Fabry DiseasePhase 1NCT01839526
A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
A Phase 1 study testing Iohexol in people with Fabry Disease, run by Genzyme, a Sanofi Company at 22 sites in 14 countries.
Trial summary
No investigational drug will be administered in this study for the treatment of Fabry disease. This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not yet initiated interventional treatment for this disease. Read more about this study
- Who is it for?
- Ages 5 to 25 years, men only
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Iohexol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 22 sitesin 14 countries
- Study dates
- May 2013to Sep 2016
- Phase
- Phase 1
- 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 5 to 25 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- The patient and/or their parent/legal guardian is willing and able to provide signed informed consent. If the patient is below the age of consent per local guidelines, he is willing to provide assent, if deemed able to do so.
- The patient must have a confirmed diagnosis of Fabry disease as documented by leukocyte α-galactosidase A (αGAL) of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory). If the leukocyte αGAL activity assay is difficult to obtain, the patient may be enrolled based on documented plasma αGAL <1.5 nmol/hr/mL (results from a central laboratory), with the agreement of the Genzyme Medical Monitor.
Who cannot join
- Patient has received prior treatment with enzyme replacement therapy (ERT) or oral pharmacological chaperone therapy for Fabry disease.
- Patient has received an investigational drug within 30 days of the screening visit.
- Patient is receiving any of the following medications and is clinically unable or unwilling to temporarily discontinue treatment with these medications for the indicated washout period prior to the renal function assessments until completion of these assessments:
- Angiotensin converting enzyme inhibitors or angiotensin receptor blockers (6 week washout);
- Non-steroidal anti-inflammatory drugs (3 day washout).
- NOTE: Patients who are on chronic dialysis or have had a kidney transplant will not be required to discontinue the above medications because renal function assessments will not be performed in these patients.
Show all 9Show fewer
- Patient has any contraindication mentioned in the labeling of iohexol. NOTE: patients with an eGFR <30 mL/min/1.73m^2 and patients who are on chronic dialysis or have had a kidney transplant may be enrolled irrespective of any contraindication to iohexol because iGFR will not be measured in these patients.
- Patient has any medical condition or extenuating circumstance which, in the opinion of the Investigator, could interfere with the patient's ability to complete all study procedures, or with the interpretation of study results (e.g., diabetes mellitus).
- The patient and/or their parent or legal guardian, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
Trial locations and contacts
22 sites in 14 countries.
22 sites in 14 countries
United States5 sites
Investigational Site Number 840003
Cincinnati, Ohio, 45229-3039
Investigational Site Number 840006
Decatur, Georgia, 30033
Investigational Site Number 840002
Fairfax, Virginia, 22030
Investigational Site Number 840010
La Jolla, California, 92093
Investigational Site Number 840009
Salt Lake City, Utah, 84132
Canada2 sites
Investigational Site Number 124001
Montreal, H4J 1C5
Investigational Site Number 124004
Toronto, M5G 1X8
France2 sites
Investigational Site Number 250002
Bron, 69677
Investigational Site Number 250001
Garches, 92380
Spain2 sites
Investigational Site Number 724001
Badalona, 08916
Investigational Site Number 724002
Girona, 17007
United Kingdom2 sites
Investigational Site Number 826004
London, NW1 2PJ
Investigational Site Number 826002
London, WC1N 3JH
About this study
Official title: A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
No investigational drug will be administered in this study for the treatment of Fabry disease.
This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not yet initiated interventional treatment for this disease. The study will consist of a screening visit(s), a clinical investigation visit(s), and a follow-up phone contact.
Read more
The objectives of the study are:
- To document renal function and other Fabry disease manifestations across age in treatment-naïve, young male patients with Fabry disease.
- To provide a reference group for comparison with interventional clinical trials of Fabry disease.
The duration of each patient's participation in the study, inclusive of the screening visit and follow-up phone contact, will be approximately 12 weeks.
Read the detailed description
Patients who meet all eligibility criteria based on screening assessments will be scheduled to return to the clinic for assessments of renal function and other disease-related parameters, which may be scheduled over one or more clinical investigation visits. The clinical investigation visit(s) will be scheduled such that renal and cardiac assessments occur after the required medication washout (see exclusion criterion); other procedures may be performed either before or after the medication washout, at the discretion of the investigator.
Up to 100 patients will be enrolled in the study, including a minimum of 15 patients in each of the following age groups (based on age at screening): 5 to 11 years, 12 to 17 years, and 18 to 25 years.
All patients will be encouraged to enroll in the Fabry Registry (NCT00196742) for continued follow-up after completion of the study.
- Conditions
- Fabry Disease
- Being tested
- Iohexol
- Keywords
- Fabry disease
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IohexolDrug
Evaluations of renal and cardiac function are standard-of-care in young patients with Fabry disease. This study uses the more precise iGFR measurement of renal function. No investigational drug will be administered in this study for the treatment of Fabry disease.
| Study group | Role | What they receive |
|---|---|---|
| Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR) | Other | Evaluations of renal and cardiac function Receives: Drug: Iohexol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Glomerular Filtration Rate Estimated from Serum Creatinine (eGFR)
Tests are performed only for patients who are not on chronic dialysis and/or have not received a kidney transplant.
Measured: Day 1 to Week 8
Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR)
Assessment of iGFR will NOT be performed if a patient is on chronic dialysis, has had a kidney transplant, or has a screening eGFR <30 mL/min/1.73 m^2 or presents any contraindication mentioned in the labeling of iohexol (Omnipaque™ 300).
