TerminatedNCT03448692PODOUpdated Apr 18, 2024Results posted

A Study to Evaluate PF-06730512 in Adults With Focal Segmental Glomerulosclerosis (FSGS)

A Phase 2 interventional study of PF-06730512 in Focal Segmental Glomerulosclerosis (FSGS), sponsored by Pfizer. Terminated at 110 sites in 12 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-04-18.

Sponsored by Pfizer · Phase 2, Interventional, and Treatment

Why this 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.

Phase: Phase 2
Study type: Interventional
Enrollment: 47
Allocation: Non-randomized
Ages: 18 Years and older
Sex: All

Study summary

The purpose of this Phase 2 adaptive study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of PF-06730512 following multiple intravenous infusions in adult subjects with FSGS.

Conditions studied

Focal Segmental Glomerulosclerosis (FSGS)

Browse trials for

Glomerulosclerosis, Focal Segmental 97

Keywords

Proteinuria

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Adults age 18 years and older who have a confirmed biopsy diagnosis of FSGS.
Estimated glomerular filtration rate (eGFR) greater than or equal to 45 ml/min/1.73 m2. If eGFR is 30 - 45 ml/min/1.73 m2, a recent biopsy (within 12 months prior to Screening) must demonstrate \< 50% tubulointerstitial fibrosis.
Urine protein:creatinine ratio (UPCR) greater than 1.5 g/g at screening.
Treated with at least one but not more than 3 classes of immunosuppressants either alone or in combination, or has a contraindication to use of an immunosuppressant or is intolerant to an immunosuppressant per investigator judgment.

Exclusion criteria

Exclusion Criteria:

Diagnosis of collapsing FSGS.
Advanced chronic changes on renal biopsy as evidenced by greater than 50% tubulointerstitial fibrosis.
Organ transplant.
History of malignancy, with the exception of basal or squamous cell carcinoma that has been treated and fully resolved for a minimum of 5 years.
Body mass index (BMI) greater than 45 kg/m2.
Subjects with a history of prior treatment with or use of interferon, lithium, pamidronate, mTOR inhibitors (eg, sirolimus), testosterone/anabolic steroids, anthracycline (eg, doxorubicin), heroin.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Sequential assignment
Masking: Triple (Participant, Care provider, Investigator)
Enrollment: 47 participants (actual)

Study arms

Experimental
PF-06730512 Cohort 1
Subjects in cohort 1 will receive dose 1 Intravenous (IV) infusion.
Drug: PF-06730512
Experimental
PF-06730512 Cohort 2
Subjects in cohort 2 will receive dose 2 IV infusion.
Drug: PF-06730512
Experimental
PF-06730512 Cohort 3 (optional)
Subjects in cohort 3 will receive dose 3 IV infusion.
Drug: PF-06730512

Interventions

DrugPF-06730512
Subjects in cohort 1 will receive Dose 1 Intravenous infusion (IV). Subjects in cohort 2 will receive Dose 2 IV. Subjects in cohort 3 will receive Dose 3 IV infusion.

What researchers measure

Primary outcomes

Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) Based on 24-hour Urine Collection at Week 13
UPCR is a ratio between two measured substances in urine: milligram of protein per millimole (mmol) of creatinine, reported in units mg/mmol. A decrease in UPCR may be associated with improved renal and cardiovascular function.
Time frame: Baseline, Week 13

