Quality of LifeNCT04002531
A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children
A study testing General and Neurological examination, Vital signs and 12 lead electrocardiogram in people with Quality of Life and Renal Insufficiency, run by Baylor Research Institute at 1 site in United States.
Trial summary
The objective of this study is to obtain follow up data on a cohort of well-studied patients with Fabry disease who have been on ERT since childhood for a total of about 15 years. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Quality of Life, Renal Insufficiency, Cardiac Event
- What is being tested?
- General and Neurological examination, Vital signs and 12 lead electrocardiogram
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Nov 2018to 13 Dec 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
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
- Patients who participated in TKT029 and who are willing and able to come to Dallas for 1 visit for standard of care testing.
- Sign the protocol informed consent form
- Have been on continuous commercial ERT since TKT029 has ended
Who cannot join
- Patients who are unable to understand the nature, scope, and possible consequences of the study.
- Patient does not give his written informed consent to participate in this study
- Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures.
- Patient has been off ERT for an extended period of time as assessed by the investigator.
Trial locations and contacts
1 site in 1 country.
United States1 site
Baylor University Medical Center
Dallas, Texas, 75246
About this study
The objective of this study is to obtain follow up data on a cohort of well-studied patients with Fabry disease who have been on ERT since childhood for a total of about 15 years.
Read the detailed description
The long-term effect of initiating ERT in childhood is unknown. Prospective studies of Children with Fabry disease on 0.2 mg/kg agalsidase alfa every other week were performed. The patients were 7-17 years of age at initial study enrollment. The first open-label protocol was TKT023, a 6 months study (August 12, 2002-October 20, 2004) that was followed by an extension study TKT029 (June 10, 2004-June 15, 2011; ClinicalTrials.gov identifier NCT00084084). Since completing TKT029, all US patients were switched to commercial agalsidase beta. Therefore, these patients have now been treated for about 15 years.This study involves a one-visit follow up on these patients using the same protocol-driven studies as were used in TKT029. The long-term follow up data gathered will consist of a rare description of the disease profile in patients who were treated with ERT since childhood.
- Conditions
- Quality of Life, Renal Insufficiency, Cardiac Event
- Being tested
- General and Neurological examination, Vital signs, 12 lead electrocardiogram, Echocardiogram, Blood draw
- Medical subject headings
- Renal Insufficiency, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
General and Neurological examinationOther
Also called MD assessment
Information about your general health, neurological symptoms and current medications with be collected
Vital signsOther
Also called Blood pressure, heart rate, respiratory rate
Height, weight, blood pressure, heart rate, and respiratory rate and temperature will be measured.
12 lead electrocardiogramProcedure or surgery
Also called EKG, ECG
A non-invasive test that measures the electrical activity of the heart
EchocardiogramProcedure or surgery
Also called Cardiac echo
A non-invasive sonogram of the heart
Blood drawProcedure or surgery
Also called Blood collection, phlebotomy, lab test
Blood will be drawn to evaluate general health and renal function (kidney health)
Urine collectionProcedure or surgery
Urine will be collection to evaluate renal function (kidney health)
2-hour Holter MonitorProcedure or surgery
Also called Holter
A non-invasive test that measures the electrical activity of the heart continuously over 2 hours
Brief Pain Inventory questionnaireOther
Also called BPI, Pain questionnaire
A questionnaire about daily pain
Quality of Life questionnaireOther
Also called SF 36
A questionnaire about the impact of disease on their activities of daily living and quality of life
| Study group | Role | What they receive |
|---|---|---|
| Single Visit | Other | 1. General and neurological examination
2. Vital signs including height, weight, blood pressure, pulse, temperature
3. 12 lead ECG
4. 2 hour Holter monitor for heart rate variability
5. Echocardiogram
6. Renal function will be assessed by the eGFR. The eGFR will be calculated from serum creatinine using CKD-EPI equation.
