NCT02934698

Brief Summary

This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
2

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Nov 2016

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

October 13, 2016

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 17, 2016

Completed
15 days until next milestone

Study Start

First participant enrolled

November 1, 2016

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2018

Completed
3 months until next milestone

Results Posted

Study results publicly available

August 6, 2018

Completed
Last Updated

August 6, 2018

Status Verified

July 1, 2018

Enrollment Period

1.5 years

First QC Date

October 13, 2016

Results QC Date

July 10, 2018

Last Update Submit

July 10, 2018

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Forced Expiratory Volume

    Absolute change in percent predicted in 1 second FEV1 from baseline through week 24

    24 weeks

Secondary Outcomes (2)

  • Sputum Results

    24 weeks

  • Sweat Chloride

    24 Weeks

Study Arms (1)

Ivacaftor

EXPERIMENTAL

There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment.

Drug: Ivacaftor

Interventions

IvacaftorDRUG

Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.

Ivacaftor

Eligibility Criteria

Age18 Years+
Sexfemale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects are \>18 years of age and able to provide informed consent.
  • Subjects reside in the US and are willing to be treated with ivacaftor.
  • Subjects have the splicing mutation of interest.
  • Subjects are willing and able to perform requirements of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

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

Results Point of Contact

Title
Patrick Flume
Organization
MUSC
flumepa@musc.edu
843-792-3167

Study Officials

  • Patrick A Flume, MD

    Medical University of South Carolina

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 13, 2016

First Posted

October 17, 2016

Study Start

November 1, 2016

Primary Completion

May 1, 2018

Study Completion

May 1, 2018

Last Updated

August 6, 2018

Results First Posted

August 6, 2018

Record last verified: 2018-07

Data Sharing

IPD Sharing
Will not share