Condition category
Urological and Genital Diseases
Date applied
19/04/2010
Date assigned
27/05/2010
Last edited
21/11/2016
Prospective/Retrospective
Prospectively registered
Overall trial status
Stopped
Recruitment status
Stopped

Plain English Summary

Not provided at time of registration

Trial website

http://www.bctu.bham.ac.uk/glomy

Contact information

Type

Scientific

Primary contact

Mrs Elizabeth Brettell

ORCID ID

Contact details

Birmingham Clinical Trials Unit
School of Cancer Sciences
College of Medical and Dental Sciences
Robert Aitken Institute
University of Birmingham
Edgbaston
Birmingham
B15 2TT
United Kingdom

Additional identifiers

EudraCT number

2009-016003-26

ClinicalTrials.gov number

Protocol/serial number

V2, EuDRACT No: 2009-016003-26

Study information

Scientific title

Randomised pilot trial of Myfortic® for the treatment of primary proteinuric glomerulonephritis

Acronym

GloMY

Study hypothesis

To determine the feasibility of running a full-scale phase III randomised trial of Myfortic ®plus short course steroids versus standard care in patients with Focal Segmental Glomerulosclerosis (FSGS) or Immunoglobulin A Nephropathy (IgAN). The trial will also provide preliminary comparative data on the efficacy of Myfortic® plus short course steroids in inducing sustained response (partial or complete) in a well-defined cohort of patients with primary proteinuric glomerulonephritis (FSGS and IgAN) that will inform the sample size required to design a large prospective randomised study investigating the effect of Myfortic®.

Ethics approval

West of Scotland Ethics Committee 1, 04/05/2010, ref: 10/S0703/27

Study design

National multicentre randomised controlled open label pilot trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Will be available at: http://www.bctu.bham.ac.uk/glomy

Condition

Renal; primary proteinuric glomerulonephritis

Intervention

Intervention group for both FSGS and IgAN patients (Myfortic® and short course of steroids):
Myfortic® 720mg b.d. continued for 2 years, along with prednisolone, starting at dose of 1mg/kg (up to a maximum of 60mg) tapered to 0mg by 10 weeks.

Standard care for FSGS patients (High-dose steroids - prednisolone):
Prednisolone, starting at dose of 1mg/kg (up to a maximum of 60mg) until complete remission or a maximum of 6 months treatment. If complete remission is achieved, prednisolone will be tapered to 0mg over the following 10 weeks. In those achieving partial remission, prednisolone will be continued for a further month, and then tapered to 5mg over 8 weeks, and then maintained at 5mg until 2 years.

Standard care for IgAN patients: No treatment.

All patients receive 2yrs treatment and are followed up until the end of the trial which is at 4yrs. So all patients will be followed up for at least two years after date of randomisation. So for patients who enter at the trial start they will have 2yrs treatment and a further 2 yrs follow-up, and patients entering at the end of the 2yr recruitment period will just have 2rys of treatment and follow-up.

Intervention type

Drug

Phase

Phase III

Drug names

Mycophenolic acid (Myfortic®), prednisolone

Primary outcome measures

Proportion of patients achieving complete or partial remission by 24 weeks sustained (relapse free) for 12 months.

Secondary outcome measures

1. Proportion achieving partial remission
2. Time to partial remission
3. Proportion achieving complete remission
4. Time to complete remission
5. Time to relapse
6. Proportion of patients requiring alternative cytotoxic agent or treatment failure and renal function (estimated Glomerular Filtration Rate, proteinuria)
7. Treatment safety:
7.1. Cumulative dose of corticosteroids
7.2. Number of patients developing steroid induced diabetes
7.3. Number of patients having serious infections
7.4. Adverse events
7.5. Markers of bone turnover
7.5.1. procollagen type 1 aminoterminal propeptide (P1NP, a marker of bone formation)
7.5.2. β-C-terminal telopeptides of type 1 collagen (β-CTx, a marker of bone resorption)

Data will be collected at baseline, at weeks 2 and 4, and then every 4 weeks out to 6 months post-randomisation, and then every 12 weeks for at least 2 years.
This study will also enable the study forms to be piloted.

