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Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.

Sponsor
National Jewish Health (Other)
Overall Status
Completed
CT.gov ID
NCT04038710
Collaborator
(none)
8
Enrollment
1
Location
17.7
Actual Duration (Months)
0.5
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access. Pulmonary health, intestinal health, and the overall health of individuals will be tracked for a year to see how effective triple combination therapy is in these people with severe cystic fibrosis disease.

Condition or Disease Intervention/Treatment Phase
  • Drug: Triple combination therapy

Study Design

Study Type:
Observational
Actual Enrollment :
8 participants
Observational Model:
Case-Only
Time Perspective:
Prospective
Official Title:
An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.
Actual Study Start Date :
Sep 5, 2019
Actual Primary Completion Date :
Feb 26, 2021
Actual Study Completion Date :
Feb 26, 2021

Arms and Interventions

Arm Intervention/Treatment
Patients with severe disease

Patients that are eligible to enroll in Vertex's triple combination therapy through the expanded access program.

Drug: Triple combination therapy
Triple combination is compromised of the drugs ivacaftor, tezacaftor and Vertex's next generation modulator.

Outcome Measures

Primary Outcome Measures

  1. pulmonary function [For a year post initiation of therapy.]

    FEV1 values.

Secondary Outcome Measures

  1. CFQ-R score [For a year following the initiation of triple combination therapy.]

    cystic fibrosis questionnaire - revised has sections to measure pulmonary health and gastrointestinal health.

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  • Confirmed diagnosis of Cystic Fibrosis

  • Ability to reproducibly perform spirometry (according to ATS criteria)

  • Physician decision to treat with TCT through the EAP program

  • Ability to understand and sign a written informed consent or assent and comply with the requirements of the study

Exclusion Criteria:

  • History of hypersensitivity to VX 445, VX 659, tezacaftor and/or ivacaftor

  • Any acute lower respiratory symptoms treated with oral, inhaled or IV antibiotics or systemic corticosteroids within the 2 weeks prior to Visit 1.

  • Major or traumatic surgery within 12 weeks prior to Visit 1.

  • Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 1.

  • Use of an investigational agent within 28 days prior to Visit 1.

  • History of lung or liver transplantation or listing for organ transplantation.

Contacts and Locations

Locations

Site City State Country Postal Code
1 National Jewish Health Denver Colorado United States 80206

Sponsors and Collaborators

  • National Jewish Health

Investigators

  • Principal Investigator: Derek Low, MD, National Jewish Health

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
National Jewish Health
ClinicalTrials.gov Identifier:
NCT04038710
Other Study ID Numbers:
  • HS-3235
First Posted:
Jul 31, 2019
Last Update Posted:
Mar 11, 2021
Last Verified:
Mar 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis

Study Results

No Results Posted as of Mar 11, 2021