Condition category
Cancer
Date applied
03/07/2008
Date assigned
25/07/2008
Last edited
28/09/2011
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Mark Drayson

ORCID ID

Contact details

Clinical Immunology and Division of Immunity and Infection
The Medical School
University of Birmingham
Edgbaston
Birmingham
B15 2TT
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

N/A

Study information

Scientific title

The use of Bezfibrate and medroxyProgesterone acetate in Acute Myeloid Leukaemia and refractory anaemia with excess of blasts (RAEB) type 2: a phase II non-randomised trial

Acronym

BaP in AML

Study hypothesis

That patients with acute myeloblastic leukaemia (AML) who would not otherwise receive anti-leukaemia therapy will respond to therapy with bezafibrate and medroxyprogesterone acetate.

Ethics approval

Ethics approval received from:
1. The South Birmingham Research Ethics Committee on the 9th April 2003 (ref: 5355)
2. University Hospitals Coventry and Warwickshire Research and Development Department on the 7th July 2004 (ref: NJ02/0304/EU)
3. The Research Ethics Committee of Glasgow Royal Infirmary on the 24th July 2003 (ref: 03HA010)

Study design

Interventional multicentre non-randomised phase II study

Primary study design

Interventional

Secondary study design

Non randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

Elderly and relapsed high risk acute myeloid leukaemia

Intervention

1. Bezafibrate (Bezalip-Mono) 400 mg daily
2. Medroxyprogesterone acetate (Provera) 200 mg twice daily

Patients will also be given a prophylactic vitamin supplement so that they are not deficient in vitamins A and D, multivitamin tablet containing minimum vitamin A 4000 units and vitamin D 400 units.

Treatment was for 18 weeks. All patients were followed up to death (range of follow up was 8 days to 102 weeks from trial entry); one patient is still alive.

Intervention type

Drug

Phase

Phase II

Drug names

Bezafibrate, medroxyprogesterone acetate

Primary outcome measures

Tumour response as measured by:
1. Full blood count
2. Transfusion dependency (frequency of red blood cells/platelet transfusions)
3. Percentage blasts in bone marrow and peripheral blood pre and post BaP therapy
4. Bone marrow morphology as determined by blood smears

Response will be assessed using Southwest Oncology Group (SWOG) criteria as modified from National Cancer Institute (NCI-) sponsored workshop guidelines.
1. Complete response (CR): less than 5% blasts in a marrow of sufficient cellularity with a peripheral neutrophil count greater than 1 x 10^9/l and platelet count of greater than 100 x 10^9/l determined by two evaluations not less than 4 weeks apart
2. Partial response (PR): as determined by two evaluations not less than 4 weeks apart:
2.1. In RAEB type 2 bone marrow should show greater than 50% decrease in myeloblasts, but not necessarily disappearance of marrow dyspoiesis. In peripheral blood, greater than 50% reduction in deficit from minimum normal levels (UHB haematology reference range) of the haemoglobin, neutrophil and platelet counts (if abnormal at baseline) with an absence of myeloblasts in the peripheral blood.
2.2. In AML bone marrow should show less than 15% myeloblasts with a decrease but not necessarily a disappearance of marrow dyspoiesis with an absence of Auer rods. Plus in peripheral blood there should be a greater than 50% reduction in deficit from minimum normal levels (UHB haematology reference range) of haemoglobin, neutrophil and platelet counts (if abnormal at baseline) with absence of myeloblasts in the peripheral blood.
3. Minor response (MR): decrease in frequency of infections or bleeding episodes and a 50% decrease in transfusion requirements, decrease of marrow dyspoiesis and improvement in peripheral counts but not enough to qualify for PR or CR nor progressive disease can be established
4. No change: neither the criteria for CR, PR, MR nor progressive disease can be established
5. Progressive disease: evidence of increased blasts in bone marrow or peripheral blood

Secondary outcome measures

No secondary outcome measures

Overall trial start date

01/06/2003

Overall trial end date

01/04/2006

Reason abandoned

Eligibility

Participant inclusion criteria

1. Patient has acute myeloid leukaemia (this can be any type of de novo or secondary AML, except acute promyelocytic leukaemia), or
2. Patient has refractory anaemia with an excess of blasts (greater than 10%) RAEB type 2 World Health Organization (WHO) criteria
3. Adult patients, either sex

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

20

Participant exclusion criteria

1. Patient has acute promyelocytic leukaemia
2. Intensive chemotherapy is considered a suitable option
3. Low dose cytotoxic chemotherapy is likely to be required to control a rising blast cell count in the next month
4. Patient has a concurrent active malignancy
5. Patient has uncontrolled systemic disease (e.g. hypertension, diabetes) or severe cardiovascular disease
6. Patient is pregnant or lactating, or are potentially fertile (both males and females) and have not agreed to take adequate contraceptive precautions during the trial
7. Patient aged under 18 years

Recruitment start date

01/06/2003

Recruitment end date

01/04/2006

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Clinical Immunology and Division of Immunity and Infection,
Birmingham
B15 2TT
United Kingdom

Sponsor information

Organisation

University Hospital Birmingham NHS Foundation Trust (UK)

Sponsor details

Trust Headquarters
PO Box 9551
Main Drive
Queen Elizabeth Medical Centre
Edgbaston
Birmingham
B15 2PR
United Kingdom

Sponsor type

Government

Website

http://www.uhb.nhs.uk/

Funders

Funder type

Government

Funder name

The University Hospital Birmingham NHS Foundation Trust (UK) - paying incidental costs

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

2010 results in http://www.ncbi.nlm.nih.gov/pubmed/20067564

Publication citations

  1. Results

    Murray JA, Khanim FL, Hayden RE, Craddock CF, Holyoake TL, Jackson N, Lumley M, Bunce CM, Drayson MT, Combined bezafibrate and medroxyprogesterone acetate have efficacy without haematological toxicity in elderly and relapsed acute myeloid leukaemia (AML)., Br. J. Haematol., 2010, 149, 1, 65-69, doi: 10.1111/j.1365-2141.2009.08055.x.

Additional files

Editorial Notes