7. CBC with differential
8. Complete metabolic panel
9. Urinalysis
10. Urine Albumin/creatinine ratio.
11. Urine and plasma samples for biomarkers (Gb3, lyso-Gb3) that will be stored in -80 freezer and assayed in our lab.
12. Brief Pain Inventory questionnaire.
13. Quality of Life Questionnaires (SF36) Receives: Other: General and Neurological examination; Other: Vital signs; Procedure: 12 lead electrocardiogram; Procedure: Echocardiogram; Procedure: Blood draw; Procedure: Urine collection; Procedure: 2-hour Holter Monitor; Other: Brief Pain Inventory questionnaire; Other: Quality of Life questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
estimated Glomerular Filtration Rate (eGFR)
Change in eGFR since previous participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" - NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Secondary outcomes (8)
Left Ventricular Mass Index
LVMI measured in g/m2 by echocardiogram and compared to LVMI results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Heart rate variability assessment
2 hour holter monitor and compared to holter monitor results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Urine albumin/creatinine ratio
Biomarker of renal function and compared to urine albumin/creatinine ratios obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Plasma Lyso-Gb3
Biomarker of disease and compared to plasma Lyso-Gb3 results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Plasma Gb3 and compared to plasma Gb3 results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Biomarker of disease
Measured: Study involves one visit only - assessed Baseline Visit
Urine Lyso-Gb3
Biomarker of disease and compared to urine Lyso-Gb3 results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Short-form Brief Pain Inventory (BPI)
Questionnaire designed to assess current level of pain from 0-10. 0 reflects no pain and 10 being the worst possible pain. Results will be compared to pediatric pain assessments obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Measured: Study involves one visit only - assessed Baseline Visit
Qualify of Life - Your Health and Well-being
Self-evaluation that describes current physical and emotional health. Questionnaire asks User to rate how Fabry disease impacts User's overall physical and emotional well-being. Questionnaire uses multiple scales to rate User's ability to perform activities of daily life, identify changes in overall health, and identify how changes in physical health and disease has impacted User's emotional well-being. User will be asked to answer multiple questions using the following scales: Poor/Fair/Good/Very good/excellent, Much better than 1 week ago/Somewhat better than 1 week ago/The same as 1 week ago/Somewhat worse than 1 week ago/Much worse than 1 week ago, Limited a lot/Limited a little/Not limited at all, All of the time/Most of the time/Some of the time/A little of the time/None of the time, Not at all/Slightly/Moderately/Quite a bit/Extremely, None/Very mild/Mild/Moderate/Severe/Very severe, Definitely true/Mostly true/Don't know/Mostly false/Definitely false.
Measured: Study involves one visit only - assessed Baseline Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2019
- Study start10 Nov 2018
- Primary completion13 Dec 2019
- Study completion13 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Quality of LifeStudies of the same condition, open and under way.
- More trials run by Baylor Research InstituteThe 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 N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Specific group of adults with Fabry disease who received Replagal infusions as children
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Shire
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04002531
- Sponsor's protocol ID
- 018-706
- Status last verified
- Jul 2020
- First submitted
- 25 Mar 2019
- Last updated
- 4 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Schiffmann R. Fabry disease. Pharmacol Ther. 2009 Apr;122(1):65-77. doi: 10.1016/j.pharmthera.2009.01.003. Epub 2009 Feb 8.
PubMed 19318041Schiffmann R, Ries M. Fabry Disease: A Disorder of Childhood Onset. Pediatr Neurol. 2016 Nov;64:10-20. doi: 10.1016/j.pediatrneurol.2016.07.001. Epub 2016 Jul 29.
PubMed 27555236Echevarria L, Benistan K, Toussaint A, Dubourg O, Hagege AA, Eladari D, Jabbour F, Beldjord C, De Mazancourt P, Germain DP. X-chromosome inactivation in female patients with Fabry disease. Clin Genet. 2016 Jan;89(1):44-54. doi: 10.1111/cge.12613. Epub 2015 Jun 22.
PubMed 25974833Dobyns WB. The pattern of inheritance of X-linked traits is not dominant or recessive, just X-linked. Acta Paediatr Suppl. 2006 Apr;95(451):11-5. doi: 10.1111/j.1651-2227.2006.tb02383.x.
PubMed 16720459MacDermot KD, Holmes A, Miners AH. Natural history of Fabry disease in affected males and obligate carrier females. J Inherit Metab Dis. 2001;24 Suppl 2:13-4; discussion 11-2. doi: 10.1023/a:1012447102358. No abstract available.
PubMed 11758673Schiffmann R, Warnock DG, Banikazemi M, Bultas J, Linthorst GE, Packman S, Sorensen SA, Wilcox WR, Desnick RJ. Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy. Nephrol Dial Transplant. 2009 Jul;24(7):2102-11. doi: 10.1093/ndt/gfp031. Epub 2009 Feb 13.
PubMed 19218538Kwon JM, Matern D, Kurtzberg J, Wrabetz L, Gelb MH, Wenger DA, Ficicioglu C, Waldman AT, Burton BK, Hopkins PV, Orsini JJ. Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease. Orphanet J Rare Dis. 2018 Feb 1;13(1):30. doi: 10.1186/s13023-018-0766-x.
PubMed 29391017Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA. 1999 Jan 20;281(3):249-54. doi: 10.1001/jama.281.3.249.
PubMed 9918480Schiffmann R, Hughes DA, Linthorst GE, Ortiz A, Svarstad E, Warnock DG, West ML, Wanner C; Conference Participants. Screening, diagnosis, and management of patients with Fabry disease: conclusions from a "Kidney Disease: Improving Global Outcomes" (KDIGO) Controversies Conference. Kidney Int. 2017 Feb;91(2):284-293. doi: 10.1016/j.kint.2016.10.004. Epub 2016 Dec 18.
