A placebo-controlled, double-blind, multicentre phase III trial to assess the efficacy and safety of miltefosine solution in the treatment of breast cancer where no other appropriate treatment is available

ISRCTN	ISRCTN91258120
DOI	https://doi.org/10.1186/ISRCTN91258120
Protocol serial number	C121
Sponsor	Cancer Research UK (CRUK) (UK)
Funder	Cancer Research UK (CRUK) (UK)

Submission date: 19/08/2002
Registration date: 19/08/2002
Last edited: 04/01/2012

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr - -
Scientific

UKCCCR Register Co-ordinator
MRC Clinical Trials Unit
222 Euston Road
London
NW1 2DA
United Kingdom

Study information

Primary study design	Interventional
Study design	Multicentre randomised double blind placebo controlled trial
Secondary study design	Randomised controlled trial
Scientific title
Study objectives	Not provided at time of registration
Ethics approval(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	Breast cancer
Intervention	Patients are randomised to receive either: 1. Treatment A: Miltefosine, a 6% solution 2. Treatment B: Placebo solution The solution whether placebo or miltefosine is applied to the affected area initially once daily (two drops per 10 cm surface area, allowing for an approximately 3 cm margin around the visible lesion). Provided this has good tolerability the dose will be escalated to twice daily applications from week two onwards. In the absence of clear progression of skin lesions or dose-limiting adverse events a minimum treatment time of eight weeks is suggested. Patients who have a complete response should continue at the same dosage, if possible, for at least a further four weeks after the complete response is observed. Patients will be treated and/or followed-up until progression or occurrence of skin lesions within the treated area, treatment stop due to poor tolerability of the study medication, or necessity for a change in systemic therapy.
Intervention type	Drug
Phase	Phase III
Drug / device / biological / vaccine name(s)	Miltefosine
Primary outcome measure(s)	Added 06/08/09: Time to treatment failure
Key secondary outcome measure(s)	Added 06/08/09 1. Rate of response 2. Cutaneous reactions
Completion date	30/04/1998

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	Female
Key inclusion criteria	1. Female, aged more than 18 years 2. Histologically or cytologically confirmed breast cancer with inoperable lesions, unsuitable for radiotherapy, inadequately manageable by radiotherapy or systemic endocrine or chemotherapy 3. Superficial nodular or "flat" skin lesions including (estimated depth 1 cm), at least one bidimensionally measurable and progressive lesion 4. Patients should have had at least one prior systemic endocrine or chemotherapy. Patients may take concomitant endocrine therapy only (endocrine therapy must have been unchanged for the last 12 weeks if ongoing at the time of study entry) 5. Performance status World Health Organisation (WHO) grade two with life expectancy of at least three months 6. Satisfactory haematological and blood chemistry values
Key exclusion criteria	1. Patients with no measurable lesions, skin lesions with estimated depth over 1 cm, ulcerated skin lesions over 10% of the area to be treated or local infection within the treated area 2. Clinical evidence of brain metastases that would limit life expectancy to less than six months 3. Patients with progressive associated systemic metastases 4. Previous malignancies within the last five years, except treated and cured carcinoma in situ of the cervix, non-melanoma skin cancer or cutaneous lymphepithelioma 5. Radiotherapy to skin lesions or chemotherapy within the last four weeks 6. Major surgery within the last two weeks 7. Uncontrolled clinically significant illness not related to cancer The only permissible concomitant therapies are irradiation of non-skin lesions for symptom relief and endocrine therapy if it has remained unchanged for at least 12 weeks
Date of first enrolment	01/01/1998
Date of final enrolment	30/04/1998

Locations

Countries of recruitment

United Kingdom
England

Study participating centre

UKCCCR Register Co-ordinator

London
NW1 2DA
United Kingdom

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/11/2001		Yes	No