Secondary outcomes

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event was considered treatment emergent relative to a given treatment if the event occurred for the first time during the investigational treatment period and was not seen prior to the start of treatment (during the lead-in period), or the event was seen prior to the start of treatment but increased in severity during treatment. Adverse events occurring during the lead-in period were considered non-treatment emergent. Events that occurred during the follow-Up period were counted as treatment emergent and attributed to the previous treatment taken.
Time frame: From Day 1 of treatment up to 9 weeks after last dose of study treatment (up to Week 33)
Number of Participants With Abnormalities in Laboratory Test Parameters
Hemoglobin (Hg), hematocrit, erythrocytes: \<0.8\*lower limits of normal (LLN); platelets: \<0.5\*LLN\>1.75\*upper limits of normal(ULN); leukocytes (leu), glucose-fasting:\<0.6\*LLN\>1.5\*ULN; lymphocytes (lym), lym/leu, neutrophils(neu),neu/leu, protein, albumin, phosphate, free thyroxine, thyroid stimulating hormone: \<0.8\*LLN\>1.2\*ULN; basophils (bas), bas/leu, eosinophils (eos), eos/leu, monocytes(mon), mon/leu: \>1.2\*ULN; bilirubin (total, direct, indirect):\>1.5\*ULN; aspartate aminotransferase(AT), alanine AT, lactate dehydrogenase, alkaline phosphatase:\>3.0\*ULN; blood urea nitrogen, creatinine, cholesterol (total,LDL,HDL),triglycerides, Hg A1C: \>1.3\*ULN; sodium: \<0.95\*LLN\>1.05\*ULN; potassium, chloride, calcium, magnesium, bicarbonate: \<0.9\*LLLN\>1.1\*ULN; prolactin: \>1.1\*ULN; creatine kinase: \>2.0\*ULN; urobilinogen: \>=1; Urine-specific gravity: \<1.003\>1.030, pH: \<4.5 \>8, glucose,protein,bilirubin,nitrite,leukocyte esterase, ketones: \>=1.Categories with at-least 1 non-zero values are reported.
Time frame: From Day 1 of treatment up to Week 33
Change From Baseline in Body Weight
Change from baseline in body weight and at baseline values were reported for this outcome measure.
Time frame: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Change From Baseline in Blood Pressure
Change from baseline in blood pressure and at baseline values were reported for this outcome measure.
Time frame: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Change From Baseline in Pulse Rate
Change from baseline in pulse rate and at baseline values were reported for this outcome measure.
Time frame: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Change From Baseline in Body Temperature
Change from baseline in body temperature and at baseline values were reported for this outcome measure.
Time frame: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Number of Participants With Abnormalities in Electrocardiogram (ECG)
ECG abnormalities criteria included: 1) QTc interval adjusted according to Bazett formula (QTcB) in millisecond (msec): greater than (\>) 450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; 2) QTc interval adjusted according to Fridericia formula (QTcF) (msec): \>450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; 3) Heart rate (bpm): RR decrease \>25% and to a VR (interval between QRS wave and T wave on ECG) \>100; RR (interval between 2 successive R waves on ECG) increase \>25% and to a VR \<50; 4) Pulse rate (msec): increase \>25% and to a value \>200; 5) QT (msec): \>450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; 6) QRS (msec): increase \>25% and to a value \>110. Categories (timepoints) with at least 1 participant having ECG abnormality in any of the reporting arms, were reported for this outcome measure.
Time frame: Weeks 3, 7, 11, 13, 17, 21, 25, 33
Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) at Weeks 2, 5, 9 and 13
UPCR is a ratio between two measured substances in urine: mmol of creatinine, reported in units mg/mmol. A decrease in UPCR may be associated with improved renal and cardiovascular function.
Time frame: Baseline, Weeks 2, 5, 9 and 13
Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR ) at Weeks 3, 5, 9 and 13
The eGFR was calculated using 4 variable formula developed by the modification of diet in renal disease (MDRD) study group. The 4 variables needed to estimate glomerular filtration rate (GFR) using this formula were serum creatinine concentration, age, sex (for females, eGFR was multiplied by 0.742) and ethnic origin (for African-Caribbean people only, eGFR was multiplied by 1.212). Thus eGFR in milliliter per minute per 1.73 square meter (mL/min/1.73 m\^2) = 175\*(sCr/88.4)\^-1.154\*(Age)\^-0.203\*(0.742 if female)\*(1.212 if African-Caribbean). Baseline eGFR was determined predose at Week 0 (Day 1). For Baseline eGFR, the "Low eGFR" group was defined as baseline eGFR \< 45 mL/min/1.73m2, and the "High eGFR" group was defined as baseline eGFR \> 45 mL/min/1.73 m2.
Time frame: Baseline, Weeks 3, 5, 9 and 13
Serum PF-06730512 Concentration Versus Time Summary
Time frame: For 12-Week treatment(WT):pre-dose on Day1,8,15,29,43,57,71,follow-up(Fup)visit on Day85,99,113,141,For 24-WT:pre-dose on Day1,8,15,29,43,57,71,85,99,113,127,141,155,Fup visit on Day169,183,197,225;1hour post-dose on Day1,71,155(only applicable for 24-WT)
Number of Participants With Positive Anti-Drug Antibody (ADA) and Neutralizing Antibody(NAb)
Number of participants with positive ADA and/or NAb were reported for this outcome measure.
Time frame: From Day 1 of treatment up to Week 33

Results

Posted Apr 18, 2024

Participant flow

Disposition Phase:Screening:up to 43Days

Participant flow — Disposition Phase:Screening:up to 43Days
Milestone	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Started	23	24
Completed	23	24
Not completed	0	0

Lead in Period:8 Weeks (W)

Participant flow — Lead in Period:8 Weeks (W)
Milestone	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Started	23	24
Completed	23	24
Not completed	0	0

Investigational Treatment Period:Upto24W

Participant flow — Investigational Treatment Period:Upto24W
Milestone	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Started	23	24
Completed	22	15
Not completed	1	9
Withdrew: Adverse event	1	0
Withdrew: Lack of efficacy	0	1
Withdrew: Physician's decision	0	1
Withdrew: Study terminated by sponsor	0	5
Withdrew: Withdrawal by subject	0	2

Follow up Period:9 Weeks

Participant flow — Follow up Period:9 Weeks
Milestone	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Started	23	23
Completed	23	23
Not completed	0	0

Outcome measures

PrimaryPercentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) Based on 24-hour Urine Collection at Week 13

UPCR is a ratio between two measured substances in urine: milligram of protein per millimole (mmol) of creatinine, reported in units mg/mmol. A decrease in UPCR may be associated with improved renal and cardiovascular function.

