assignment
RecruitingA Phase 2 Study to Evaluate the Safety and Tolerability of ADX-038 in Participants With Complement-Mediated Kidney Disease
- Trial ID
- 2025-521752-26-00
- Protocol
- ADX-038-201
- Sponsor
- Adarx Pharmaceuticals Inc.
Trial statistics
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test molecules
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research sites
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countries
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diseases
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investigators
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vendors
Diseases & Conditions
Objectives
The primary objective of this phase 2 study is to evaluate the safety and tolerability of ADX-038 in patients diagnosed with complement-mediated kidney disease. The secondary objective is to assess the effect of the investigational product on proteinuria.
Participants
This study involves 38 participants diagnosed with complement-mediated kidney disease. The study population consists of both male and female patients within the age range of 18 to 65 years. Eligible individuals must demonstrate clinical evidence of active kidney disease characterized by a mean urine protein-to-creatinine ratio (UPCR) of ≥0.8 g/g. Participants are required to maintain stable supportive care, including a specific dosing regimen of ACE inhibitors or angiotensin II receptor blockers. Additionally, those receiving renoprotective medications, such as SGLT2 inhibitors or mineralocorticoid antagonists, must have maintained stable doses for at least 90 days. Inclusion requires a mean estimated glomerular filtration rate (eGFR) of ≥30 mL/min/1.73m2. The study population is stratified into cohorts based on specific diagnoses, including IgA nephropathy, C3 glomerulopathy, or immune complex-mediated membranoproliferative glomerulonephritis, all of which must be confirmed via kidney biopsy. The primary objectives are:
- To evaluate the safety of ADX-038.
- To evaluate the tolerability of ADX-038.
Plans and Procedures
This Phase 2 study is designed to evaluate the safety and tolerability of ADX-038 in adults diagnosed with complement-mediated kidney disease. The primary objective is to monitor the incidence and severity of treatment-emergent adverse events. The secondary objectives include assessing changes from baseline in the urinary protein-to-creatinine ratio using both 24-hour urine and spot urine collections. The clinical investigation involves a screening period to confirm eligibility through measures such as estimated glomerular filtration rate, kidney biopsy results, and specific vaccination requirements. Following screening, participants receive ADX-038 via subcutaneous injection. The study includes subsequent visits for monitoring until the end-of-study visit. Total participant involvement is determined by the clinical course and study requirements. Early termination may occur based on investigator judgment or specific clinical conditions.
Treatment
The experimental medication is ADX-038, which is administered in the form of an injection. The study involves the administration of 400 mg/ml via subcutaneous injection.
Efficacy
The efficacy of ADX-038 will be evaluated through the assessment of the urinary protein-to-creatinine ratio (UPCR). This parameter will be measured via 24-hour urine collection and spot urine analysis to determine the change from baseline over time.
Inclusion and Exclusion Criteria
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Inclusion Criteria
- Age ≥18 years at the time of signing informed consent.
- Women must not be breastfeeding.
- Fertile men must agree to use acceptable contraceptive methods if engaged in sexual activity with a partner of childbearing potential (refer to Section 13.2) from the time of signing the ICF until the EOS Visit or 28 days after the last dose of study drug, whichever is longer.
- Fertile men must agree not to donate sperm after study drug administration on Day 1 until the EOS Visit or 28 days after the last dose of study drug, whichever is longer.
- Participants with IgAN (Cohort 1 and Cohort 3): Has a diagnosis of primary IgAN as confirmed by a kidney biopsy performed within 60 months prior to Screening or during Screening. The biopsy report must be reviewed and the diagnosis confirmed by the Investigator and must also show less than 50% global fibrosis and less than 50% glomeruli with cellular crescents.
- Participants with C3G or IC-MPGN (Cohort 2): Has a diagnosis of C3G (including DDD) or IC-MPGN as confirmed by a kidney biopsy performed within 18 months prior to Screening or during Screening. The biopsy report must be reviewed and the diagnosis confirmed by the Investigator and must also show less than 50% global fibrosis and less than 50% glomeruli with cellular crescents.
