C0221002 - The PODO Trial
Research type
Research Study
Full title
A PHASE 2, 12 WEEK, ADAPTIVE, OPEN LABEL, SEQUENTIAL COHORT TRIAL TO EVALUATE THE EFFICACY, SAFETY, TOLERABILITY AND PHARMACOKINETICS OF PF 06730512 FOLLOWING MULTIPLE DOSES IN ADULT SUBJECTS WITH FOCAL SEGMENTAL GLOMERULOSCLEROSIS (FSGS)
IRAS ID
238013
Contact name
Moin Saleem
Contact email
Sponsor organisation
Pfizer Inc.
Eudract number
2019-003607-35
Clinicaltrials.gov Identifier
133056, US IND
Duration of Study in the UK
2 years, 8 months, 5 days
Research summary
Focal Segmental Glomerulosclerosis (FSGS), a disease in which scar tissue develops on the parts of the kidneys that filter waste from the blood (glomeruli), which causes kidney failure.
The purpose of this study is to learn about effects, overall safety, how well the study drug (PF-06730512) is tolerated (tolerability), and to see how much drug is in blood (pharmacokinetics, PK), following intravenous administration every two weeks over 12 weeks, in eligible patients with Focal FSGS.
There will be 2 groups (Cohorts) on this study which will receive different dose levels of the study drug. There is NO placebo (Fake Drug) on this study, since it is a rare disease with a small sample size. One cohort will receive 1000 mg of study drug and the other 300 mg. The 300 mg dose can be adjusted, but will not exceed 1000 mg.
The study has 3 periods, a 8 Week lead in period, a 12 Week treatment period and a 9 week follow-up period. The study aims to enrol 44 patients across 72 different research sites. Each participant would spend about 8.5 months on the study and will need to visit the study site about 14 times, and there will also be about 5 telephonic visits. Patients will first need to pass screening, which may be split into two visits, to determine eligibility for this study.
8 week lead in period is observational only, followed by 12 week treatment period where study drug will be administered intravenously (In the vein). The follow-up period will be for 9 weeks. Study procedures such as physical examinations, blood and urine tests, blood pressure, pulse rate and temperature will be completed during all the study periods. About 25 Table spoons (500 ml) of blood, per patient in total will be drawn over the 8.5 month, during this study.
Summary of Results
Study C0221002 is a research study in patients who were at least 18 years old with the condition called Focal segmental glomerulosclerosis (FSGS). The purpose of this study was to learn about the effects on kidney function (the amount of protein in the urine, eGFR), safety, how well the study drug (PF-06730512) is tolerated, and levels of the study drug in blood, following intravenous administration every two weeks over 12 or 24 weeks. Intravenous administration means that the study drug is infused into one of your veins on your arm. The primary goal of this study was to obtain an early sign that the drug works.This study was designed to investigate up to 3 doses of PF-06730512 in up to 3 cohorts, each consisting of up to approximately 22 participants.
A total of 47 participants were enrolled into the study; 23 participants were in Cohort 1 (1000 mg IV dosing group) and 24 participants were in Cohort 2 (300 mg IV dosing group). Optional Cohort 3 was not conducted as study was terminated. The study was terminated due to lack of efficacy at both tested doses on 5th December 2022. The decision to terminate the study is not related to a safety concern.
For the Cohort 1, the mean percentage change in protein in urine was -12.283% after 12 weeks of treatment. For the Cohort 2, the mean percentage change in protein in urine after 12 weeks of treatment was -0.045%
37 out of 47 (78.7%) participants in this study had at least 1 medical problem.
One (1) participant in Cohort 1 stopped the study intervention due to medical problem, and 5◦participants in Cohort 2 stopped the study intervention due to study termination. None of participants in Cohort 2 stopped the study intervention due to medical problem.The most frequently reported medical problems in ≥5% of participant in the study were feeling tired and headache (10.6% each), COVID positive and COVID infection and loose stools (8.5% each), and vomiting and acute kidney injury (6.4% each).
4 out of 47 participants - 2 participants in in Cohort 1 (1 kidney failure and fluid overload, 1 acute kidney injury and loose stools) and 2 participants in Cohort 2 (1 COVID-infection, 1 FSGS disease progression) had serious medical problems (i.e. life-threatening, needs hospital care, or causes lasting problems). All serious medical problems were not related to the study medication
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
20/NW/0181
Date of REC Opinion
7 May 2020
REC opinion
Further Information Favourable Opinion