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Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations

NCT03624101 · Status: TERMINATED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-04-16

Study results available
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Summary

Based on previous clinical findings, the investigator hypothesize that ivacaftor will have synergistic effects with drugs that facilitate truncated but partially active W1282X CFTR protein processing (tezacaftor) in patients with W1282X CFTR. In the current study, the investigators propose to directly test the efficacy of tezacaftor/ivacaftor (TEZ/IVA) and Trikafta for W1282X CFTR therapy in the clinic in comparison to ivacaftor alone.

Conditions

Interventions

DRUG

Symdeko

CFTR modulator

DRUG

Ivacaftor and Symdeko

CFTR modulator

Sponsors & Collaborators

  • University of Alabama at Birmingham

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-12-01
Primary Completion
2019-10-03
Completion
2019-10-03
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03624101 on ClinicalTrials.gov