Measured: Day 1 to Week 8
Protein Excretion Assessed from Three First-Morning Urine Voids
Measured: Day 1 to Week 12
Secondary outcomes (1)
Cardiovascular Function
Assessed by electrocardiogram (ECG) and echocardiography
Measured: Day 1 to Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2013 (estimated)
- Study startMay 2013
- Primary completionSep 2016
- Study completionSep 2016
Why it stopped: Enrollment period not extended and screening stopped for slow recruitment and in accordance with provisions of the protocol. Not linked to any safety concern
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies 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
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01839526
- Sponsor's protocol ID
- AGAL19110
- Other IDs
- 2012-001966-14, MSC 12711
- Status last verified
- Oct 2016
- First submitted
- 11 Apr 2013
- Last updated
- 16 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT01839526
- Acronym / sponsor ID
- AGAL19110
- Conditions
- Fabry Disease
- Keywords
- Fabry disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment period not extended and screening stopped for slow recruitment and in accordance with provisions of the protocol. Not linked to any safety concern
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- May 2013
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 25 Apr 2013 (estimated)
- 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.
No investigational drug will be administered in this study for the treatment of Fabry disease.
This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not yet initiated interventional treatment for this disease. The study will consist of a screening visit(s), a clinical investigation visit(s), and a follow-up phone contact.
The objectives of the study are:
- To document renal function and other Fabry disease manifestations across age in treatment-naïve, young male patients with Fabry disease.
- To provide a reference group for comparison with interventional clinical trials of Fabry disease.
The duration of each patient's participation in the study, inclusive of the screening visit and follow-up phone contact, will be approximately 12 weeks.
Detailed description
Patients who meet all eligibility criteria based on screening assessments will be scheduled to return to the clinic for assessments of renal function and other disease-related parameters, which may be scheduled over one or more clinical investigation visits. The clinical investigation visit(s) will be scheduled such that renal and cardiac assessments occur after the required medication washout (see exclusion criterion); other procedures may be performed either before or after the medication washout, at the discretion of the investigator.
Up to 100 patients will be enrolled in the study, including a minimum of 15 patients in each of the following age groups (based on age at screening): 5 to 11 years, 12 to 17 years, and 18 to 25 years.
All patients will be encouraged to enroll in the Fabry Registry (NCT00196742) for continued follow-up after completion of the study.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR) | Other | Drug: IohexolEvaluations of renal and cardiac function |
Who may qualify
- Ages
- 5 to 25 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- The patient and/or their parent/legal guardian is willing and able to provide signed informed consent. If the patient is below the age of consent per local guidelines, he is willing to provide assent, if deemed able to do so.
- The patient must have a confirmed diagnosis of Fabry disease as documented by leukocyte α-galactosidase A (αGAL) of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory). If the leukocyte αGAL activity assay is difficult to obtain, the patient may be enrolled based on documented plasma αGAL <1.5 nmol/hr/mL (results from a central laboratory), with the agreement of the Genzyme Medical Monitor.
Key exclusion criteria (9 listed)
- Patient has received prior treatment with enzyme replacement therapy (ERT) or oral pharmacological chaperone therapy for Fabry disease.
- Patient has received an investigational drug within 30 days of the screening visit.
- Patient is receiving any of the following medications and is clinically unable or unwilling to temporarily discontinue treatment with these medications for the indicated washout period prior to the renal function assessments until completion of these assessments:
- Patient has any contraindication mentioned in the labeling of iohexol. NOTE: patients with an eGFR <30 mL/min/1.73m^2 and patients who are on chronic dialysis or have had a kidney transplant may be enrolled irrespective of any contraindication to iohexol because iGFR will not be measured in these patients.
- Patient has any medical condition or extenuating circumstance which, in the opinion of the Investigator, could interfere with the patient's ability to complete all study procedures, or with the interpretation of study results (e.g., diabetes mellitus).
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 |
|---|---|---|
| IohexolEvaluations of renal and cardiac function are standard-of-care in young patients with Fabry disease. This study uses the more precise iGFR measurement of renal function. No investigational drug will be administered in this study for the treatment of Fabry disease. | Drug | Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR) |
Primary outcome
Glomerular Filtration Rate Estimated from Serum Creatinine (eGFR)
Time frame: Day 1 to Week 8
Tests are performed only for patients who are not on chronic dialysis and/or have not received a kidney transplant.
Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR)
Time frame: Day 1 to Week 8
Assessment of iGFR will NOT be performed if a patient is on chronic dialysis, has had a kidney transplant, or has a screening eGFR <30 mL/min/1.73 m^2 or presents any contraindication mentioned in the labeling of iohexol (Omnipaque™ 300).
Protein Excretion Assessed from Three First-Morning Urine Voids
Time frame: Day 1 to Week 12
1 secondary outcome
Cardiovascular Function
Time frame: Day 1 to Week 8
Results and safety
The study was stopped early (reason given: Enrollment period not extended and screening stopped for slow recruitment and in accordance with provisions of the protocol. Not linked to any safety concern). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 2020.
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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 Monitor study director
Genzyme, a Sanofi Company
22 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Iohexol in people with Fabry Disease, run by Genzyme, a Sanofi Company at 22 sites in 14 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 2 inclusion and 9 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: stopped early, as of the sponsor's last update on 16 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Canada, France, Spain, United Kingdom and 9 more countries. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Enrollment period not extended and screening stopped for slow recruitment and in accordance with provisions of the protocol. Not linked to any safety concern). Results are not available in the source record.
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?
Glomerular Filtration Rate Estimated from Serum Creatinine (eGFR) (time frame: Day 1 to Week 8). The study lists 3 primary outcomes in all. 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 16 Dec 2020.