Immunoglobulin A (IgA) NephropathyPhase 1/2NCT07182227
PS-002 for the Treatment of IgA Nephropathy in Adults
A Phase 1/2 study testing PS-002 in people with Immunoglobulin A (IgA) Nephropathy, run by Purespring Therapeutics Limited at 10 sites in 2 countries.
Trial summary
The purpose of the study is to evaluate safety, tolerability, and preliminary effectiveness following administration of PS-002 in adults with primary Immunoglobulin A (IgA) nephropathy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immunoglobulin A (IgA) Nephropathy
- What is being tested?
- PS-002
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 30 Mar 2026to Sep 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary IgA nephropathy (IgAN) as evidenced by renal biopsy.
- A historic kidney biopsy performed within 36 months prior to screening with reported evidence of complement component 3 (C3) deposition. If the participant had a kidney biopsy performed over 36 months prior to Screening, a new kidney biopsy should be carried out during the Screening period. This biopsy must exhibit signs of ongoing complement system activity.
- Proteinuria assessed during screening as UPCR greater than or equal to 0.75 g/g (greater than or equal to 750 mg/g) OR total protein excretion greater than or equal to 1 g/24 h (greater than or equal to 1000 mg/24h) sampled from 24 h urine collection.
- eGFR calculated using the CKD-EPI formula greater than or equal to 30 mL/min/1.73m^2.
- Sitting office systolic blood pressure equal to or less than 140 mmHg, diastolic blood pressure equal to or less than 90 mmHg.
- All participants must have been on best supportive care for IgAN, as per region-specific requirements defined in the protocol.
Who cannot join
- A participant has nephrotic syndrome, defined for this purpose as 24 h urine protein greater than 3.5g with concurrent hypoalbuminemia (serum albumin less than 3.0 g/dL [less than 30 g/L]).
- Any secondary IgAN defined as associated with gastrointestinal and liver disorders (liver cirrhosis, celiac disease, Crohn's disease, ulcerative colitis), autoimmune disorders (dermatitis herpetiformis, psoriasis, seronegative arthritis, systemic lupus erythematosus, rheumatoid arthritis), malignancy (IgA myeloma, lymphoma, lung cancer, renal cell cancer, cutaneous T-cell lymphoma), respiratory disorders (bronchiolitis obliterans, idiopathic pulmonary fibrosis) etc.
- Having a major concurrent non-IgAN-related disease that, in the opinion of the investigator, prevents the assessment of IgAN.
- History of malignancy; or bone marrow or organ transplant.
- History of, or currently active primary or secondary immunodeficiency, including known history of human immunodeficiency virus infection, and other severe immunodeficiency blood disorders.
- Presence of other significant medical conditions that would create an unacceptable procedure or anesthesia risk.
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- Aspartate aminotransferase or alanine aminotransferase greater than 1.5 times the upper limit of normal.
- History of serious infection requiring parenteral antibiotics within the past 8 weeks prior to study drug administration.
- Participants previously treated with immunosuppressive/immunomodulatory agents such as, but not limited to, cyclophosphamide, infliximab, complement inhibitor, canakinumab, mycophenolate mofetil, mycophenolate sodium, cyclosporine, tacrolimus, everolimus, or systemic corticosteroids (exposure greater than 7.5mg/day prednisone/prednisolone equivalent) within 90 days (or 180 days for rituximab) prior to Screening. Participants previously or currently receiving oral budesonide (Kinpeygo/Tarpeyo) require wash out for 90 days prior to the study drug administration.
- Exposed to a live or attenuated vaccine within the 6 weeks prior to study drug administration.
- Participants with a known sensitivity or intolerance to corticosteroid therapy.
- Known hypersensitivity to study drug ingredients.
- Prior treatment with PS-002 or any other gene therapy, or participation in any other investigational trial during this study.
- Positive serology for hepatitis B or C, i.e., positive hepatitis B surface antigen or hepatitis C ribonucleic acid (RNA) viral load positive.
- Participants treated with potentially hepatotoxic medications unless they have been monitored in accordance with the drug label and have received a stable dose since >90 days prior to dosing without clinically significant liver enzyme fluctuations.
