AN OPEN-LABEL, RANDOMIZED, CONTROLLED, PHASE 2 STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PEGCETACOPLAN IN THE TREATMENT OF POST-TRANSPLANT RECURRENCE OF C3G OR IC*MPG
- Conditions
- Post-Transplant Recurrence10027665
- Registration Number
- NL-OMON52109
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 1
Individuals must meet all of the following criteria at screening visits to be
included in the study:
1. At least 18 years of age at screening
2. Must have clinical and pathologic evidence of recurrent C3G or IC-MPGN, as
evidenced by all of the following:
a. A diagnosis of C3G or IC-MPGN, with at least 2+ staining for C3c in the
renal allograft, confirmed by a central pathologist, based on the screening
renal allograft biopsy
b. C3G or IC-MPGN must be primary and not secondary to another condition (eg,
infection, malignancy, monoclonal gammopathy, autoimmunity, chronic
antibody-mediated rejection, chronic thrombotic microangiopathy, or a
medication)
3. Stable (not improving) or worsening disease, in the opinion of the
investigator, in the 2 months preceding the first dose of pegcetacoplan
4. eGFR >=15 mL/min/1.73 m2, calculated by the Chronic Kidney Disease-
Epidemiology Collaboration (CKD-EPI) creatinine equation for adults
5. No more than 50% glomerulosclerosis or interstitial fibrosis on the
screening renal biopsy
6. Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the
screening renal allograft biopsy and from the time of the screening renal
allograft biopsy until randomization
7. Have required vaccinations against N. meningitides, S. pneumoniae, and H.
influenzae(type B) or agree to receive vaccinations if applicable vaccination
records are not available. Vaccination is mandatory unless documented evidence
exists that subjects are nonresponders to vaccination.
8. Women of childbearing potential, defined as any women who have experienced
menarche and who are not permanently sterile or postmenopausal, must have a
negative blood pregnancy test at screening (and negative urine pregnancy at
Visit 4) and must agree to use protocol defined methods of contraception from
screening through 12 weeks after receiving last dose of pegcetacoplan
9. Men must agree to use protocol-defined methods of contraception and agree to
refrain from donating semen from screening through 12 weeks after receiving
last dose of pegcetacoplan
10. Willing and able to provide written informed consent
11. Able to understand and willing to comply with all scheduled procedures and
other requirements of the study in the opinion of the investigator
12. Willing and able to self-administer pegcetacoplan or have an identified
caregiver who can perform the administration
Individuals meeting any of the following criteria at screening or baseline are
ineligible to participate in this study:
1. Absolute neutrophil count <1000 cells/mm3 during screening (not including
Day 1)
2. Previous treatment with pegcetacoplan
3. Evidence of rejection on the screening renal allograft biopsy that requires
treatment
4. Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or
positive serology at screening indicative of infection with any of these
viruses.
5. Weight more than 100 kg at screening
6. Hypersensitivity to pegcetacoplan or any of the excipients
7. History of menigococcal disease
8. Malignancy, except for the following:
a. Cured basal or squamous cell skin cancer
b. Curatively treated in situ disease
c. Malignancy free and off treatment for >=5 years
9. Significant renal disease in the renal allograft secondary to another
condition (eg, infection, malignancy, monoclonal gammopathy, rejection, or a
medication) that would , in the opinion of the investigator, confound
interpretation of the study results
10. Participation in any other investigational drug trial or exposure to other
investigational agent, device, or procedure within 30 days or 5 half-lives from
the last dose of the investigational agent (whichever is longer) prior to
screening period
11. Women who are pregnant, or who are currently breastfeeding
12. Inability to cooperate or any condition that, in the opinion of the
investigator, could increase the subject's risk by participating in the study
or confound the outcome of the study
13. Evidence of drug or alcohol abuse or dependence, in the opinion of the
investigator
14. Known or suspected hereditary fructose intolerance.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary Efficacy Endpoint:<br /><br>The primary efficacy endpoint is the proportion of subjects with reduction in<br /><br>C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary Efficacy endpoints:<br />• The proportion of subjects with reduction in C3c staining on renal biopsy<br />after 52 weeks of treatment<br />• The proportion of subjects with stabilization or improvement in estimated<br />glomerular filtration rate (eGFR), over time<br />• The proportion of subjects with stabilization or improvement in serum<br />creatinine concentration, over time<br />• Changes from baseline biopsy in C3c staining over time<br />• Changes and percentage changes from baseline in eGFR and serum creatinine<br />concentration over time</p>