Clinical Trials List
Protocol NumberAPL2-FSG-319
NCT Number(ClinicalTrials.gov Identfier)NCT07213960
Not yet recruiting
2026-10-01 - 2030-03-31
Phase II/III
Not yet recruiting5
A sequential phase 2/3 trial, initially a single-arm, open-label trial in adults, followed by a randomized, placebo-controlled, double-blind, multicenter trial in adults and adolescents, will evaluate the efficacy and safety of Pegcetacoplan in treating focal segmental glomerulosclerosis.
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Sponsor
Thermo fisher Co., Ltd. Taiwan Branch
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Trial scale
Multi-Regional Multi-Center
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Update
2026/09/11
Investigators and Locations
Co-Principal Investigator
- Yen-Chung Lin Division of Nephrology
- 陳靜怡 Division of Nephrology
- Chih-Chin Kao Division of Nephrology
- Hsi-Hsien Chen Division of Nephrology
- Te-Chao Fang Division of Nephrology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- Cai-Mei Zheng Division of Nephrology
- Mei-Yi Wu Division of Nephrology
- 高芷華 Division of Nephrology
- Chia-Te Liao Division of Nephrology
- Li-Yee Hong Division of Nephrology
- Yu-Wei Chen Division of Nephrology
- 林冠宏 Division of Nephrology
- YUNG-HO HSU Division of Nephrology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- WEN-CHIH CHIANG Division of General Internal Medicine
- - - Division of General Internal Medicine
- 趙家德 Division of General Internal Medicine
- 黃道民 Division of General Internal Medicine
- 蘇祺婷 Division of General Internal Medicine
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Principal Investigator
Guan-Hsing Chen
Co-Principal Investigator
- Cheng-Chia Lee Division of Nephrology
- Kun-Hua Tu Division of Nephrology
- 吳欣旭 Division of Nephrology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Condition/Disease
Focal segmental glomerulosclerosis
Objectives
This trial is a sequential Phase 2/3 trial designed to evaluate the efficacy and safety of twice-weekly subcutaneous (SC) infusion of pegcetacoplan in patients diagnosed with focal segmental glomerulosclerosis (FSGS). The initial Phase 2 portion of the trial is a single-arm, open-label trial in adults diagnosed with FSGS. Phase 2 will begin prior to randomization in Phase 3. The Phase 3 portion of the trial is a randomized, placebo-controlled, double-blind, multicenter trial in adults and adolescents diagnosed with FSGS. An independent data monitoring committee will review efficacy and safety data from Phase 2 adult participants before enrolling adolescents in Phase 3.
Test Drug
Pegcetacoplan (APL-2) 1080 mg in 20 mL (54 mg/mL) or Placebo solution
Pegcetacoplan (APL-2) 1080 mg in 20mL (54mg/ml) solution
Pegcetacoplan (APL-2) 1080 mg in 20mL (54mg/ml) solution
Active Ingredient
PEGCETACOPLAN
PEGCETACOPLAN
PEGCETACOPLAN
Dosage Form
Subcutaneous injections
Subcutaneous injections
Subcutaneous injections
Dosage
1080 mg/20ml
1080mg/20ml
1080mg/20ml
Endpoints
The primary objective was to assess the efficacy of twice-weekly SC administration of pegcetacoplan compared to placebo in patients with FSGS, based on the reduction in proteinuria. The primary efficacy endpoint was the change in the log-transformed ratio of urine protein/creatinine (uPCR) at weeks 52 and 104 compared to baseline.
Inclution Criteria
1. Age
• Phase 2: Adults ≥18 years of age
• Phase 3: Adults ≥18 years of age at the time of signing the subject consent form and consent (adolescent); adolescents (12-17 years of age) if approved
2. Weight at screening: ≥30 kg and ≤100 kg
3. FSGS Diagnosis
• Phase 2: Primary, hereditary, or unexplained FSGS diagnosed by renal tissue section and confirmed by the center
• Phase 3: Primary, hereditary, or unexplained FSGS diagnosed by renal tissue section or confirmed by an identified podocyte gene mutation and confirmed by the center
4. 24-hour urinary protein excretion at screening: at least 1.5 g/day, and uPCR at least 1.5 g/g in at least two FMU samples collected during the screening period
5. Estimated glomerular filtration rate (eGFR): ≥25 mL/min/1.73 m2 is calculated using the following formulas: Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine formula (for adults) or the creatinine-based U25 formula for pediatric chronic kidney disease (for adolescents).
6. A stable FSGS treatment regimen has been received for at least 12 weeks prior to the first IP dose, and this regimen has no planned or anticipated adjustments or dose changes, such as any or all of the following:
• An angiotensin-converting enzyme inhibitor, angiotensin receptor blocker, and/or sodium-glucose cotransporter-2 inhibitor therapy that is stable and optimized (maximum tolerated dose) as assessed by the trial administrator.