- Note: Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
10 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
10 sites in 2 countries
United Kingdom8 sites, 8 recruiting
- Recruiting
Southmead Hospital
Bristol, BS10 5NB
- Not yet recruiting
Cardiff and Vale University Health Board
Cardiff, CF14 4XW
- Recruiting
Royal Infirmary of Edinburgh Clinical Research Facility
Edinburgh, EH16 4SA
- Not yet recruiting
Leicester General Hospital
Leicester, Leicestershire, LE5 4PW
- Recruiting
The Royal London Hospital
London, E1 1FR
- Recruiting
Manchester University NHS Foundation Trust
Manchester, Greater Manchester, M13 9WL
- Recruiting
Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Manchester, Greater Manchester, M13 9WL
- Recruiting
Nottingham University Hospitals NHS Trust
Nottingham, Nottinghamshire, NG5 1PB
United States2 sites, 2 recruiting
- Recruiting
The Johns Hopkins Hospital
Baltimore, Maryland, 21287
- Recruiting
University of Miami Hospital
Miami, Florida, 33136
About this study
Official title: A Phase 1/2 Multicenter, Open Label, Two-part Study (Single Ascending Dose [Part 1], and Dose Expansion [Part 2]) to Evaluate Safety, Tolerability and Efficacy of PS-002, a Gene Therapy for the Treatment of Adult Participants With Primary IgA Nephropathy
The purpose of the study is to evaluate safety, tolerability, and preliminary effectiveness following administration of PS-002 in adults with primary Immunoglobulin A (IgA) nephropathy. This will be a first-in-human study and will include participants at high risk of disease progression despite receiving current standard-of-care treatment.
Participants will be monitored for up to one year after receiving PS-002 and invited to take part in a long-term follow-up study (total follow-up: 5 years).
- Conditions
- Immunoglobulin A (IgA) Nephropathy
- Being tested
- PS-002
- Keywords
- Glomerulonephritis, Nephritis, Kidney Diseases, Autoimmune Diseases, Immune System Diseases, IGA
- Medical subject headings
- Kidney Diseases, Glomerulonephritis, Nephritis, Autoimmune Diseases, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PS-002Genetic
Adeno-associated viral vector containing the human Complement Factor I (CFI) gene
| Study group | Role | What they receive |
|---|---|---|
| PS-002 | Experimental | Part 1: Dose escalation in three groups: Group 1: Low dose, Group 2: Intermediate Dose, Group 3: High Dose.
Part 2: Dose expansion in a fourth group with a selected dose. Receives: Genetic: PS-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with: Treatment-Emergent Adverse Events (TEAEs) and serious TEAEs, TEAEs and serious TEAEs related to PS-002, TEAEs and serious TEAEs related to the PS-002 administration procedure
Measured: Screening up to Week 48
Secondary outcomes (7)
Change in proteinuria as measured by Urine Protein:Creatinine Ratio (UPCR) derived from 24 hr urine collection
Measured: Baseline to Week 36
Change from baseline in proteinuria as measured by urine protein creatinine ratio (UPCR) from spot urine collection. [UPCR (g/g) will be calculated from spot urine collection (first morning void)]
Measured: Baseline to Week 48
Change from baseline in proteinuria as measured by urine albumin:creatinine ratio (UACR). [UACR (g/g) will be calculated from spot urine collection (first morning void)]
Measured: Baseline to Week 48
Change from baseline in proteinuria as measured by UACR from 24 hour urine collection. [UACR (mg/day) will be calculated from 24 hour urine collection at Week 36 only]
Measured: Baseline to Week 36
Change from baseline in creatinine and estimated Glomerular Filtration Rate (eGFR) values calculated using the Chronic Kidney Disease Epidemiology (CKD-EPI) creatinine formula
Measured: Baseline to Week 48
Change from baseline in urinary soluble terminal complement complex (sC5b-9) levels
Measured: Baseline to Week 48
Time to worsening of kidney function. Defined as: 1) sustained eGFR decline by at least 40 percent, 2) onset of end-stage kidney disease, 3) initiation of renal replacement therapy, or 4) all-cause mortality
Measured: Baseline to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2025
- Study start30 Mar 2026
- Primary completionSep 2028 (estimated)
- Study completionSep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immunoglobulin A (IgA) NephropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Part 1: In the dose exploration of the study, 3 different ascending dose levels will be administered using a sentinel dosing strategy Part 2: Participants will receive the optimal biological dose identified in Part 1 of the study
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Purespring Therapeutics Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07182227
- Sponsor's protocol ID
- PS-002-101
- Other IDs
- 2025-523201-14-00, 2025
- Status last verified
- Sep 2026
- First submitted
- 11 Sep 2025
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: IPD data are not planned to be shared at this time due to patient privacy concerns with consideration of the small sample size for this open-label study
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Multicenter, Open Label, Two-part Study (Single Ascending Dose [Part 1], and Dose Expansion [Part 2]) to Evaluate Safety, Tolerability and Efficacy of PS-002, a Gene Therapy for the Treatment of Adult Participants With Primary IgA Nephropathy
- ClinicalTrials.gov ID
- NCT07182227
- Acronym / sponsor ID
- PS-002-101
- Conditions
- Immunoglobulin A (IgA) Nephropathy
- Keywords
- Glomerulonephritis, Nephritis, Kidney Diseases, Autoimmune Diseases, Immune System Diseases, IGA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Purespring Therapeutics LimitedIndustry (pharma, biotech, devices)
- Study start
- 30 Mar 2026 (actual)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Sep 2029 (estimated)
- First posted
- 19 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate safety, tolerability, and preliminary effectiveness following administration of PS-002 in adults with primary Immunoglobulin A (IgA) nephropathy. This will be a first-in-human study and will include participants at high risk of disease progression despite receiving current standard-of-care treatment.
