Targeted Therapy With Gefitinib in Patients With USP8-mutated Cushing's Disease
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|ClinicalTrials.gov Identifier: NCT02484755|
Recruitment Status : Unknown
Verified June 2015 by Zhaoyun Zhang, Huashan Hospital.
Recruitment status was: Recruiting
First Posted : June 30, 2015
Last Update Posted : June 30, 2015
|Condition or disease||Intervention/treatment||Phase|
|Cushing's Disease Corticotrophin Adenoma||Drug: Gefitinib||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Targeted Therapy With Gefitinib in Patients With USP8-mutated Cushing's Disease|
|Study Start Date :||June 2015|
|Estimated Primary Completion Date :||September 2015|
gifitinib 250 mg oral administration once daily for a total of 4 weeks.
See Arm Description
- Change in levels of 24 hour urinary free cortisol after 4 weeks [ Time Frame: Baseline, Week 4 ]Full response is defined as the reduction of 24 hour urinary free cortisol more than 50% or normalized. Partial response is defined as the reduction of 24 hour urinary free cortisol between 25%~ 50%. Poor response or resistance is defined as the reduction of 24 hour urinary free cortisol less than 25%.
- Number of participants with a normalized 24 hour urinary free cortisol after 4 weeks [ Time Frame: 4 weeks ]
- Change in levels of Late-Night Salivary Cortisol after 4 weeks [ Time Frame: Baseline, Week 4 ]
- Change in levels of pituitary hormones （a composite of pituitary panel) [ Time Frame: Baseline, Week 4 ]Pituitary Panel (TSH,FT4, FT3, PRL, IGF-1, FSH, Testosterone Total, Free Testosterone, Estradial, SHBG)
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 4 weeks ]
- Changes in tumor size [ Time Frame: Baseline, Week 4 ]
- Changes in clinical signs (the typical signs of Cushing's Disease such as facial rubor, hirsutism, striae, bruising and supraclavicular and dorsal fat pad) [ Time Frame: Baseline, Week 4 ]
The following symptoms will be evaluated using photographs taken at baseline and at the end of the study (Week 4):
facial rubor, hirsutism, striae, bruising and supraclavicular and dorsal fat pad. The photographs will be assessed (scored) by the investigator as well as a second qualified physician at the site, who is blinded to the timepoint at which the photographs were taken.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02484755
|Contact: Ming Shen, MD||+86 firstname.lastname@example.org|
|Shanghai, Shanghai, China, 200040|
|Contact: Ming Shen, MD +86 13818795785 email@example.com|
|Principal Investigator: Yao Zhao, MD|
|Principal Investigator:||Yao Zhao, MD||Department of Neurosurgery, Huashan Hospital, Fudan University|