Time frame:: Baseline, Week 13
Reported as:: Least squares mean · Percentage change

Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) Based on 24-hour Urine Collection at Week 13
Percentage change	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) Based on 24-hour Urine Collection at Week 13	-12.283 (-26.096 to 4.112)	-0.045 (-9.528 to 10.432)

SecondaryNumber of Participants With Treatment Emergent Adverse Events (TEAEs)

An adverse event was considered treatment emergent relative to a given treatment if the event occurred for the first time during the investigational treatment period and was not seen prior to the start of treatment (during the lead-in period), or the event was seen prior to the start of treatment but increased in severity during treatment. Adverse events occurring during the lead-in period were considered non-treatment emergent. Events that occurred during the follow-Up period were counted as treatment emergent and attributed to the previous treatment taken.

Time frame:: From Day 1 of treatment up to 9 weeks after last dose of study treatment (up to Week 33)
Reported as:: Count of participants · Participants

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Participants	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	20	17

SecondaryNumber of Participants With Abnormalities in Laboratory Test Parameters

Hemoglobin (Hg), hematocrit, erythrocytes: \<0.8\*lower limits of normal (LLN); platelets: \<0.5\*LLN\>1.75\*upper limits of normal(ULN); leukocytes (leu), glucose-fasting:\<0.6\*LLN\>1.5\*ULN; lymphocytes (lym), lym/leu, neutrophils(neu),neu/leu, protein, albumin, phosphate, free thyroxine, thyroid stimulating hormone: \<0.8\*LLN\>1.2\*ULN; basophils (bas), bas/leu, eosinophils (eos), eos/leu, monocytes(mon), mon/leu: \>1.2\*ULN; bilirubin (total, direct, indirect):\>1.5\*ULN; aspartate aminotransferase(AT), alanine AT, lactate dehydrogenase, alkaline phosphatase:\>3.0\*ULN; blood urea nitrogen, creatinine, cholesterol (total,LDL,HDL),triglycerides, Hg A1C: \>1.3\*ULN; sodium: \<0.95\*LLN\>1.05\*ULN; potassium, chloride, calcium, magnesium, bicarbonate: \<0.9\*LLLN\>1.1\*ULN; prolactin: \>1.1\*ULN; creatine kinase: \>2.0\*ULN; urobilinogen: \>=1; Urine-specific gravity: \<1.003\>1.030, pH: \<4.5 \>8, glucose,protein,bilirubin,nitrite,leukocyte esterase, ketones: \>=1.Categories with at-least 1 non-zero values are reported.

Time frame:: From Day 1 of treatment up to Week 33
Reported as:: Count of participants · Participants

Number of Participants With Abnormalities in Laboratory Test Parameters
Participants	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Hematology: Hemoglobin < 0.8 * Lower Limit Number (LLN)	5	4
Hematology: Hematocrit < 0.8 * LLN	5	7
Hematology: Erythrocytes < 0.8 * LLN	3	4
Hematology: Ery. Mean Corpuscular Volume < 0.9 * LLN	1	0
Hematology: Ery. Mean Corpuscular Hemoglobin < 0.9 * LLN	1	0
Hematology: Platelets> 1.75 * ULN	1	0
Hematology: Lymphocytes< 0.8 * LLN	0	4
Hematology: Lymphocytes/Leukocytes < 0.8 * LLN	1	6
Hematology: Lymphocytes/Leukocytes > 1.2 * ULN	0	1
Hematology: Neutrophils < 0.8 * LLN	1	2
Hematology: Neutrophils > 1.2 * ULN	3	4
Hematology: Neutrophils/Leukocytes < 0.8 * LLN	1	1
Hematology: Neutrophils/Leukocytes > 1.2 * ULN	0	1
Hematology: Eosinophils > 1.2 * ULN	2	1
Hematology: Eosinophils/Leukocytes> 1.2 * ULN	3	3
Hematology: Monocytes/Leukocytes> 1.2 * ULN	0	1
Clinical chemistry: Aspartate Aminotransferase > 3.0 * ULN	1	0
Clinical chemistry: Alanine Aminotransferase > 3.0 * ULN	1	0
Clinical chemistry: Protein < 0.8 * LLN	9	11
Clinical chemistry: Albumin < 0.8 * LLN	8	11
Clinical chemistry: Blood Urea Nitrogen > 1.3 * ULN	10	8
Clinical chemistry: Creatinine > 1.3 * ULN	8	12
Clinical chemistry: Urate > 1.2 * ULN	3	2
Clinical chemistry: Cholesterol > 1.3 * ULN	6	7
Clinical chemistry: HDL Cholesterol < 0.8 * LLN	1	0
Clinical chemistry: LDL Cholesterol > 1.2 * ULN	6	5
Clinical chemistry: Triglycerides > 1.3 * ULN	6	8
Clinical chemistry: Sodium < 0.95 * LLN	1	0
Clinical chemistry: Potassium < 0.9 * LLN	1	0
Clinical chemistry: Potassium > 1.1 * ULN	1	0
Clinical chemistry: Calcium < 0.9 * LLN	1	3
Clinical chemistry: Bicarbonate < 0.9 * LLN	0	1
Clinical chemistry: Glucose > 1.5 * ULN	2	2
Urinalysis: Specific Gravity > 1.030	5	6
Urinalysis: pH > 8	1	0
Urinalysis: Urine Glucose >= 1	5	10
Urinalysis: Urine Protein >=1	22	24
Urinalysis: Urine Hemoglobin >=1	15	17
Urinalysis: Nitrite >= 1	1	3
Urinalysis: Leukocyte Esterase >= 1	5	6
Urinalysis: Urine Erythrocytes >= 20	3	3
Urinalysis: Urine Leukocytes >= 20	2	5
Urinalysis: Granular Casts > 1	4	2
Urinalysis: Hyaline Casts > 1	17	15
Urinalysis: Urine Creatinine <40	9	8
Urinalysis: Urine Creatinine >300	1	0
Urinalysis: Urine (24HR) Creatinine > 1.1 * ULN	2	6

SecondaryChange From Baseline in Body Weight

Change from baseline in body weight and at baseline values were reported for this outcome measure.

