Condition category
Urological and Genital Diseases
Date applied
Date assigned
Last edited
Prospectively registered
Overall trial status
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information



Primary contact

Mrs Elizabeth Brettell


Contact details

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

Additional identifiers

EudraCT number

2009-016003-26 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



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 approved on the 4th of May 2010 (ref: 10/S0703/27)

Study design

National multicentre randomised controlled open label pilot trial

Primary study design


Secondary study design

Randomised controlled trial

Trial setting


Trial type


Patient information sheet

Will be available at:


Renal; primary proteinuric glomerulonephritis


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



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


Overall trial end date


Reason abandoned


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


Age group




Target number of participants


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


Recruitment end date



Countries of recruitment

United Kingdom

Trial participating centre

Birmingham Clinical Trials Unit
B15 2TT
United Kingdom

Sponsor information


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
B15 2TT
United Kingdom

Sponsor type

Not defined



Funder type


Funder name

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

Alternative name(s)

Funding Body Type

Funding Body Subtype


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

Publication citations

Additional files

Editorial Notes