Focal Segmental GlomerulosclerosisPhase 1NCT00816478
Effect of Oral Galactose on Focal Segmental Glomerulosclerosis (FSGS) Permeability Factor
A Phase 1 study testing Galactose in people with Focal Segmental Glomerulosclerosis, run by Northwell Health.
Trial summary
This study is a proof-of-concept clinical study designed to test the hypothesis that oral administration of galactose can lower the level of a circulating factor that increases glomerular permeability to albumin in patients with resistant FSGS. Read more about this study
- Who is it for?
- Ages 2 to 60 years
- Conditions the study is for
- Focal Segmental Glomerulosclerosis
- What is being tested?
- Galactose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- Dec 2008to Dec 2009
- 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 2 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Primary FSGS
- Resistance to steroids and another immunosuppressive medication
Who cannot join
- Secondary FSGS
About this study
Official title: Effect of Galactose on Permeability Factor in Patients With Focal Segmental Glomerulosclerosis (FSGS)
This study is a proof-of-concept clinical study designed to test the hypothesis that oral administration of galactose can lower the level of a circulating factor that increases glomerular permeability to albumin in patients with resistant FSGS.
Read the detailed description
Patients with resistant FSGS have persistent proteinuria and a high risk of progression to end stage kidney disease (ESKD). A circulating factor that increases glomerular permeability to albumin (Palb) has been detected in over 50% of these patients. While the molecular identity of the factor has not been fully established, in vitro studies and limited clinical experience suggest that galactose can reduce the level of the FSGS permeability factor.
This study is a pilot study to determine if oral administration of galactose can lower the circulating level of the FSGS permeability factor.
Two groups of patients with biopsy proven primary FSGS who are resistant to steroids and one other immunosuppressive agents will be studied:
- FSGS, pre-ESKD 2. FSGS, with ESKD on renal replacement therapy
The only eligibility factor is presence of the FSGS permeability factor.
Five patients will be included in each group.
The experimental intervention is administration of galactose, orally, 0.2 g/kg body weight/dose twice daily. the duration of treatment will be 28 days,.
Patients will be seen on days 0, 14, and 28 of treatment. They will be seen at week 8, 16 and 24 after discontinuation of the galactose.
Physical examination and routine laboratory tests (SMAC, CBC, urine protein:creatinine ratio in an early morning sample) will be done at each visit. The FSGS permeability factor will be assayed at days 0 and 28 of treatment and 6 months after discontinuation of the galactose. The permeability factor will be tested in the laboratory of Virginia Savin MD (Medical college of Wisconsin) using previously described methods.
All other treatments will be unchanged during the 28 day oral galactose Treatment Period.
The study will be analyzed based on the number of patients in whom the FSGS permeability factor is lowered to normal levels.
- Conditions
- Focal Segmental Glomerulosclerosis
- Being tested
- Galactose
- Keywords
- FSGS, Permeability factor, Galactose, Primary FSGS, resistant to immunosuppressive medication
- Medical subject headings
- Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GalactoseDrug
Oral galactose, 0.2 g/kg/dose twice daily for 28 days
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Galactose Receives: Drug: Galactose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in FSGS permeability factor
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jan 2009 (estimated)
- Study startDec 2008
- Primary completionDec 2009
- Study completionDec 2009
Why it stopped: Study closed by Institutional Review Board
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Focal Segmental GlomerulosclerosisStudies of the same condition, open and under way.
- More trials run by Northwell HealthThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Howard Trachtman MD, Northwell Health
- Investigators
- Howard Trachtman, MD, Principal investigator, Schneider Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00816478
- Sponsor's protocol ID
- NEPHCURE 1
- Status last verified
- Sep 2015
- First submitted
- 31 Dec 2008
- Last updated
- 10 Sep 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Trachtman H, Greenbaum LA, McCarthy ET, Sharma M, Gauthier BG, Frank R, Warady B, Savin VJ. Glomerular permeability activity: prevalence and prognostic value in pediatric patients with idiopathic nephrotic syndrome. Am J Kidney Dis. 2004 Oct;44(4):604-10.
PubMed 15384010
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Galactose on Permeability Factor in Patients With Focal Segmental Glomerulosclerosis (FSGS)
- ClinicalTrials.gov ID
- NCT00816478
- Acronym / sponsor ID
- NEPHCURE 1
- Conditions
- Focal Segmental Glomerulosclerosis
- Keywords
- FSGS, Permeability factor, Galactose, Primary FSGS, resistant to immunosuppressive medication
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study closed by Institutional Review Board
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Northwell HealthUniversity, hospital or other
- Study start
- Dec 2008
- Primary completion
- Dec 2009 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 1 Jan 2009 (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.
This study is a proof-of-concept clinical study designed to test the hypothesis that oral administration of galactose can lower the level of a circulating factor that increases glomerular permeability to albumin in patients with resistant FSGS.
Detailed description
Patients with resistant FSGS have persistent proteinuria and a high risk of progression to end stage kidney disease (ESKD). A circulating factor that increases glomerular permeability to albumin (Palb) has been detected in over 50% of these patients. While the molecular identity of the factor has not been fully established, in vitro studies and limited clinical experience suggest that galactose can reduce the level of the FSGS permeability factor.
This study is a pilot study to determine if oral administration of galactose can lower the circulating level of the FSGS permeability factor.
Two groups of patients with biopsy proven primary FSGS who are resistant to steroids and one other immunosuppressive agents will be studied:
- FSGS, pre-ESKD 2. FSGS, with ESKD on renal replacement therapy
The only eligibility factor is presence of the FSGS permeability factor.
Five patients will be included in each group.
The experimental intervention is administration of galactose, orally, 0.2 g/kg body weight/dose twice daily. the duration of treatment will be 28 days,.
Patients will be seen on days 0, 14, and 28 of treatment. They will be seen at week 8, 16 and 24 after discontinuation of the galactose.
Physical examination and routine laboratory tests (SMAC, CBC, urine protein:creatinine ratio in an early morning sample) will be done at each visit. The FSGS permeability factor will be assayed at days 0 and 28 of treatment and 6 months after discontinuation of the galactose. The permeability factor will be tested in the laboratory of Virginia Savin MD (Medical college of Wisconsin) using previously described methods.
All other treatments will be unchanged during the 28 day oral galactose Treatment Period.
The study will be analyzed based on the number of patients in whom the FSGS permeability factor is lowered to normal levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: GalactoseGalactose |
Who may qualify
- Ages
- 2 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Primary FSGS
- Resistance to steroids and another immunosuppressive medication
Key exclusion criteria (1 listed)
- Secondary FSGS
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 |
|---|---|---|
| GalactoseOral galactose, 0.2 g/kg/dose twice daily for 28 days | Drug | 1 |
Primary outcome
Reduction in FSGS permeability factor
Time frame: 28 days
Results and safety
The study was stopped early (reason given: Study closed by Institutional Review Board). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2015.
- Background: PubMed 15384010Trachtman H, Greenbaum LA, McCarthy ET, Sharma M, Gauthier BG, Frank R, Warady B, Savin VJ. Glomerular permeability activity: prevalence and prognostic value in pediatric patients with idiopathic nephrotic syndrome. Am J Kidney Dis. 2004 Oct;44(4):604-10.
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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.
Howard Trachtman, MD principal investigator
Schneider Children's Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Galactose in people with Focal Segmental Glomerulosclerosis, run by Northwell Health.
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 1 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 10 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
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: Study closed by Institutional Review Board). 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?
Reduction in FSGS permeability factor (time frame: 28 days). 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 NCT00816478, which also lists 1 linked publication.
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 10 Sep 2015.