Time frame:: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Reported as:: Mean · Kilogram

Change From Baseline in Body Weight
Kilogram	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Baseline	78.3 ± 17.8	88.9 ± 23.5
Week 2	1.0 ± 2.2	-0.3 ± 1.9
Week 3	0.4 ± 2.5	-0.3 ± 2.2
Week 5	0.5 ± 3.0	-0.5 ± 4.8
Week 7	0.6 ± 2.6	-1.0 ± 5.3
Week 9	0.1 ± 3.1	-0.4 ± 3.4
Week 11	0.7 ± 2.8	-0.1 ± 4.2
Week 13	-0.2 ± 1.9	-1.0 ± 3.4
Week15	-0.3 ± 1.0	-0.7 ± 4.0
Week 17	-0.2 ± 1.2	-1.2 ± 4.8
Week 19	-0.4 ± 1.0	-1.3 ± 4.8
Week 21	0.3 ± 1.7	-1.3 ± 4.9
Week 23	0.3 ± 1.2	-1.5 ± 5.3
Week 25	-0.1 ± 1.6	-1.1 ± 4.8
Week 27	0.7 ± 1.4	-0.6 ± 5.2
Week 29	0.5 ± 1.5	-1.0 ± 4.5
Week 33	0.5 ± 1.6	-0.6 ± 5.4

SecondaryChange From Baseline in Blood Pressure

Change from baseline in blood pressure and at baseline values were reported for this outcome measure.

Time frame:: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Reported as:: Mean · Millimeter of mercury (mmHg)

Change From Baseline in Blood Pressure
Millimeter of mercury (mmHg)	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Baseline	131.2 ± 12.8	125.3 ± 11.8
Week 2	-1.8 ± 13.1	-1.3 ± 7.8
Week 3	-1.3 ± 10.2	1.7 ± 7.5
Week 5	-3.0 ± 10.6	-1.9 ± 9.0
Week 7	-2.0 ± 11.3	-1.2 ± 9.0
Week 9	-5.1 ± 10.7	-2.6 ± 7.6
Week 11	-1.5 ± 12.5	-1.6 ± 6.1
Week 13	-2.0 ± 11.3	-1.0 ± 10.0
Week15	-7.5 ± 5.1	-2.3 ± 6.2
Week 17	-7.3 ± 12.7	-7.7 ± 12.2
Week 19	-8.0 ± 8.4	0.4 ± 10.7
Week 21	-8.0 ± 16.4	-5.8 ± 10.1
Week 23	1.0 ± 9.0	-3.6 ± 8.2
Week 25	-9.0 ± 13.2	-2.6 ± 11.2
Week 27	-3.0 ± 10.4	3.5 ± 10.5
Week 29	-8.3 ± 8.4	0.4 ± 11.2
Week 33	-12.0 ± 20.7	4.6 ± 8.9

SecondaryChange From Baseline in Pulse Rate

Change from baseline in pulse rate and at baseline values were reported for this outcome measure.

Time frame:: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Reported as:: Mean · Beats per minute

Change From Baseline in Pulse Rate
Beats per minute	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Baseline	74.1 ± 11.8	74.2 ± 11.0
Week 2	2.1 ± 9.6	0.6 ± 6.9
Week 3	-0.6 ± 8.0	-1.1 ± 6.0
Week 5	0.0 ± 7.3	-0.3 ± 8.0
Week 7	-0.3 ± 9.2	0.0 ± 7.9
Week 9	-1.0 ± 11.1	1.5 ± 9.0
Week 11	-1.7 ± 10.8	0.6 ± 6.6
Week 13	3.3 ± 11.9	0.5 ± 6.9
Week15	-5.8 ± 10.4	5.0 ± 10.1
Week 17	-6.3 ± 8.8	-2.2 ± 5.0
Week 19	-3.5 ± 13.7	2.9 ± 6.5
Week 21	-5.5 ± 12.8	-1.6 ± 5.0
Week 23	-3.3 ± 10.5	-3.1 ± 9.0
Week 25	-6.3 ± 12.5	1.8 ± 7.6
Week 27	-0.8 ± 16.0	2.9 ± 7.2
Week 29	2.5 ± 14.8	5.9 ± 8.8
Week 33	-0.5 ± 15.3	6.2 ± 10.0

SecondaryChange From Baseline in Body Temperature

Change from baseline in body temperature and at baseline values were reported for this outcome measure.