PubMed 27998644Schiffmann R, Pastores GM, Lien YH, Castaneda V, Chang P, Martin R, Wijatyk A. Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study. Orphanet J Rare Dis. 2014 Nov 26;9:169. doi: 10.1186/s13023-014-0169-6.
PubMed 25425121
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children
- ClinicalTrials.gov ID
- NCT04002531
- Acronym / sponsor ID
- 018-706
- Conditions
- Quality of Life, Renal Insufficiency, Cardiac Event
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Baylor Research InstituteUniversity, hospital or other
- Collaborators
- Shire
- Study start
- 10 Nov 2018 (actual)
- Primary completion
- 13 Dec 2019 (actual)
- Study completion
- 13 Dec 2019 (actual)
- First posted
- 28 Jun 2019 (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 objective of this study is to obtain follow up data on a cohort of well-studied patients with Fabry disease who have been on ERT since childhood for a total of about 15 years.
Detailed description
The long-term effect of initiating ERT in childhood is unknown. Prospective studies of Children with Fabry disease on 0.2 mg/kg agalsidase alfa every other week were performed. The patients were 7-17 years of age at initial study enrollment. The first open-label protocol was TKT023, a 6 months study (August 12, 2002-October 20, 2004) that was followed by an extension study TKT029 (June 10, 2004-June 15, 2011; ClinicalTrials.gov identifier NCT00084084). Since completing TKT029, all US patients were switched to commercial agalsidase beta. Therefore, these patients have now been treated for about 15 years.This study involves a one-visit follow up on these patients using the same protocol-driven studies as were used in TKT029. The long-term follow up data gathered will consist of a rare description of the disease profile in patients who were treated with ERT since childhood.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Specific group of adults with Fabry disease who received Replagal infusions as children
| Arm | Type | Receives |
|---|---|---|
| Single Visit | Other | Other: General and Neurological examination, Other: Vital signs, Procedure: 12 lead electrocardiogram, Procedure: Echocardiogram, Procedure: Blood draw, Procedure: Urine collection, Procedure: 2-hour Holter Monitor, Other: Brief Pain Inventory questionnaire, Other: Quality of Life questionnaire1. General and neurological examination 2. Vital signs including height, weight, blood pressure, pulse, temperature 3. 12 lead ECG 4. 2 hour Holter monitor for heart rate variability 5. Echocardiogram 6. Renal function will be assessed by the eGFR. The eGFR will be calculated from serum creatinine using CKD-EPI equation. 7. CBC with differential 8. Complete metabolic panel 9. Urinalysis 10. Urine Albumin/creatinine ratio. 11. Urine and plasma samples for biomarkers (Gb3, lyso-Gb3) that will be stored in -80 freezer and assayed in our lab. 12. Brief Pain Inventory questionnaire. 13. Quality of Life Questionnaires (SF36) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients who participated in TKT029 and who are willing and able to come to Dallas for 1 visit for standard of care testing.
- Sign the protocol informed consent form
- Have been on continuous commercial ERT since TKT029 has ended
Key exclusion criteria (4 listed)
- Patients who are unable to understand the nature, scope, and possible consequences of the study.
- Patient does not give his written informed consent to participate in this study
- Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures.
- Patient has been off ERT for an extended period of time as assessed by the investigator.
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 |
|---|---|---|
| General and Neurological examinationAlso: MD assessmentInformation about your general health, neurological symptoms and current medications with be collected | Other | Single Visit |
| Vital signsAlso: Blood pressure, heart rate, respiratory rateHeight, weight, blood pressure, heart rate, and respiratory rate and temperature will be measured. | Other | Single Visit |
| 12 lead electrocardiogramAlso: EKG, ECGA non-invasive test that measures the electrical activity of the heart | Procedure or surgery | Single Visit |
| EchocardiogramAlso: Cardiac echoA non-invasive sonogram of the heart | Procedure or surgery | Single Visit |
| Blood drawAlso: Blood collection, phlebotomy, lab testBlood will be drawn to evaluate general health and renal function (kidney health) | Procedure or surgery | Single Visit |
| Urine collectionUrine will be collection to evaluate renal function (kidney health) | Procedure or surgery | Single Visit |
| 2-hour Holter MonitorAlso: HolterA non-invasive test that measures the electrical activity of the heart continuously over 2 hours | Procedure or surgery | Single Visit |
| Brief Pain Inventory questionnaireAlso: BPI, Pain questionnaireA questionnaire about daily pain | Other | Single Visit |
| Quality of Life questionnaireAlso: SF 36A questionnaire about the impact of disease on their activities of daily living and quality of life | Other | Single Visit |
Primary outcome
estimated Glomerular Filtration Rate (eGFR)
Time frame: Study involves one visit only - assessed Baseline Visit
Change in eGFR since previous participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" - NCT00084084
8 secondary outcomes
Left Ventricular Mass Index
Time frame: Study involves one visit only - assessed Baseline Visit
Heart rate variability assessment
Time frame: Study involves one visit only - assessed Baseline Visit
Urine albumin/creatinine ratio
Time frame: Study involves one visit only - assessed Baseline Visit
Plasma Lyso-Gb3
Time frame: Study involves one visit only - assessed Baseline Visit
Plasma Gb3 and compared to plasma Gb3 results obtained during participation in study "Replagal Enzyme Replacement Therapy for Children With Fabry Disease" NCT00084084
Time frame: Study involves one visit only - assessed Baseline Visit
Urine Lyso-Gb3
Time frame: Study involves one visit only - assessed Baseline Visit
Short-form Brief Pain Inventory (BPI)
Time frame: Study involves one visit only - assessed Baseline Visit
Qualify of Life - Your Health and Well-being
Time frame: Study involves one visit only - assessed Baseline Visit
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2026.