- Participants with C3G or IC-MPGN (Cohort 2): Participants on systemic corticosteroids or mycophenolic acid derivatives (ie, MMF or MPA), must be on a stable dose for at least 3 months prior to Screening and is expected to remain stable for the duration of the study. The systemic corticosteroid dose must be ≤15 mg/day prednisone or equivalent.
- Has provided written informed consent and any authorizations required by local law and be willing to comply with all study requirements for the duration of the study.
- Has a mean eGFR ≥30 mL/min/1.73m2 (using the creatinine-based the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] formula 2021 for adults) from both Screening Visits (Inker 2021).
- Has clinical evidence of active kidney disease based on mean UPCR ≥0.8 g/g from two 24 hour urine collections within the Screening Period closest to Day 1 attributed to the kidney disease of the cohort in which they are enrolling, per the Investigator’s opinion.
- Has been on supportive care including a stable dosing regimen of ACEi or ARB at the maximally tolerated dose (per Investigator’s judgment and local practice and not exceeding the locally approved maximal daily dose) for at least 30 days before Day 1 and the dosing regimen is expected to remain stable for the duration of the study. Participants with allergies or intolerance to ACEi/ARB are eligible, but the Investigator must document the reason for not taking these medications.
- If taking diuretics, other antihypertensive therapy dose should be stable for 30 days. If taking renoprotective medications (including mineralocorticoid antagonists, SGLT2 inhibitors, GLP-1, sparsentan), the doses should be stable for at least 90 days prior to Day 1 and the dosing regimen is expected to remain stable for the duration of the study.
- The following vaccine requirements need to be completed at least 2 weeks prior to Day 1: a. Completed vaccination schedule for Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria meningitidis serotypes ACWY with appropriate boosters per local guidance. b. Completed at least 2 doses of a 3-dose vaccine series for Neisseria meningitidis serotype B (MenB). The third dose may be administered during the study.
- Participants agree to receive vaccine boosters for MenACWY, MenB, Streptococcus pneumoniae, and Haemophilus influenzae as appropriate per local guidance during the study.
- Women of childbearing potential must have 2 negative serum pregnancy tests during Screening and a negative urine pregnancy test on Day 1 before study drug administration and must agree to use highly effective contraceptive methods if engaged in sexual activity of childbearing potential (refer to Section 13.2) from the time of signing the informed consent form (ICF) until the EOS Visit or 28 days after the last dose of study drug, whichever is longer.
- Participants must have been offered, or unable to take (due to reasons such as intolerance, availability, or patient/physician preference), any approved medications for the disease under study.
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Exclusion Criteria
- Has a known or suspected hereditary or acquired complement deficiency.
- Received a kidney transplant or has received renal replacement therapy for >72 hours consecutively at any time.
- Has a history of a major solid organ transplant (eg, heart, lung, liver) or has received a hematopoetic stem cell/bone marrow transplant.
- Has other significant kidney diseases (outside of cohort in which they are enrolling) that would interfere with interpretation of the study.
- Has presence of rapidly progressive glomerular nephritis or acute kidney injury.
- Has a history of recurrent invasive infections caused by encapsulated bacteria (eg, meningococcus or pneumococcus).
- Has a major concurrent comorbidity, including but not limited to advanced cardiac disease (eg, New York Heart Association class IV) or severe pulmonary disease (eg, severe pulmonary hypertension [World Health Organization class IV]). Any major cardiovascular event in the past year, including a myocardial infarction or a cerebrovascular event requiring hospitalization.
- Has an active malignancy and/or a history of malignancy in the past 5 years, with the exception of completely excised non-melanoma skin cancer or low-grade cervical intraepithelial neoplasia and with no evidence of recurrence for ≥3 years prior to Day 1.
- Has a history of splenectomy.
- Has evidence of monoclonal gammopathy of unclear significance (MGUS), infections, malignancy, autoimmune diseases, or other conditions to which C3G, IC-MPGN, or IgAN is secondary. This includes a diagnosis of Henoch-Schonlein Purpura (IgA vasculitis).