Time frame:: Baseline, Change at Weeks 2, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 29, 33
Reported as:: Mean · Degree Celsius

Change From Baseline in Body Temperature
Degree Celsius	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Baseline	36.4 ± 0.4	36.6 ± 0.2
Week 2	0.1 ± 0.4	-0.0 ± 0.3
Week 3	0.0 ± 0.2	-0.1 ± 0.3
Week 5	0.0 ± 0.4	-0.1 ± 0.3
Week 7	0.1 ± 0.3	-0.0 ± 0.3
Week 9	0.0 ± 0.4	-0.1 ± 0.3
Week 11	0.1 ± 0.3	-0.0 ± 0.3
Week 13	-0.1 ± 0.4	-0.0 ± 0.2
Week 15	0.2 ± 0.2	0.1 ± 0.2
Week 17	0.1 ± 0.2	0.1 ± 0.2
Week 19	0.1 ± 0.4	0.1 ± 0.3
Week 21	-0.0 ± 0.1	-0.1 ± 0.5
Week 23	0.2 ± 0.2	0.0 ± 0.3
Week 25	0.1 ± 0.2	0.0 ± 0.2
Week 27	0.1 ± 0.1	0.1 ± 0.2
Week 29	0.1 ± 0.3	-0.1 ± 0.4
Week 33	0.1 ± 0.1	-0.1 ± 0.3

SecondaryNumber of Participants With Abnormalities in Electrocardiogram (ECG)

ECG abnormalities criteria included: 1) QTc interval adjusted according to Bazett formula (QTcB) in millisecond (msec): greater than (\>) 450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; 2) QTc interval adjusted according to Fridericia formula (QTcF) (msec): \>450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; 3) Heart rate (bpm): RR decrease \>25% and to a VR (interval between QRS wave and T wave on ECG) \>100; RR (interval between 2 successive R waves on ECG) increase \>25% and to a VR \<50; 4) Pulse rate (msec): increase \>25% and to a value \>200; 5) QT (msec): \>450, \>480, \>500, increase from baseline \>30, increase from baseline \>60; 6) QRS (msec): increase \>25% and to a value \>110. Categories (timepoints) with at least 1 participant having ECG abnormality in any of the reporting arms, were reported for this outcome measure.

Time frame:: Weeks 3, 7, 11, 13, 17, 21, 25, 33
Reported as:: Count of participants · Participants

Number of Participants With Abnormalities in Electrocardiogram (ECG)
Participants	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Week 3 (Not Clinically Significant)	3	4
Week 3 (Clinically Significant)	1	0
Week 7 (Not Clinically Significant)	3	4
Week 7 (Clinically Significant)	0	0
Week 11 (Not Clinically Significant)	4	4
Week 11 (Clinically Significant)	1	0
Week 13 (Not Clinically Significant)	6	4
Week 13 (Clinically Significant)	1	0
Week 17 (Not Clinically Significant)	0	0
Week 17 (Clinically Significant)	0	0
Week 21 (Not Clinically Significant)	1	1
Week 21 (Clinically Significant)	0	0
Week 25 (Not Clinically Significant)	1	1
Week 25 (Clinically Significant)	0	0
Week 33 (Not Clinically Significant)	0	2
Week 33 (Clinically Significant)	0	0

SecondaryPercentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) at Weeks 2, 5, 9 and 13

UPCR is a ratio between two measured substances in urine: mmol of creatinine, reported in units mg/mmol. A decrease in UPCR may be associated with improved renal and cardiovascular function.

Time frame:: Baseline, Weeks 2, 5, 9 and 13
Reported as:: Least squares mean · Percent change

Percentage Change From Baseline in Urinary Protein to Creatinine Ratio (UPCR) at Weeks 2, 5, 9 and 13
Percent change	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Week 5	6.250 (-7.035 to 21.433)	-3.788 (-11.344 to 4.411)
Week 9	-11.335 (-20.746 to -0.807)	-2.354 (-11.069 to 7.215)
Week 13	-12.283 (-26.096 to 4.112)	-0.045 (-9.528 to 10.432)

SecondaryPercentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR ) at Weeks 3, 5, 9 and 13

The eGFR was calculated using 4 variable formula developed by the modification of diet in renal disease (MDRD) study group. The 4 variables needed to estimate glomerular filtration rate (GFR) using this formula were serum creatinine concentration, age, sex (for females, eGFR was multiplied by 0.742) and ethnic origin (for African-Caribbean people only, eGFR was multiplied by 1.212). Thus eGFR in milliliter per minute per 1.73 square meter (mL/min/1.73 m\^2) = 175\*(sCr/88.4)\^-1.154\*(Age)\^-0.203\*(0.742 if female)\*(1.212 if African-Caribbean). Baseline eGFR was determined predose at Week 0 (Day 1). For Baseline eGFR, the "Low eGFR" group was defined as baseline eGFR \< 45 mL/min/1.73m2, and the "High eGFR" group was defined as baseline eGFR \> 45 mL/min/1.73 m2.