- Background: PubMed 19318041Schiffmann R. Fabry disease. Pharmacol Ther. 2009 Apr;122(1):65-77. doi: 10.1016/j.pharmthera.2009.01.003. Epub 2009 Feb 8.
- Background: PubMed 27555236Schiffmann R, Ries M. Fabry Disease: A Disorder of Childhood Onset. Pediatr Neurol. 2016 Nov;64:10-20. doi: 10.1016/j.pediatrneurol.2016.07.001. Epub 2016 Jul 29.
- Background: PubMed 25974833Echevarria L, Benistan K, Toussaint A, Dubourg O, Hagege AA, Eladari D, Jabbour F, Beldjord C, De Mazancourt P, Germain DP. X-chromosome inactivation in female patients with Fabry disease. Clin Genet. 2016 Jan;89(1):44-54. doi: 10.1111/cge.12613. Epub 2015 Jun 22.
- Background: PubMed 16720459Dobyns WB. The pattern of inheritance of X-linked traits is not dominant or recessive, just X-linked. Acta Paediatr Suppl. 2006 Apr;95(451):11-5. doi: 10.1111/j.1651-2227.2006.tb02383.x.
- Background: PubMed 11758673MacDermot KD, Holmes A, Miners AH. Natural history of Fabry disease in affected males and obligate carrier females. J Inherit Metab Dis. 2001;24 Suppl 2:13-4; discussion 11-2. doi: 10.1023/a:1012447102358. No abstract available.
- Background: PubMed 19218538Schiffmann R, Warnock DG, Banikazemi M, Bultas J, Linthorst GE, Packman S, Sorensen SA, Wilcox WR, Desnick RJ. Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy. Nephrol Dial Transplant. 2009 Jul;24(7):2102-11. doi: 10.1093/ndt/gfp031. Epub 2009 Feb 13.
- Background: PubMed 29391017Kwon JM, Matern D, Kurtzberg J, Wrabetz L, Gelb MH, Wenger DA, Ficicioglu C, Waldman AT, Burton BK, Hopkins PV, Orsini JJ. Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease. Orphanet J Rare Dis. 2018 Feb 1;13(1):30. doi: 10.1186/s13023-018-0766-x.
- Background: PubMed 9918480Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA. 1999 Jan 20;281(3):249-54. doi: 10.1001/jama.281.3.249.
- Background: PubMed 27998644Schiffmann R, Hughes DA, Linthorst GE, Ortiz A, Svarstad E, Warnock DG, West ML, Wanner C; Conference Participants. Screening, diagnosis, and management of patients with Fabry disease: conclusions from a "Kidney Disease: Improving Global Outcomes" (KDIGO) Controversies Conference. Kidney Int. 2017 Feb;91(2):284-293. doi: 10.1016/j.kint.2016.10.004. Epub 2016 Dec 18.
- Background: PubMed 25425121Schiffmann R, Pastores GM, Lien YH, Castaneda V, Chang P, Martin R, Wijatyk A. Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study. Orphanet J Rare Dis. 2014 Nov 26;9:169. doi: 10.1186/s13023-014-0169-6.
- Is it Fabry Disease?Link provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing General and Neurological examination, Vital signs and 12 lead electrocardiogram in people with Quality of Life and Renal Insufficiency, run by Baylor Research Institute at 1 site in United States.
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 4 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
estimated Glomerular Filtration Rate (eGFR) (time frame: Study involves one visit only - assessed Baseline Visit). 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 NCT04002531, which also lists 10 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 4 Mar 2026.