Focal Segmental GlomerulosclerosisPhase 1NCT00816504
Effect of Galactose on Permeblity Factor in Patients With FSGS and CKD Stage 5
A Phase 1 study testing Galactose in people with Focal Segmental Glomerulosclerosis, run by Northwell Health at 1 site in United States.
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
- 0 participants
- Where
- 1 siteUnited States
- 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
- CKD Stage 5
- Resistance to steroids and another immunosuppressive medication
Who cannot join
- Secondary FSGS
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: Effect of Galactose on Permeability Factor in Patients With Focal Segmental Glomerulosclerosis (FSGS)and Chronic Kidney Disease Stage 5
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.
Five patients with biopsy proven primary FSGS - native or transplant kidney, receiving plasmapheresis - who are resistant to steroids and one other immunosuppressive agents will be studied.
The only eligibility factor is presence of the FSGS permeability factor.
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: Under additional IRB Review
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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Collaborators
- Medical College of Wisconsin
- 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
- NCT00816504
- Sponsor's protocol ID
- 08-124
- Status last verified
- Sep 2015
- First submitted
- 31 Dec 2008
- Last updated
- 7 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
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 7 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)and Chronic Kidney Disease Stage 5
- ClinicalTrials.gov ID
- NCT00816504
- Acronym / sponsor ID
- 08-124
- Conditions
- Focal Segmental Glomerulosclerosis
- Keywords
- FSGS, Permeability factor, Galactose, Primary FSGS, resistant to immunosuppressive medication
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Under additional IRB Review
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Northwell HealthUniversity, hospital or other
- Collaborators
- Medical College of Wisconsin
- 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.
Five patients with biopsy proven primary FSGS - native or transplant kidney, receiving plasmapheresis - who are resistant to steroids and one other immunosuppressive agents will be studied.
The only eligibility factor is presence of the FSGS permeability factor.
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 (3 listed)
- Primary FSGS
- CKD Stage 5
- 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 withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 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
1 site in 1 country: see every site and its status.
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 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 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: withdrawn, as of the sponsor's last update on 7 Sep 2015. 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.
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 withdrawn before enrolling participants, so there are no results.
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 NCT00816504, 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 7 Sep 2015.