Time frame:: Baseline, Weeks 3, 5, 9 and 13
Reported as:: Least squares mean · Percent change

Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR ) at Weeks 3, 5, 9 and 13
Percent change	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Week 3	5.657 (-0.722 to 12.446)	-0.180 (-4.179 to 3.987)
Week 5	2.174 (-3.012 to 7.637)	-2.038 (-8.008 to 4.319)
Week 9	-1.536 (-15.575 to 14.838)	-5.017 (-11.596 to 2.052)
Week 13	2.892 (-6.096 to 12.740)	-12.217 (-18.831 to -5.063)

SecondarySerum PF-06730512 Concentration Versus Time Summary

Time frame:: For 12-Week treatment(WT):pre-dose on Day1,8,15,29,43,57,71,follow-up(Fup)visit on Day85,99,113,141,For 24-WT:pre-dose on Day1,8,15,29,43,57,71,85,99,113,127,141,155,Fup visit on Day169,183,197,225;1hour post-dose on Day1,71,155(only applicable for 24-WT)
Reported as:: Mean · Microgram per milliliter

Serum PF-06730512 Concentration Versus Time Summary
Microgram per milliliter	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
Day 1: Pre-dose (12,24WT)	0 ± 0	0 ± 0
Day 1: Post-dose (12,24WT)	279.4 ± 86.548	71.94 ± 29.668
Day 8: Pre-dose (12,24WT)	39.50 ± NA	13.09 ± 6.9363
Day 15: Pre-dose (12,24WT)	16.45 ± 11.266	5.096 ± 3.1184
Day 29: Pre-dose (12,24WT)	24.92 ± 20.256	6.916 ± 5.0154
Day 43: Pre-dose (12,24WT)	29.96 ± 22.062	6.991 ± 5.5862
Day 57: Pre-dose (12,24WT)	32.56 ± 23.031	7.664 ± 6.1325
Day 71: Pre-dose (12,24WT)	27.32 ± 16.688	6.736 ± 5.6237
Day 71: Post-dose(12,24WT)	298.6 ± 208.87	129.2 ± 201.50
Day 85: Pre-dose (24WT)	41.15 ± 23.516	7.597 ± 6.9184
Day 85/Fup (12 WT)	27.19 ± 21.589	6.884 ± 7.1511
Day 99/Fup (12 WT)	11.50 ± 12.948	2.313 ± 3.1827
Day 113/Fup (12 WT)	4.991 ± 6.1121	0.6356 ± 0.81118
Day 141/Fup (12 WT)	1.385 ± 2.1196	0.1438 ± 0.21666
Day 99: Pre-dose (24WT)	35.65 ± 16.065	7.084 ± 6.9224
Day 113: Pre-dose (24WT)	42.40 ± 25.049	9.442 ± 8.6965
Day 127: Pre-dose (24WT)	30.67 ± 5.3463	6.732 ± 5.2295
Day 141: Pre-dose (24WT)	38.00 ± 13.880	8.957 ± 10.753
Day 155: Pre-dose (24WT)	32.57 ± 5.8287	7.585 ± 8.1601
Day 155: Post-dose (24WT)	313.3 ± 63.793	62.71 ± 41.324
Day 169: Pre-dose/Fup (24WT)	29.32 ± 15.338	7.989 ± 7.4740
Day 183/Fup (24WT)	25.79 ± 30.417	3.046 ± 3.8895
Day 197/Fup (24WT)	6.108 ± 4.0049	1.048 ± 1.4978
Day 225/Fup (24WT)	2.009 ± 1.6808	0.2659 ± 0.45714

SecondaryNumber of Participants With Positive Anti-Drug Antibody (ADA) and Neutralizing Antibody(NAb)

Number of participants with positive ADA and/or NAb were reported for this outcome measure.

Time frame:: From Day 1 of treatment up to Week 33
Reported as:: Count of participants · Participants

Number of Participants With Positive Anti-Drug Antibody (ADA) and Neutralizing Antibody(NAb)
Participants	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
ADA Positive	1	0
nAb Positive	0	0

Adverse events

Collected over From Day 1 of treatment up to 9 weeks after last dose of study treatment (up to Week 33). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
PF-06730512 1000 mg IV	0/23 (0%)	2/23 (8.7%)	11/23 (47.8%)
PF-06730512 300 mg IV	0/24 (0%)	2/24 (8.3%)	12/24 (50%)

Most frequent serious events

Most frequent serious events
Event	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
DiarrhoeaGastrointestinal disorders	1/23	0/24
HypervolaemiaMetabolism and nutrition disorders	1/23	0/24
Acute kidney injuryRenal and urinary disorders	1/23	0/24
Renal impairmentRenal and urinary disorders	1/23	0/24
Disease progressionGeneral disorders	0/23	1/24
COVID-19Infections and infestations	0/23	1/24

Most frequent other events

Showing 10 of 18

Most frequent other events
Event	PF-06730512 1000 mg IV	PF-06730512 300 mg IV
FatigueGeneral disorders	3/23	2/24
HeadacheNervous system disorders	2/23	3/24
VomitingGastrointestinal disorders	2/23	1/24
PyrexiaGeneral disorders	2/23	0/24
Viral infectionInfections and infestations	2/23	0/24
SARS-CoV-2 test positiveInvestigations	2/23	2/24
DehydrationMetabolism and nutrition disorders	2/23	0/24
InsomniaPsychiatric disorders	2/23	0/24
CoughRespiratory, thoracic and mediastinal disorders	2/23	0/24
DiarrhoeaGastrointestinal disorders	1/23	2/24

Baseline characteristics

Safety Analysis Set (SAS) was defined as all enrolled participants who had received at least one dose of study treatment.