- For IgAN, has taken chronic systemic corticosteroids at any dose (including budesonide) within 3 months prior to Screening, with the exception of corticosteroids used for <7 days for an acute reason >2 weeks prior to Day 1. For C3G or IC MPGN, has taken systemic corticosteroids >15 mg/day prednisone or equivalent for >7 days within 2 weeks prior to Day 1.
- Received complement inhibitor treatments (including eculizumab or ravulizumab) within 6 months prior to Day 1 or are considered to be nonresponders to complement inhibitor treatments.
- Is currently using any systemic immunosuppressant biologics or broad immunosuppressants such as cyclophosphamide, JAK inhibitors, or CNI within 3 months prior to Day 1 with the exception of those permitted (eg, MMF for C3GN or IC MPGN). Use of longer acting biologics such as rituximab or obinutuzumab within 6 months prior to Day 1 is also exclusionary.
- Has a history of active tuberculosis (TB [treated or untreated]), untreated latent TB infection (LTBI), or evidence of active TB during Screening (preferred testing is by Quantiferon when available and per local regulations). Note: Participants with history of treated latent TB are permitted to enroll if they have evidence of completion of treatment and they meet all other eligibility criteria.
- Has an active systemic viral (including COVID-19), bacterial, or fungal infection within 14 days prior to Day 1.
- Has known HIV infection (per participant history and/or medical records) or a positive HIV test during Screening.
- Has a positive serology test for hepatitis B surface antigen (HBsAg), has a prior diagnosis of untreated latent hepatitis B, or positive serology test for hepatitis C virus (HCV) with a detectable RNA concentration during Screening.
- Has liver dysfunction as indicated by any of the following abnormal LFTs during Screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >1.5 × upper limit of normal (ULN). b. Total bilirubin >1.5 × ULN (unless due to Gilbert’s syndrome).
- Has a systolic blood pressure >160 mmHg or a diastolic blood pressure >90 mmHg on Day 1. Optional retest may be performed as needed.
- Has any of the following laboratory parameters during Screening: a. White blood cell count >1.2 × ULN or <0.8 × lower limit of normal (LLN). b. Hemoglobin <9 mg/dL. c. Platelet count <100,000/μL.
- Participated in an interventional drug study within the last 90 days or 5 half-lives, whichever is longer, prior to Screening.
- Donated any blood products (>200 mL) within 30 days prior to Screening.
- Received a blood transfusion within 90 days prior to Screening.
- Received prior treatment with another CFB RNA/DNA-based therapy.
- Has any other significant medical conditions that, in the opinion of the Investigator, would make the participant unsuitable for inclusion in the study, or could interfere with study assessments or put the participant at risk for experiencing significant adverse effects during the study.
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 Italy | Recruiting | 15 Jan 2026 | 3 |
 Spain | Recruiting | 15 Jan 2026 | 4 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
Nimenrix powder and solvent for solution for injection in pre-filled syringe Meningococcal groups A, C, W-135 and Y conjugate vaccine | Other | POWDER AND SOLVENT FOR SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE | INTRAMUSCULAR USE | 0.5 | 12 | PRD6528429 |
Nimenrix powder and solvent for solution for injection in pre-filled syringe Meningococcal groups A, C, W-135 and Y conjugate vaccine | Other | POWDER AND SOLVENT FOR SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE | INTRAMUSCULAR USE | 0.5 | 12 | PRD6532969 |
Infanrix hexa, Powder and suspension for suspension for injection. Diphtheria (D), tetanus (T), pertussis (acellular, component) (Pa), hepatitis B (rDNA) (HBV), poliomyelitis (inactivated) (IPV) and Haemophilus influenzae type b (Hib) conjugate vaccine (adsorbed). | Other | POWDER AND SUSPENSION FOR SUSPENSION FOR INJECTION | INTRAMUSCULAR USE | 0.5 | 1 | PRD5145847 |
Bexsero suspension for injection in pre-filled syringe Meningococcal group B VaccinerDNA, component, adsorbed | Other | SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE | INTRAMUSCULAR USE | 0.5 | 24 | PRD2149122 |