• Other medications at stable doses that may affect proteinuria (e.g., steroids, mycophenolate mofetil, calcineurin inhibitors, and/or other immunosuppressants currently used by the subject for the treatment of FSGS).
• If the subject is using prednisone (or other systemic corticosteroids) to treat FSGS, the dose is stable and does not exceed 10 mg/day (or prednisone...). (Other corticosteroid equivalent doses)
7. Subjects have completed pneumococcal and meningococcal (types A, C, W, Y, and B) vaccinations. These vaccinations are mandatory unless data supports that the subject has received the recommended vaccinations for adults or children with complement deficiency. If a subject is required to receive a vaccination as detailed in the trial protocol, the first vaccination in that series should be administered at least 14 days before the first dose of IP; the second vaccination (if applicable) should be administered at least 8 weeks after the first vaccination.
8. Subjects are willing and able to administer pegcetacoplan themselves, or have a designated caregiver to assist with administration.
9. Female subjects of reproductive age (defined as any female subject who has reached menarche and is not permanently sterilized or has not reached menopause) must have a negative urine pregnancy test on day 1 at screening and must agree to use the method of contraception specified in the trial protocol for at least 90 days from the start of the screening period until the last dose of IP.
10. Male participants must agree to use the contraceptive method specified in the trial protocol and avoid donating semen for at least 90 days from the start of the screening period until after receiving the last dose of IP.
11. Subjects of legal age of consent must be willing and able to provide informed consent, as required by local regulations. For subjects below the legal age of consent, their legal representative or guardian must be willing and able to provide informed consent; where applicable, the subject below the legal age of consent must also provide consent to participate in the trial (for adolescents).
• Phase 2: Adults ≥18 years of age
• Phase 3: Adults ≥18 years of age at the time of signing the subject consent form and consent (adolescent); adolescents (12-17 years of age) if approved
2. Weight at screening: ≥30 kg and ≤100 kg
3. FSGS Diagnosis
• Phase 2: Primary, hereditary, or unexplained FSGS diagnosed by renal tissue section and confirmed by the center
• Phase 3: Primary, hereditary, or unexplained FSGS diagnosed by renal tissue section or confirmed by an identified podocyte gene mutation and confirmed by the center
4. 24-hour urinary protein excretion at screening: at least 1.5 g/day, and uPCR at least 1.5 g/g in at least two FMU samples collected during the screening period
5. Estimated glomerular filtration rate (eGFR): ≥25 mL/min/1.73 m2 is calculated using the following formulas: Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine formula (for adults) or the creatinine-based U25 formula for pediatric chronic kidney disease (for adolescents).
6. A stable FSGS treatment regimen has been received for at least 12 weeks prior to the first IP dose, and this regimen has no planned or anticipated adjustments or dose changes, such as any or all of the following:
• An angiotensin-converting enzyme inhibitor, angiotensin receptor blocker, and/or sodium-glucose cotransporter-2 inhibitor therapy that is stable and optimized (maximum tolerated dose) as assessed by the trial administrator.
• Other medications at stable doses that may affect proteinuria (e.g., steroids, mycophenolate mofetil, calcineurin inhibitors, and/or other immunosuppressants currently used by the subject for the treatment of FSGS).
• If the subject is using prednisone (or other systemic corticosteroids) to treat FSGS, the dose is stable and does not exceed 10 mg/day (or prednisone...). (Other corticosteroid equivalent doses)
7. Subjects have completed pneumococcal and meningococcal (types A, C, W, Y, and B) vaccinations. These vaccinations are mandatory unless data supports that the subject has received the recommended vaccinations for adults or children with complement deficiency. If a subject is required to receive a vaccination as detailed in the trial protocol, the first vaccination in that series should be administered at least 14 days before the first dose of IP; the second vaccination (if applicable) should be administered at least 8 weeks after the first vaccination.
8. Subjects are willing and able to administer pegcetacoplan themselves, or have a designated caregiver to assist with administration.
9. Female subjects of reproductive age (defined as any female subject who has reached menarche and is not permanently sterilized or has not reached menopause) must have a negative urine pregnancy test on day 1 at screening and must agree to use the method of contraception specified in the trial protocol for at least 90 days from the start of the screening period until the last dose of IP.
10. Male participants must agree to use the contraceptive method specified in the trial protocol and avoid donating semen for at least 90 days from the start of the screening period until after receiving the last dose of IP.
11. Subjects of legal age of consent must be willing and able to provide informed consent, as required by local regulations. For subjects below the legal age of consent, their legal representative or guardian must be willing and able to provide informed consent; where applicable, the subject below the legal age of consent must also provide consent to participate in the trial (for adolescents).