Participants will be monitored for up to one year after receiving PS-002 and invited to take part in a long-term follow-up study (total follow-up: 5 years).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Part 1: In the dose exploration of the study, 3 different ascending dose levels will be administered using a sentinel dosing strategy Part 2: Participants will receive the optimal biological dose identified in Part 1 of the study
| Arm | Type | Receives |
|---|---|---|
| PS-002 | Experimental | Genetic: PS-002Part 1: Dose escalation in three groups: Group 1: Low dose, Group 2: Intermediate Dose, Group 3: High Dose. Part 2: Dose expansion in a fourth group with a selected dose. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of primary IgA nephropathy (IgAN) as evidenced by renal biopsy.
- A historic kidney biopsy performed within 36 months prior to screening with reported evidence of complement component 3 (C3) deposition. If the participant had a kidney biopsy performed over 36 months prior to Screening, a new kidney biopsy should be carried out during the Screening period. This biopsy must exhibit signs of ongoing complement system activity.
- Proteinuria assessed during screening as UPCR greater than or equal to 0.75 g/g (greater than or equal to 750 mg/g) OR total protein excretion greater than or equal to 1 g/24 h (greater than or equal to 1000 mg/24h) sampled from 24 h urine collection.
- eGFR calculated using the CKD-EPI formula greater than or equal to 30 mL/min/1.73m^2.
- Sitting office systolic blood pressure equal to or less than 140 mmHg, diastolic blood pressure equal to or less than 90 mmHg.
Key exclusion criteria (16 listed)
- A participant has nephrotic syndrome, defined for this purpose as 24 h urine protein greater than 3.5g with concurrent hypoalbuminemia (serum albumin less than 3.0 g/dL [less than 30 g/L]).
- Any secondary IgAN defined as associated with gastrointestinal and liver disorders (liver cirrhosis, celiac disease, Crohn's disease, ulcerative colitis), autoimmune disorders (dermatitis herpetiformis, psoriasis, seronegative arthritis, systemic lupus erythematosus, rheumatoid arthritis), malignancy (IgA myeloma, lymphoma, lung cancer, renal cell cancer, cutaneous T-cell lymphoma), respiratory disorders (bronchiolitis obliterans, idiopathic pulmonary fibrosis) etc.
- Having a major concurrent non-IgAN-related disease that, in the opinion of the investigator, prevents the assessment of IgAN.
- History of malignancy; or bone marrow or organ transplant.
- History of, or currently active primary or secondary immunodeficiency, including known history of human immunodeficiency virus infection, and other severe immunodeficiency blood disorders.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PS-002Adeno-associated viral vector containing the human Complement Factor I (CFI) gene | Genetic | PS-002 |
Primary outcome
Number of participants with: Treatment-Emergent Adverse Events (TEAEs) and serious TEAEs, TEAEs and serious TEAEs related to PS-002, TEAEs and serious TEAEs related to the PS-002 administration procedure
Time frame: Screening up to Week 48
7 secondary outcomes
Change in proteinuria as measured by Urine Protein:Creatinine Ratio (UPCR) derived from 24 hr urine collection
Time frame: Baseline to Week 36
Change from baseline in proteinuria as measured by urine protein creatinine ratio (UPCR) from spot urine collection. [UPCR (g/g) will be calculated from spot urine collection (first morning void)]
Time frame: Baseline to Week 48
Change from baseline in proteinuria as measured by urine albumin:creatinine ratio (UACR). [UACR (g/g) will be calculated from spot urine collection (first morning void)]
Time frame: Baseline to Week 48
Change from baseline in proteinuria as measured by UACR from 24 hour urine collection. [UACR (mg/day) will be calculated from 24 hour urine collection at Week 36 only]
Time frame: Baseline to Week 36
Change from baseline in creatinine and estimated Glomerular Filtration Rate (eGFR) values calculated using the Chronic Kidney Disease Epidemiology (CKD-EPI) creatinine formula
Time frame: Baseline to Week 48
Change from baseline in urinary soluble terminal complement complex (sC5b-9) levels
Time frame: Baseline to Week 48
Time to worsening of kidney function. Defined as: 1) sustained eGFR decline by at least 40 percent, 2) onset of end-stage kidney disease, 3) initiation of renal replacement therapy, or 4) all-cause mortality
Time frame: Baseline to Week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2026.
- Individual participant dataSharing planned: No. IPD data are not planned to be shared at this time due to patient privacy concerns with consideration of the small sample size for this open-label study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PS-002 in people with Immunoglobulin A (IgA) Nephropathy, run by Purespring Therapeutics Limited at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United Kingdom, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Operations (+44 (0)20 3855 6324, contact@purespringtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with: Treatment-Emergent Adverse Events (TEAEs) and serious TEAEs, TEAEs and serious TEAEs related to PS-002, TEAEs and serious TEAEs related to the PS-002 administration procedure (time frame: Screening up to Week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026.