Age, Continuous
Age, Continuous(Years)	PF-06730512 1000 mg IV	PF-06730512 300 mg IV	Total
Mean	42.7 ± 13.64	41.0 ± 14.60	41.8 ± 14.01

Sex: Female, Male
Sex: Female, Male(Participants)	PF-06730512 1000 mg IV	PF-06730512 300 mg IV	Total
Female	12	10	22
Male	11	14	25

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	PF-06730512 1000 mg IV	PF-06730512 300 mg IV	Total
Hispanic or Latino	5	3	8
Not Hispanic or Latino	18	21	39
Unknown or Not Reported	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	PF-06730512 1000 mg IV	PF-06730512 300 mg IV	Total
American Indian or Alaska Native	0	1	1
Asian	3	5	8
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	0	2	2
White	20	16	36
More than one race	0	0	0
Unknown or Not Reported	0	0	0

Study locations

110 sites

The Kirklin Clinic of University Alabama Birmingham Hospital
Birmingham, Alabama 35233, United States
Investigational Drug Service Pharmacy UAB Hosptial
Birmingham, Alabama 35249, United States
Clinical Research Unit at UAB Hospital
Birmingham, Alabama 35294, United States
UAB Nephrology Research Clinic at Paula Building
Birmingham, Alabama 35294, United States
Academic Medical Research Institute
Los Angeles, California 90022, United States
Cedars Sinai Medical Center
Los Angeles, California 90048, United States
Cedars-Sinai Ambulatory Infusion Center
Los Angeles, California 90048, United States
Cedars-Sinai Comprehensive Transplant Center
Los Angeles, California 90048, United States
Ronald Reagan UCLA Medical Center
Los Angeles, California 90095, United States
UCLA Clinical and Translational Research Center
Los Angeles, California 90095, United States
UCLA Department of Medicine
Los Angeles, California 90095, United States
Clinical and Translation Research Unit
Palo Alto, California 94304, United States
Stanford University-Nephrology Division
Palo Alto, California 94304, United States
University of California, San Francisco
San Francisco, California 94143, United States
Stanford Health Care Investigational Pharmacy
Stanford, California 94305, United States
Stanford University-Nephrology Division
Stanford, California 94305, United States
Stanford University
Stanford, California 94305, United States
Yale Center for Clinical Investigation Hospital Research Unit
New Haven, Connecticut 06510, United States
Yale Nephrology Clinical Research Clinic
New Haven, Connecticut 06510, United States
Yale University School of Medicine - Yale-New Haven Hospital
New Haven, Connecticut 06510, United States
Yale University
New Haven, Connecticut 06510, United States
Yale New Haven Hospital
New Haven, Connecticut 06511, United States
Yale Center for Clinical Investigation - Church Street Research Unit
New Haven, Connecticut 06519, United States
University of Miami Hospital and Clinics
Miami, Florida 33136, United States
University of Miami Katz Family Division of Nephrology
Miami, Florida 33136, United States
University of Miami
Miami, Florida 33136, United States
Georgia Nephrology Research Institute
Lawrenceville, Georgia 30046, United States
Georgia Nephrology
Lawrenceville, Georgia 30046, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242, United States
Johnson County Clin Trials
Lenexa, Kansas 66219, United States
Boston Medical Center - Interventional Radiology
Boston, Massachusetts 02118, United States
Boston Medical Center - Nephrology
Boston, Massachusetts 02118, United States
Boston Medical Center
Boston, Massachusetts 02118, United States
Boston University - GCRU
Boston, Massachusetts 02118, United States
Henry Ford Hospital
Detroit, Michigan 48202, United States
Clinical Research Consultants, LLC
Kansas City, Missouri 64111, United States
St. Luke's Hospital
Kansas City, Missouri 64111, United States
New York University Grossman School of Medicine - CTSI
New York, New York 10016, United States
UNC Eastowne
Chapel Hill, North Carolina 27514, United States
UNC Clinical and Translational Research Center
Chapel Hill, North Carolina 27599-7064, United States
University of Cincinnati at DCI McMillan Research Unit
Cincinnati, Ohio 45206, United States
Hoxworth Center Subspecialties Clinic
Cincinnati, Ohio 45219, United States
UC Health Barrett Center
Cincinnati, Ohio 45219, United States
UC Health Medical Arts Building
Cincinnati, Ohio 45219, United States
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio 45229, United States
The Cleveland Clinic - Investigational Drug Pharmacy
Cleveland, Ohio 44195, United States
The Cleveland Clinic
Cleveland, Ohio 44195, United States
CarePoint East at The Ohio State University
Columbus, Ohio 43203, United States
The Ohio State University Clinical Research Center
