Safety, compliance with and activity of Bezafibrate and medroxyProgesterone acetate (BaP) as non-toxic therapy against myeloid and lymphoid cancers

ISRCTN	ISRCTN99131400
DOI	https://doi.org/10.1186/ISRCTN99131400
EudraCT/CTIS number	2011-001955-35
Secondary identifying numbers	RG_11-054

Submission date: 16/09/2011
Registration date: 25/10/2011
Last edited: 11/01/2024

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

http://cancerhelp.cancerresearchuk.org/trials/trial-looking-new-combination-drugs-some-types-leukaemia-lymphoma

Contact information

Prof Mark Drayson
Scientific

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

Study information

Study design	Phase II single arm four centre pilot study
Primary study design	Interventional
Secondary study design	Non randomised controlled trial
Study setting(s)	Hospital
Study type	Screening
Participant information sheet	Not available in web format, please use the contact details below to request a patient information sheet
Scientific title	Single arm phase II trial assessing the safety, compliance with and activity of Bezafibrate and medroxyProgesterone acetate (BaP) as non-toxic therapy against myeloid and lymphoid cancers
Study acronym	BaP
Study hypothesis	To test in patients with acute myeloblastic leukaemia (AML) or high risk myelodysplasia (RAEB2 WHO criteria), B cell Chronic Lymphocytic Leukaemia (CLL) and B cell Non Hodgkins Lymphoma (BNHL) the following outcomes of BaP administration over 18 weeks: 1. Safety 2. Compliance (feasibility of delivery) 3. Anti-cancer activity
Ethics approval(s)	NRES Committee East Midlands - Nottingham 2, 13/11/2012, ref: 11/EM/0426
Condition	Acute Myeloblastic Leukaemia or high risk myelodysplasia (RAEB2 WHO criteria) (AML), B cell Chronic Lymphocytic Leukaemia (CLL) and B cell Non Hodgkins Lymphoma (BNHL)
Intervention	All patients will receive BaP. BaP is Bezafibrate at 6 x 400 mg twice daily and medroxyProgesterone acetate at 5 x 200 mg daily. Patients will commence BaP at registration and continue for 18 weeks where the primary endpoint will be assessed. Patient may continue beyond 18 weeks at the discretion of the treating clinician.
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Phase II
Drug / device / biological / vaccine name(s)	Bezafibrate, Medroxyprogesterone acetate
Primary outcome measure	1. Safety: The number of grade 3 and 4 Adverse Reactions and Serious Adverse Reactions (SARs) attributable to the trial drugs 2. Patient compliance: Percentage of allocated treatment taken 3. Activity: 3.1. Haematological Response in the first 18 weeks of treatment 3.2. Clinical Response in the first 18 weeks of treatment
Secondary outcome measures	Quality of life questionnaires: 1. EQ-5D 2. EORTC QLQ-C30 Measured at baseline, between week 7-11 and at the final assessment at week 18
Overall study start date	01/12/2011
Overall study end date	10/09/2014

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	Both
Target number of participants	60
Participant inclusion criteria	1. Patients with one of the following diagnoses: 1.1. AML or high risk myelodysplasia (RAEB-2 WHO criteria) 1.2. CLL 1.3. BNHL 2. Be 18 years or older 3. Have given written informed consent For AML and MDS 1. Haemopoiesis must be impaired by the disease as judged by an abnormal full blood count (FBC) (International Working Group response criteria in myelodysplasia) and, where there is doubt as to the cause of impaired haemopoiesis, there must be bone marrow aspirate evidence that impaired haemopoiesis is due to cancer involvement of the bone marrow. 2. Abnormal values are haemoglobin level less than 11 g/dL or red blood cells (RBC) transfusion dependence, platelet count less than 100 x 109/L or platelet-transfusion dependence, absolute neutrophil count less than 1.0x 109/L. Pretreatment baseline measures of cytopenias are averages of at least two measurements (not influenced by transfusions, i.e. no RBC transfusions for at least 1 week and no platelet transfusions for at least 3 days) over at least 1 week prior to therapy. For CLL and BNHL 1. Patients must have either measurable disease (tumour cells in blood at >5 x 109/L, or lymphadenopathy > 1cm) or bone marrow failure due to disease as stated above for MDS / AML.
Participant exclusion criteria	1. Patient considered suitable for other forms of anti-cancer therapy (either accepted standard therapy or therapy in the context of a clinical trial) other than palliative corticosteroids or hydroxyurea 2. Estimated Glomerular Filtration Rate (eGFR) < 30ml/min 3. Patient known to be allergic to trial drugs 4. Patient has received treatment with any investigational medicinal product within the previous 28 days 5. Patient unable to swallow orally administered medications 6. Patient has uncontrolled seizures 7. Patient has active infection requiring systemic antibiotics, antifungal or antiviral drugs 8. Patient has concurrent severe and/or uncontrolled medical condition [e.g. severe chronic obstructive pulmonary disorder (COPD), severe Parkinsonss disease] or psychiatric condition 9. Women of child-bearing potential and men who have partners of child-bearing potential who are not willing to practise effective contraception for the duration of the study and for three months after the last study drug administration 10. Pregnant or lactating women. Women of child bearing potential must have a negative urine or serum pregnancy test within 7 days prior to registration.
Recruitment start date	01/10/2012
Recruitment end date	01/07/2014

Locations

Countries of recruitment

England
United Kingdom

Study participating centres

Queen Elizabeth Hospital

Stadium Rd
London
SE18 4QH
United Kingdom

Heartlands Hospital

Bordesley Green E
Birmingham
B9 5SS
United Kingdom

Good Hope Hospital

Rectory Rd
Sutton Coldfield
B75 7RR
United Kingdom

Worcestershire Royal Hospital

Charles Hastings Way
Worcester
WR5 1DD
United Kingdom

New Cross Hospital

Wednesfield Rd
WV10 0QP
WV10 0QP
United Kingdom

Sponsor information

University of Birmingham (UK)
University/education

Research Support Group
Institute of Research and Development
Birmingham Research Park
Vincent Drive
Edgbaston
Birmingham
B15 2SQ
England
United Kingdom

Website	http://www.birmingham.ac.uk/
"ROR"	https://ror.org/03angcq70

Funders

Funder type

Charity

Queen Elizabeth Hospital Charity (UK)

No information available

Results and Publications

Intention to publish date	30/06/2019
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	10/04/2019	10/12/2019	Yes	No
HRA research summary			28/06/2023	No	No
Plain English results			11/01/2024	No	Yes

Editorial Notes

11/01/2024: A link to plain English results was added.
10/12/2019: Publication reference added.
11/06/2018: Added intention to publish date.
07/06/2018: No publications found, verifying study status with scientific contact.
07/06/2018: EudraCTnumber added
11/05/2016: Internal review
06/05/2016: Verified study information with principal investigator. Changed overall study end date from 01/12/2013 to 10/09/2014. Chanrged recruitment date period from 01/12/2011-01/12/2013 to 01/10/2012-01/07/2014. Added trial participating centres and ethics approval information
26/04/2016: No publications found, verifying study status with principal investigator