NCT01549314

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
79

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Apr 2012

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 2, 2012

Completed
7 days until next milestone

First Posted

Study publicly available on registry

March 9, 2012

Completed
23 days until next milestone

Study Start

First participant enrolled

April 1, 2012

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2018

Completed
1.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2020

Completed
2 months until next milestone

Results Posted

Study results publicly available

March 25, 2020

Completed
Last Updated

April 1, 2020

Status Verified

March 1, 2020

Enrollment Period

6.1 years

First QC Date

March 2, 2012

Results QC Date

February 25, 2020

Last Update Submit

March 24, 2020

Conditions

Cystic Fibrosis Related Bone Disease

Keywords

Cystic fibrosis related bone diseaseCystic fibrosisIvacaftor

Outcome Measures

Primary Outcomes (1)

  • Bone Microarchitecture and Strength Measures of the Radius and Tibia

    Change in cortical volumetric bone mineral density at the radius

    Baseline and 24 months

Secondary Outcomes (2)

  • Areal Bone Mineral Density as Measured by DXA

    Baseline and 24 months

  • Bone Turnover Markers

    Baseline and 24 months

Study Arms (3)

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.

Eligibility Criteria

Age6 Years - 75 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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.

You may qualify if:

  • 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

You may not qualify if:

  • 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.
  • Age 6 to 75 years old
  • Established diagnosis of CF
  • 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.
  • Age 6 to 75 years old
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Related Publications (6)

  • 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.

    PMID: 22047557BACKGROUND

  • 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.

    PMID: 21083385BACKGROUND

  • 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.

    PMID: 17229586BACKGROUND

  • 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.

    PMID: 15613415BACKGROUND

  • 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.

    PMID: 17600296BACKGROUND

  • 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.

    PMID: 33258950DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma and urine

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Limitations and Caveats

This was a prospective observational multiple cohort study and not a randomized or interventional clinical trial.

Results Point of Contact

Title
Dr. Melissa Putman
Organization
Massachusetts General Hospital Endocrine Unit
msputman@partners.org
857-218-5017

Study Officials

  • Joel Finkelstein, MD

    Massachusetts General Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

March 2, 2012

First Posted

March 9, 2012

Study Start

April 1, 2012

Primary Completion

May 1, 2018

Study Completion

February 1, 2020

Last Updated

April 1, 2020

Results First Posted

March 25, 2020

Record last verified: 2020-03

Locations

US(1)