Primary HyperoxaluriaPhase 3ILLUMINATE-BNCT03905694
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
A Phase 3 study testing Lumasiran in people with Primary Hyperoxaluria and Primary Hyperoxaluria Type 1 (PH1), run by Alnylam Pharmaceuticals at 9 sites in 5 countries.
Trial summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1). Read more about this study
- Who is it for?
- Ages 0 to 5 years
- Conditions the study is for
- Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
- What is being tested?
- Lumasiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 22 Apr 2019to 26 Jul 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 0 to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
- Meets urinary oxalate excretion requirements
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Who cannot join
- If <12 months old at screening, has an abnormally high serum creatinine
- If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m^2
- Clinical evidence of systemic oxalosis
- History of kidney or liver transplant
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
Israel3 sites
Clinical Trial Site
Haifa
Clinical Trial Site
Jerusalem
Clinical Trial Site
Nahariya
United States2 sites
Clinical Trial Site
Houston, Texas, 77030
Clinical Trial Site
Rochester, Minnesota, 55905
About this study
Official title: ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Being tested
- Lumasiran
- Keywords
- PH1, Primary Hyperoxaluria, Hyperoxaluria, RNAi Therapeutic, siRNA, AGT
- Medical subject headings
- Hyperoxaluria, Primary, Primary hyperoxaluria type 1, Hyperoxaluria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LumasiranDrug
Also called ALN-GO1
Lumasiran will be administered by subcutaneous (SC) injection.
| Study group | Role | What they receive |
|---|---|---|
| Lumasiran | Experimental | Lumasiran will be administered by subcutaneous (SC) injection. Receives: Drug: Lumasiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
Measured: Baseline to Month 6
Secondary outcomes (17)
Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60])
A negative change from baseline indicates a favorable outcome.
Measured: From Month 6 to Month 60
Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age)
Percentage of time that spot urinary oxalate: creatinine (UOx:Cr) ratio level is at or below ≤ 1.5xULN is calculated as (cumulative months at or below near normalization threshold divided by cumulative months of assessments)*100. Cumulative months in near-normalization is defined as the summation across all intervals that met the near-normal threshold and cumulative months of valid assessments is defined as the summation across all valid post-baseline collections. ULN levels of spot urinary oxalate to creatinine ratio where urine oxalate levels were analyzed using enzymatic assay. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
Measured: Up to 60 months
Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline
The absolute change is represented as ratio of millimoles of urinary oxalate to millimoles of urinary creatinine.
Measured: From Baseline to Month 6 and Month 60
Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age
The percentage of participants meeting the criteria (UOx:Cr ratio ≤ the ULN for age) at least at one post-baseline visit were reported for this outcome measure. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
Measured: Up to 60 months
Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age
The percentage of participants meeting the criteria (UOx:Cr ratio ≤ 1.5xULN for age) at least at one post-baseline visit were reported for this outcome measure. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
Measured: Up to 60 months
Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)
The lower limit of quantification (LLOQ) was 5.55 micromoles per liter (μmol/L). A negative change from baseline indicates a favorable outcome.
Measured: From Baseline to Month 6 and Month 60
Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)
The LLOQ was 5.55 μmol/L. A negative change from Baseline indicates a favorable outcome.
Measured: From Baseline to Month 6 and Month 60
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
Cmax was the maximum plasma concentration post-dose within the pharmacokinetic (PK) sampling time frame. Higher Cmax generally indicates higher drug exposure.
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran
Tmax was estimated by calculating the time required to reach the maximum plasma concentration (Cmax) after the drug administration. Lower Tmax generally indicates faster drug absorption from the administration site.
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Elimination Half-life (t1/2beta) of Lumasiran
Elimination half-life was estimated from the terminal phase of the plasma concentration-time profile post-dose. Shorter half-life generally indicates rapid drug elimination from the body.
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to 24 Hours (AUC0-24) of Lumasiran
AUC0-24 was the total drug exposure calculated as the area under the plasma concentration-time curve from the time of dosing (t = 0) to 24 hours.
Measured: 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to Last Quantifiable Concentration (AUC0-last) of Lumasiran
AUC0-last was the total drug exposure calculated as the area under the plasma concentration-time curve from time 0 to the time of the last measurable (quantifiable) concentration (C_last).
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to Infinity (AUC0-infinity) of Lumasiran
AUC0-infinity was the total drug exposure estimated as the area under the plasma concentration-time curve from time 0 extrapolated to infinity.
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Apparent Clearance (CL/F) of Lumasiran
Apparent clearance was calculated by dividing the area under the plasma concentration-time curve from zero infinity by the dose administered. A higher clearance generally indicates faster elimination from the body.
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Apparent Volume of Distribution (V/F) of Lumasiran
Apparent Volume of Distribution generally indicates the extent of drug distribution in the body.
Measured: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
eGFR [in milliliters per minute per 1.73 meters square (mL/min/1.73m^2)] was calculated from serum creatinine (SCr) based on the Schwartz Bedside Formula for participants ≥12 months of age at the time of assessment.
