study of Cystic Fibrosis Related Bone Disease, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
The purpose of this study is to determine whether ivacaftor, a recently FDA-approved CFTR potentiator, improves bone micro-architecture and strength in patients with cystic fibrosis with at least one G551D CFTR mutation. Read more about this study
- Who is it for?
- Ages 6 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis Related Bone Disease
- How many people
- 79 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2012to Feb 2020
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 75 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Subjects with CF will be recruited from the Cystic Fibrosis Centers at Children's Hospital Boston and Massachusetts General Hospital. Healthy subjects will be a community sample recruited from nearby communities.
Who may be able to join
- 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
- Age 6 to 75 years old
- Established diagnosis of CF
- Age 6 to 75 years old
- Clinically stable, deemed able to complete the screening, baseline, and scheduled study visits.
Who cannot join
- 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.
Show all 20Show fewer
- 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.
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
The purpose of this study is to determine whether ivacaftor, a recently FDA-approved CFTR potentiator, improves bone micro-architecture and strength in patients with cystic fibrosis with at least one G551D CFTR mutation.
Read the detailed description
Ivacaftor, a CFTR potentiator, has recently been FDA approved for the treatment of cystic fibrosis in patients with at least one G551D CFTR mutation. Given the possible role of CFTR in bone, we hypothesize that this medication may also improve bone health in CF patients. The purpose of this study is to test this hypothesis using high resolution peripheral quantitative computed tomography, a research tool that measures bone micro-architecture and volumetric bone density and has the ability to detect small changes in bone that might otherwise be missed with standard bone imaging techniques such as bone density testing.
- Conditions
- Cystic Fibrosis Related Bone Disease
- Keywords
- Cystic fibrosis related bone disease, Cystic fibrosis, Ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone Microarchitecture and Strength Measures of the Radius and Tibia
Change in cortical volumetric bone mineral density at the radius
Measured: Baseline and 24 months
Secondary outcomes (2)
Areal Bone Mineral Density as Measured by DXA
Change in PA spine bone mineral density
Measured: Baseline and 24 months
Bone Turnover Markers
Change in osteocalcin
Measured: Baseline and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2012 (estimated)
- Study startApr 2012
- Primary completionMay 2018
- Study completionFeb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis Related Bone DiseaseStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Boston Children's Hospital
- Responsible party
- Principal investigator, Melissa Susan Putman, Massachusetts General Hospital
- Investigators
- Joel Finkelstein, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01549314
- Sponsor's protocol ID
- MGH 2012P000269
- Status last verified
- Mar 2020
- First submitted
- 2 Mar 2012
- Last updated
- 1 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 22047557Accurso 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.
PubMed 21083385MacNeil 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.
PubMed 17229586Aris 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.
PubMed 15613415Shead 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.
PubMed 17600296Putman 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.
PubMed 33258950
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cystic Fibrosis Related Bone Disease: the Role of CFTR
- ClinicalTrials.gov ID
- NCT01549314
- Acronym / sponsor ID
- MGH 2012P000269
- Conditions
- Cystic Fibrosis Related Bone Disease
- Keywords
- Cystic fibrosis related bone disease, Cystic fibrosis, Ivacaftor
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Boston Children's Hospital
- Study start
- Apr 2012 (actual)
- Primary completion
- May 2018 (actual)
- Study completion
- Feb 2020 (actual)
- First posted
- 9 Mar 2012 (estimated)
- Results
- Posted 25 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether ivacaftor, a recently FDA-approved CFTR potentiator, improves bone micro-architecture and strength in patients with cystic fibrosis with at least one G551D CFTR mutation.
Detailed description
Ivacaftor, a CFTR potentiator, has recently been FDA approved for the treatment of cystic fibrosis in patients with at least one G551D CFTR mutation. Given the possible role of CFTR in bone, we hypothesize that this medication may also improve bone health in CF patients. The purpose of this study is to test this hypothesis using high resolution peripheral quantitative computed tomography, a research tool that measures bone micro-architecture and volumetric bone density and has the ability to detect small changes in bone that might otherwise be missed with standard bone imaging techniques such as bone density testing.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 6 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Subjects with CF will be recruited from the Cystic Fibrosis Centers at Children's Hospital Boston and Massachusetts General Hospital. Healthy subjects will be a community sample recruited from nearby communities.
Key inclusion criteria (7 listed)
- 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
- Age 6 to 75 years old
- Established diagnosis of CF
Key exclusion criteria (20 listed)
- Psychiatric or mental incapacity that would preclude subject from assenting to study participation
- Current pregnancy
- History of organ transplantation
- History of Burkholderia dolosa infection
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Bone Microarchitecture and Strength Measures of the Radius and Tibia
Time frame: Baseline and 24 months
Change in cortical volumetric bone mineral density at the radius
2 secondary outcomes
Areal Bone Mineral Density as Measured by DXA
Time frame: Baseline and 24 months
Bone Turnover Markers
Time frame: Baseline and 24 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 22047557Ramsey 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.
- Background: PubMed 21083385Accurso 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.
- Background: PubMed 17229586MacNeil 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.
- Background: PubMed 15613415Aris 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.
- Background: PubMed 17600296Shead 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.
- Derived from this study: PubMed 33258950Putman 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joel Finkelstein, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis Related Bone Disease, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bone Microarchitecture and Strength Measures of the Radius and Tibia (time frame: Baseline and 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01549314, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2020.