Trials / Unknown
UnknownNCT02484755
Targeted Therapy With Gefitinib in Patients With USP8-mutated Cushing's Disease
- Status
- Unknown
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 6 (estimated)
- Sponsor
- Huashan Hospital · Academic / Other
- Sex
- All
- Age
- 18 Years – 70 Years
- Healthy volunteers
- Not accepted
Summary
The USP8 gene and its downstream target, epidermal growth factor receptor (EGFR), is a potential therapeutic target of Cushing disease. The EGFR inhibitor, Gefitinib, has been shown to reduce the production of ACTH both in vitro and in vivo, especially in USP8-mutated corticotrophin adenomas. The investigators hypothesize that Gefitinib will suppress pituitary corticotroph tumor ACTH production and normalize urinary free cortisol levels in patients with USP8-mutated Cushing's disease. Gefitinib is an FDA approved drug used to treat non-small cell lung cancer. However, in this study, the drug will be used to treat corticotrophin adenoma.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Gefitinib | See Arm Description |
Timeline
- Start date
- 2015-06-01
- Primary completion
- 2015-09-01
- First posted
- 2015-06-30
- Last updated
- 2015-06-30
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT02484755. Inclusion in this directory is not an endorsement.