Exclusion Criteria
1. Previous exposure to pegcetacoplan
2. Evidence of improvement in kidney disease within 8 weeks prior to screening or during screening, based on available data; improvement in kidney disease is defined as an increase in eGFR >30% or a decrease in proteinuria >50%.
3. FSGS secondary to other conditions (e.g., infection, diabetes, drug-induced, obesity, preterm birth, sickle cell anemia, vesicoureteral reflux, congenital kidney and urinary tract abnormalities).
4. Type 1 diabetes or poorly controlled type 2 diabetes (HbA1C ≥8%).
5. History of kidney transplantation.
6. Current or previous diagnosis of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection, or a positive serological or viral load during screening indicating active infection with any of these viruses.
7. Hypersensitivity to pegcetacoplan or any of its excipients
8. History of meningococcal disease
9. Malignancy, except for the following:
• Cured basal cell carcinoma or squamous cell carcinoma of the skin
• Carcinoma in situ that has received radical treatment
• No malignancy and treatment discontinued ≥5 years
10. Severe infection within 14 days prior to the first IP dose (e.g., requiring intravenous antibiotic treatment); intravenous treatment for severe infection must be completed >14 days prior to the first IP dose.
11. Absolute neutrophil count at screening <1000 cells/mm3
12. Other major kidney disease that may confound the interpretation of test results, as assessed by the trial administrator.
13. Participation in any other investigational drug trial or exposure to other investigational drugs, devices, or procedures prior to the screening period, and still within 30 days or 5 half-lives (whichever is longer) of the last administration of that investigational drug. 14. Prior to the screening period, the patient had used rituximab, belimumab, or any approved or investigational anticomplement therapy and is still within 5 half-lives of the investigational drug.
15. The patient is pregnant or breastfeeding and does not wish to discontinue breastfeeding for at least 90 days during the trial and after the last IP dose.
16. The patient is unable to cooperate, or, as assessed by the trial administrator, has any condition that would pose an undue risk to the patient or potentially obscure the interpretation of trial results.
17. As assessed by the trial administrator, there is evidence of persistent drug or alcohol abuse or dependence.
18. Known or suspected hereditary fructose intolerance.
19. The patient has or is suspected of having a severe infection (including but not limited to recurrent or chronic infections) during the screening period, and, as assessed by the trial administrator, this infection could pose an unacceptable risk to the patient's participation in the trial.
2. Evidence of improvement in kidney disease within 8 weeks prior to screening or during screening, based on available data; improvement in kidney disease is defined as an increase in eGFR >30% or a decrease in proteinuria >50%.
3. FSGS secondary to other conditions (e.g., infection, diabetes, drug-induced, obesity, preterm birth, sickle cell anemia, vesicoureteral reflux, congenital kidney and urinary tract abnormalities).
4. Type 1 diabetes or poorly controlled type 2 diabetes (HbA1C ≥8%).
5. History of kidney transplantation.
6. Current or previous diagnosis of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection, or a positive serological or viral load during screening indicating active infection with any of these viruses.
7. Hypersensitivity to pegcetacoplan or any of its excipients
8. History of meningococcal disease
9. Malignancy, except for the following:
• Cured basal cell carcinoma or squamous cell carcinoma of the skin
• Carcinoma in situ that has received radical treatment
• No malignancy and treatment discontinued ≥5 years
10. Severe infection within 14 days prior to the first IP dose (e.g., requiring intravenous antibiotic treatment); intravenous treatment for severe infection must be completed >14 days prior to the first IP dose.
11. Absolute neutrophil count at screening <1000 cells/mm3
12. Other major kidney disease that may confound the interpretation of test results, as assessed by the trial administrator.
13. Participation in any other investigational drug trial or exposure to other investigational drugs, devices, or procedures prior to the screening period, and still within 30 days or 5 half-lives (whichever is longer) of the last administration of that investigational drug. 14. Prior to the screening period, the patient had used rituximab, belimumab, or any approved or investigational anticomplement therapy and is still within 5 half-lives of the investigational drug.
15. The patient is pregnant or breastfeeding and does not wish to discontinue breastfeeding for at least 90 days during the trial and after the last IP dose.
16. The patient is unable to cooperate, or, as assessed by the trial administrator, has any condition that would pose an undue risk to the patient or potentially obscure the interpretation of trial results.
17. As assessed by the trial administrator, there is evidence of persistent drug or alcohol abuse or dependence.
18. Known or suspected hereditary fructose intolerance.
19. The patient has or is suspected of having a severe infection (including but not limited to recurrent or chronic infections) during the screening period, and, as assessed by the trial administrator, this infection could pose an unacceptable risk to the patient's participation in the trial.
The Estimated Number of Participants
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Taiwan
16 participants
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Global
265 participants
