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Trial registered on ANZCTR


Registration number
ACTRN12611000104954
Ethics application status
Approved
Date submitted
30/01/2011
Date registered
31/01/2011
Date last updated
18/02/2014
Type of registration
Prospectively registered

Titles & IDs
Public title
Combination Gefitinib and Methotrexate for the Treatment of Persistent Gestational Trophoblastic Disease: A Phase I Clinical Trial
Scientific title
Combination Gefitinib and Methotrexate for the Treatment of Persistent Gestational Trophoblastic Disease: A Phase I Clinical Trial
Secondary ID [1] 253503 0
None
Universal Trial Number (UTN)
Trial acronym
GEM GTD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Persistent Gestational Trophoblastic Disease 261053 0
Condition category
Condition code
Reproductive Health and Childbirth 259197 259197 0 0
Other reproductive health and childbirth disorders
Cancer 259202 259202 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Standard 14-day methotrexate chemotherapy cycle (1mg/kg IM methotrexate on days 1, 3, 5, and 7 with alternate day rescue folinic acid 5mg PO on days 2, 4, 6 and 8) with addition of oral 250mg gefitinib in the following dose escalation protocol: the first 3 participants will take gefitinib on day 1 of chemotherapy only. If this is well tolerated, the next 3 participants will take gefitinib on days 1, 2 and 3 of chemotherapy, and the last 6 participants will take it on days 1-7. Concomitant gefitinib treatment will only occur in the first methotrexate chemotherapy cycle.
Intervention code [1] 257936 0
Treatment: Drugs
Comparator / control treatment
There is no control group in the study design as this is a phase IB toxicity trial. Data obtained from this study will be compared to standard methotrexate/leucovorin treatment outcomes for patients treated but not recruited during the conduct of the study, as logged in the RWH GTD registry.
Control group
Active

Outcomes
Primary outcome [1] 262045 0
Treatment of persistent gestational trophoblastic tissue as evidenced by a falling serum beta human chorionic gonadotropin level, a very sensitive biomarker of viable trophoblastic tissue.
Timepoint [1] 262045 0
Day 5 and 12.
Secondary outcome [1] 273025 0
Safety and tolerability of combination treatment to patients.
Subjectively - self-reporting of any side-effects. Main side effects of methotrexate: nausea, vomiting, bloating, abdominal pain, lethargy, mouth ulcers. Main side effects of gefitinib: diarrhoea, rash.
Objectively - history taking (systems review) and clinical examination esp. respiratory, abdominal and dermatological examination, as well as review of blood tests as markers for organ/systems function.
Timepoint [1] 273025 0
Second-daily patient review to day 7 and on day 15, and biochemical markers on days 5 and 12.

Eligibility
Key inclusion criteria
Persistent gestational trophoblastic disease diagnosed on tissue histopathology and confirmed by persistence of serum beta human chorionic gonadotropin levels.
Minimum age
18 Years
Maximum age
45 Years
Sex
Females
Can healthy volunteers participate?
No
Key exclusion criteria
Chronic pulmonary disease (excluding mild asthma), severe gastrointesitinal or dermatological disease, Japanese ethinicity, allergy to methotrexate or gefitinib.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Potential participants will be identified at a highly specialised clinic at the Royal Women's Hospital, where the state registry of gestational trophoblastic disease patients is kept. They will be allocated to treatment length based on order of recruitment i.e. the first three women recruited will receive 1 day of 250mg oral gefitinib, the next three women (4-6) 3 days of treatment, and the final six women recruited (7-12) will receive the full week's course of once daily oral 250mg gefitinib.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Order of recruitment.
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Dose-escalation protocol; based on order of recruitment, participants will be allocated to increasing length of treatment with once-daily gefitinib. Dose-escalation will occur after the first three participants are treated, then again after the next three participants are treated, provided the previous dose has been well-tolerated. Doses will start at one tablet of 250mg gefitinib orally on day 1, then three days of once daily oral gefitinib 250mg and in the final six women recruited, 7 days of once daily oral gefitinib 250mg.
Phase
Phase 1
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Date of first participant enrolment
Anticipated
15/09/2011
Actual
25/11/2011
Date of last participant enrolment
Anticipated
30/07/2013
Actual
29/10/2012
Date of last data collection
Anticipated
Actual
Sample size
Target
12
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 258404 0
University
Name [1] 258404 0
Monash Institute of Medical Research Flagship Grant, Monash University
Country [1] 258404 0
Australia
Primary sponsor type
Individual
Name
Miss Orla McNally, Director Gynaecological Oncology & Dysplasia
Address
The Royal Women's Hospital, Melbourne
Cnr. Grattan Street and Flemington Road,
Parkville Victoria 3052.
Country
Australia
Secondary sponsor category [1] 257552 0
Individual
Name [1] 257552 0
Associate Professor Stephen Tong
Address [1] 257552 0
Department of Obstetrics and Gynaecology,
Level 4, Mercy Hospital for Women
163 Studley Road
Heidelberg Victoria 3084
Country [1] 257552 0
Australia
Other collaborator category [1] 251785 0
Individual
Name [1] 251785 0
Dr Monika Skubisz
Address [1] 251785 0
C/O Department of Obstetrics & Gynaecology
Level 5
Monash Medical Centre
246 Clayton Road
Clayton Victoria 3168
Country [1] 251785 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 260377 0
The Royal Women's Hospital Human Research Ethics Committee
Ethics committee address [1] 260377 0
Ethics committee country [1] 260377 0
Australia
Date submitted for ethics approval [1] 260377 0
19/01/2011
Approval date [1] 260377 0
Ethics approval number [1] 260377 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 32147 0
Miss Orla McNally
Address 32147 0
The Royal Women's Hospital, Melbourne
Cnr. Grattan Street and Flemington Road,
Parkville Victoria 3052.
Country 32147 0
Australia
Phone 32147 0
+61 3 83453562
Fax 32147 0
Email 32147 0
orla.mcnally@thewomens.org.au
Contact person for public queries
Name 15394 0
Dr Monika Skubisz
Address 15394 0
C/O Department of Obstetrics & Gynaecology
Level 5
Monash Medical Centre
246 Clayton Road
Clayton Victoria 3168
Country 15394 0
Australia
Phone 15394 0
+61 3 9594 5405
Fax 15394 0
+61 3 9594 5489
Email 15394 0
monika.skubisz@monash.edu
Contact person for scientific queries
Name 6322 0
Dr Monika Skubisz
Address 6322 0
C/O Department of Obstetrics & Gynaecology
Level 5
Monash Medical Centre
246 Clayton Road
Clayton Victoria 3168
Country 6322 0
Australia
Phone 6322 0
+61 3 9594 5405
Fax 6322 0
+61 3 9594 5489
Email 6322 0
monika.skubisz@monash.edu

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.