Columbus, Ohio 43210, United States
The Ohio State University Investigational Drug Services
Columbus, Ohio 43210, United States
The Ohio State University Wexner Medical Center- Nephrology Clinical Trials Unit
Columbus, Ohio 43210, United States
The Ohio State University
Columbus, Ohio 43210, United States
Kidney and Hypertension Care Center, PA
Houston, Texas 77004, United States
Southside Pharmacy
Houston, Texas 77030, United States
Prolato Clinical Research Center (PCRC)
Houston, Texas 77054, United States
Baylor Scott & White Clinic - Temple South Loop
Temple, Texas 76502, United States
University of Alberta - Pharmacy Research Office
Edmonton, Alberta T6G 1Z1, Canada
University of Alberta - Clinical Investigation Unit
Edmonton, Alberta T6G 287, Canada
University of Alberta Hospital
Edmonton, Alberta T6G 2B7, Canada
Pacific Nephrology Group
Vancouver, British Columbia V5Z 1M9, Canada
Vancouver General Hospital/Vancouver Coastal Health
Vancouver, British Columbia V5Z 1M9, Canada
St. Paul's Hospital/Providence Health Care
Vancouver, British Columbia V6Z 1Y6, Canada
St. Paul's Hospital/Providence Health Care
Vancouver, British Columbia V6Z 2K8, Canada
Sunnybrook Health Sciences Centre - Kidney Care Centre at the CNIB
Toronto, Ontario M4G 3E8, Canada
University Health Network
Toronto, Ontario M5G 2C4, Canada
CIUSSS de l'Est-de-l'Ile-de-Montreal - installation Hopital Maisonneuve-Rosemont
Montreal, Quebec H1T 2M4, Canada
Centre for Innovative Medicine, Research Institute of the McGill University Health Centre
Montreal, Quebec H4A 3J1, Canada
CHU de Quebec-Universite Laval
Quebec, G1R 2J6, Canada
Fakultni nemocnice Hradec Kralove
Hradec Kralove, 500 05, Czechia
Vseobecna fakultni nemocnice v Praze
Praha 2, 128 08, Czechia
Hopital Henri Mondor
Creteil, 94010, France
CHU de Nice - Hopital Pasteur
Nice Cedex 1, 06001, France
Hopital Necker - Enfants Malades
Paris, 75015, France
Universitätsklinikum Dresden, Medizinische Klinik III, Nephrologie
Dresden, Saxony 01307, Germany
Universitaetsklinikum Aachen
Aachen, 52074, Germany
Charite - Universitaetsmedizin Berlin
Berlin, 10117, Germany
Universitaetsklinikum Erlangen
Erlangen, 91054, Germany
Medizinische Hochschule Hannover
Hannover, 30625, Germany
Uniklinik Koeln
Koeln, 50937, Germany
Universitaetsklinikum Mannheim
Mannheim, 68167, Germany
Nephrologisches Zentrum Villingen-Schwenningen
Villingen-Schwenningen, 78052, Germany
Sidonia Apotheke
Villingen-Schwenningen, 78052, Germany
Ics Maugeri Spa-Sb Irccs
Pavia PV, Pavia 27100, Italy
Asst Papa Giovanni Xxiii
Bergamo, 24127, Italy
Nagoya University Hospital
Nagoya, Aichi 466-8560, Japan
Tohoku University Hospital
Sendai, Miyagi 980-8574, Japan
Osaka University Hospital
Suita, Osaka 565-0871, Japan
National Hospital Organization Chiba-East Hospital
Chiba, 260-8712, Japan
Niigata University Medical & Dental Hospital
Niigata, 951-8510, Japan
Hospital Universitario "Dr Jose Eleuterio Gonzalez"
Monterrey, Nuevo LEON 64460, Mexico
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Mexico City, 14080, Mexico
SMIQ S de RL de CV
Queretaro, 76070, Mexico
Samodzielny Publiczny Zaklad Opieki Zdrowotnej
Lodz, 92-213, Poland
Apteka Szpitalna SPZOZ
Lodz, 92-216, Poland
Centrum Zdrowia MDM
Warszawa, 00-631, Poland
Miedzyleski Szpital Specjalistyczny w Warszawie
Warszawa, 04-749, Poland
Fakultna nemocnica s poliklinikou F.D. Roosevelta Banska Bystrica
Banska Bystrica, 975 01, Slovakia
Fakultna nemocnica s poliklinikou F.D. Roosevelta Banska Bystrica
Banska Bystrica, 975 17, Slovakia
Univerzitna nemocnica Bratislava
Bratislava, 831 01, Slovakia
Narodny ustav detskych chorob
Bratislava, 833 40, Slovakia

Showing the first 100 of 110 sites across 12 countries.

References and documents

Publications

Beck LH Jr, Berasi SP, Copley JB, Gorman D, Levy DI, Lim CN, Henderson JM, Salant DJ, Lu W. PODO: Trial Design: Phase 2 Study of PF-06730512 in Focal Segmental Glomerulosclerosis. Kidney Int Rep. 2021 Apr 3;6(6):1629-1633. doi: 10.1016/j.ekir.2021.03.892. eCollection 2021 Jun. PubMed 34169203 ↗

Study documents

Study protocol · Aug 18, 2021
Statistical analysis plan · Feb 23, 2023

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Registry details

Key details

Study ID: NCT03448692
Lead sponsor: Pfizer
Responsible party: Sponsor
First posted: Feb 28, 2018
Start date: Oct 15, 2018
Primary completion: Feb 14, 2023
Completion: Feb 14, 2023
Results posted: Apr 18, 2024
Last update: Apr 18, 2024

Study contacts

Pfizer CT.gov Call Center

study director · Pfizer

Oversight

FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.

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