Measured: From Baseline to Month 6 and Month 60
Number of Participants With Adverse Events (AEs)
An Adverse Event (AE) is any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Measured: Up to 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2019
- Study start22 Apr 2019
- Primary completion29 Jun 2020
- Study completion26 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Primary HyperoxaluriaStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03905694
- Sponsor's protocol ID
- ALN-GO1-004
- Other IDs
- 2018-004014-17, 2018
- Acronym
- ILLUMINATE-B
- Status last verified
- Jan 2025
- First submitted
- 1 Apr 2019
- Last updated
- 14 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU). Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
Publications
Frishberg Y, Hayes W, Shasha-Lavsky H, Sas DJ, Michael M, Sellier-Leclerc AL, Hogan J, Willey R, Gansner JM, Magen D. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial. Front Pediatr. 2024 Sep 16;12:1392644. doi: 10.3389/fped.2024.1392644. eCollection 2024.
PubMed 39355649Sankar A, Y S RK, Singh A, Roy R, Shukla R, Verma B. Next-generation therapeutics for rare genetic disorders. Mutagenesis. 2024 Apr 24;39(3):157-171. doi: 10.1093/mutage/geae002.
PubMed 38332115Hayes W, Sas DJ, Magen D, Shasha-Lavsky H, Michael M, Sellier-Leclerc AL, Hogan J, Ngo T, Sweetser MT, Gansner JM, McGregor TL, Frishberg Y. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. Pediatr Nephrol. 2023 Apr;38(4):1075-1086. doi: 10.1007/s00467-022-05684-1. Epub 2022 Aug 1.
PubMed 35913563Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children. Genet Med. 2022 Mar;24(3):654-662. doi: 10.1016/j.gim.2021.10.024. Epub 2021 Dec 8.
PubMed 34906487
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
- ClinicalTrials.gov ID
- NCT03905694
- Acronym / sponsor ID
- ILLUMINATE-B / ALN-GO1-004
- Keywords
- PH1, Primary Hyperoxaluria, Hyperoxaluria, RNAi Therapeutic, siRNA, AGT
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 22 Apr 2019 (actual)
- Primary completion
- 29 Jun 2020 (actual)
- Study completion
- 26 Jul 2024 (actual)
- First posted
- 5 Apr 2019 (actual)
- Results
- Posted 19 Jul 2021
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 this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lumasiran | Experimental | Drug: LumasiranLumasiran will be administered by subcutaneous (SC) injection. |
Who may qualify
- Ages
- 0 to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
- Meets urinary oxalate excretion requirements
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Key exclusion criteria (4 listed)
- If <12 months old at screening, has an abnormally high serum creatinine
- If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m^2
- Clinical evidence of systemic oxalosis
- History of kidney or liver transplant
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 |
|---|---|---|
| LumasiranAlso: ALN-GO1Lumasiran will be administered by subcutaneous (SC) injection. | Drug | Lumasiran |
Primary outcome
Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Time frame: Baseline to Month 6
Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
17 secondary outcomes
Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60])
Time frame: From Month 6 to Month 60
Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age)
Time frame: Up to 60 months
Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline
Time frame: From Baseline to Month 6 and Month 60
Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age
Time frame: Up to 60 months
Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age
Time frame: Up to 60 months
Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Time frame: From Baseline to Month 6 and Month 60
Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Time frame: From Baseline to Month 6 and Month 60
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Elimination Half-life (t1/2beta) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to 24 Hours (AUC0-24) of Lumasiran
Time frame: 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to Last Quantifiable Concentration (AUC0-last) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to Infinity (AUC0-infinity) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Apparent Clearance (CL/F) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Apparent Volume of Distribution (V/F) of Lumasiran
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time frame: From Baseline to Month 6 and Month 60
Number of Participants With Adverse Events (AEs)
Time frame: Up to 60 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39355649Frishberg Y, Hayes W, Shasha-Lavsky H, Sas DJ, Michael M, Sellier-Leclerc AL, Hogan J, Willey R, Gansner JM, Magen D. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial. Front Pediatr. 2024 Sep 16;12:1392644. doi: 10.3389/fped.2024.1392644. eCollection 2024.
- Derived from this study: PubMed 38332115Sankar A, Y S RK, Singh A, Roy R, Shukla R, Verma B. Next-generation therapeutics for rare genetic disorders. Mutagenesis. 2024 Apr 24;39(3):157-171. doi: 10.1093/mutage/geae002.
- Derived from this study: PubMed 35913563Hayes W, Sas DJ, Magen D, Shasha-Lavsky H, Michael M, Sellier-Leclerc AL, Hogan J, Ngo T, Sweetser MT, Gansner JM, McGregor TL, Frishberg Y. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. Pediatr Nephrol. 2023 Apr;38(4):1075-1086. doi: 10.1007/s00467-022-05684-1. Epub 2022 Aug 1.
- Derived from this study: PubMed 34906487Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children. Genet Med. 2022 Mar;24(3):654-662. doi: 10.1016/j.gim.2021.10.024. Epub 2021 Dec 8.
- Individual participant dataSharing planned: Yes. Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU). Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
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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. This study is not recruiting at the moment.
Medical Director study director
Alnylam Pharmaceuticals
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lumasiran in people with Primary Hyperoxaluria and Primary Hyperoxaluria Type 1 (PH1), run by Alnylam Pharmaceuticals at 9 sites in 5 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 3 inclusion and 4 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: finished, as of the sponsor's last update on 14 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Israel, United States, France, Germany, United Kingdom. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Jul 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 (time frame: Baseline to Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03905694, which also lists 4 linked publications.
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 14 Feb 2025; AzTrials last refreshed its copy on 26 Oct 2026.