Overall trial start date

01/06/2010

Overall trial end date

02/10/2012

Reason abandoned

Participant recruitment issue

Eligibility

Participant inclusion criteria

1. Patients with new onset biopsy proven (within last year) primary FSGS with albumin <30g/dl OR patients with primary IgAN with biopsy findings E1 and T<2 using the Oxford classification and a minimum of 8 glomeruli in the biopsy
2. Proteinuria (Protein Creatinine Ratio, PCR>150) following at least 4 weeks treatment with maximal blood pressure lowering therapy (to include angiotensin blockade) to target blood pressure <125/75 mmHg
3. If female and of childbearing potential, must not be pregnant or breastfeeding, and agree to avoid pregnancy during and for 6 weeks following the last dose of study treatment
4. If male with a partner of childbearing potential, must agree to use adequate, medically approved, contraceptive precautions during and for 6 weeks following the last dose of study treatment.

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

100

Participant exclusion criteria

1. Age <18 years
2. Secondary causes of FSGS
3. Secondary IgAN
4. Deteriorating renal function >20μmol/l each week for 3 weeks or more
5. Estimated Glomerular Filtration Rate (eGFR) <20 ml/min (using modification of diet in renal disease [MDRD] equation)
6. Poor blood pressure control (e.g. blood pressure ≥140/80 mmHg)
7. Previous treatment with immunosuppression therapies
8. Unable to receive immunosuppression treatments due to malignancy or active infection
9. Patients with systemic infection unless specific anti-infective therapy is employed
10. Diabetes
11. Known to have hepatitis B or C
12. Known to be HIV positive
13. Inability to give informed consent

Recruitment start date

21/10/2010

Recruitment end date

12/08/2012

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Birmingham Clinical Trials Unit
Birmingham
B15 2TT
United Kingdom

Sponsor information

Organisation

University Hospitals Birmingham NHS Foundation Trust (UK)

Sponsor details

Dr. Chris Counsell
Research & Development Manager
Research & Development Office
HSRC Building
College of Medical and Dental Sciences
West Campus
University of Birmingham
Edgbaston
Birmingham
B15 2TT
United Kingdom

Sponsor type

Not defined

Website

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

Funders

Funder type

Industry

Funder name

Novartis (UK) - Educational Grant (ref: ERL080AGB09T)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

As the study showed that it was not feasible to run a full-scale phase III randomised trial of Myfortic plus short course steroids versus standard care in patients with FSGS or IgAN, and we obtained very limited data to assess the preliminary comparative efficacy of Myfortic plus short course steroids in inducing sustained response in patients with FSGS or IgAN, there are no plans for publication. The study outcome has been disseminated to all collaborators and the GloMY collaborators will all be sent a summary of this report. All participants were informed of the outcome and end of trial via a Research Ethics Committee approved letter via their clinician.

IPD Sharing plan:
The datasets generated during and/or analysed during the current study is not expected to be made available. As the study showed that it was not feasible to run a full-scale phase III randomised trial of Myfortic plus short course steroids versus standard care in patients with FSGS or IgAN, and we obtained very limited data to assess the preliminary comparative efficacy of Myfortic plus short course steroids in inducing sustained response in patients with FSGS or IgAN, there are no plans for publication.

Intention to publish date

Participant level data

Not expected to be available

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes

21/11/2016: Only 12 patients were recruited to this study. This was despite recruitment being open for 2 years, 26 sites participating and several protocol amendments to facilitate recruitment. At this rate of patient entry, the recruitment phase of the study would have needed to continue for a further 7-10 years. The primary aim of the study was to assess the feasibility of conducting an interventional trial in primary glomerulonephritis within the UK rather than to assess efficacy of Myfortic in the treatment of these diseases. Given this aim, the TSC did not think it was feasible to continue recruitment for such a prolonged period of time, hence the decision to close the trial. The study therefore showed that it was not feasible to run a full-scale phase III randomised trial of Myfortic plus short course steroids versus standard care in patients with FSGS or IgAN. The overall trial end date has been updated from 01/06/2014 to 02/10/2012 and the recruitment dates from 01/06/2010 - 01/06/2014 to 21/10/2010 - 21/08/2012. 18/11/2016: No publications found in PubMed, verifying study status with principal investigator.