Nimenrix powder and solvent for solution for injection in pre-filled syringe Meningococcal groups A, C, W-135 and Y conjugate vaccine | Other | POWDER AND SOLVENT FOR SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE | INTRAMUSCULAR USE | 0.5 | 12 | PRD6533082 |
Infanrix hexa, Powder and suspension for suspension for injection. Diphtheria (D), tetanus (T), pertussis (acellular, component) (Pa), hepatitis B (rDNA) (HBV), poliomyelitis (inactivated) (IPV) and Haemophilus influenzae type b (Hib) conjugate vaccine (adsorbed). | Other | POWDER AND SUSPENSION FOR SUSPENSION FOR INJECTION | INTRAMUSCULAR USE | 0.5 | 1 | PRD5145848 |
ADX-038 | Test | INJECTION | SUBCUTANEOUS INJECTION | 400 | 12 | PRD12728624 |
Bexsero suspension for injection in pre-filled syringe Meningococcal group B VaccinerDNA, component, adsorbed | Other | SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE | INTRAMUSCULAR USE | 0.5 | 24 | PRD2149126 |
Bexsero suspension for injection in pre-filled syringe Meningococcal group B VaccinerDNA, component, adsorbed | Other | SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE | INTRAMUSCULAR USE | 0.5 | 24 | PRD2149130 |
Infanrix hexa, Powder and suspension for suspension for injection. Diphtheria (D), tetanus (T), pertussis (acellular, component) (Pa), hepatitis B (rDNA) (HBV), poliomyelitis (inactivated) (IPV) and Haemophilus influenzae type b (Hib) conjugate vaccine (adsorbed). | Other | POWDER AND SUSPENSION FOR SUSPENSION FOR INJECTION | INTRAMUSCULAR USE | 0.5 | 1 | PRD344877 |
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Conditions Studied in This Trial
Interventions Studied in This Trial
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HAEMOPHILUS TYPE B POLYSACCHARIDE CONJUGATED TO TETANUS TOXOID ADSORBED ON ALUMINIUM PHOSPHATE
4 trials
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HEPATITIS B SURFACE ANTIGEN (RDNA) ADSORBED ON ALUMINIUM PHOSPHATE [PRODUCED IN S. CEREVISAE CELLS BY RDNA]
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Outer Membrane Vesicles (Omv) From Neisseria Meningitidis Group B Strain Nz98/254 Measured As Amount Of Total Protein Containing The Pora P1.4 Adsorbed On Aluminium Hydroxide
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Pneumococcal Polysaccharide Serotype 11A Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 12F Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 14 Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 15B Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 18C Conjugated To Crm197 Adsorbed On Aluminium Phosphate
16 trials
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Pneumococcal Polysaccharide Serotype 19A Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 19F Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 22F Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 23F Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 3 Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 33F Conjugated To Crm197 Adsorbed On Aluminium Phosphate
10 trials
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Pneumococcal Polysaccharide Serotype 5 Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 6A Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 6B Conjugated To Crm197 Adsorbed On Aluminium Phosphate
16 trials
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Pneumococcal Polysaccharide Serotype 7F Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 8 Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Pneumococcal Polysaccharide Serotype 9V Conjugated To Crm197 Adsorbed On Aluminium Phosphate
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Recombinant Neisseria Meningitidis Group B Fhbp Fusion Protein Produced In E. Coli Cells By Recombinant Dna Technology Adsorbed On Aluminium Hydroxide
12 trials
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Recombinant Neisseria Meningitidis Group B Nada Protein Produced In E. Coli Cells By Recombinant Dna Technology Adsorbed On Aluminium Hydroxide
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Recombinant Neisseria Meningitidis Group B Nhba Fusion Protein Produced In E. Coli Cells By Recombinant Dna Technology Adsorbed On Aluminium Hydroxide
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TETANUS TOXOID
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ADX-038
2 trials