Trial Outcomes & Findings for Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome (NCT NCT01113385)

Results Overview

FSPF is reported in relation to its induction of glomerular albumin permeability (Palb) of isolated glomeruli on a range from 0 to 1, with 0 indicative of normal glomeruli and 1 indicative of injury to the permeability barrier. Results will be considered clinically significant if the following criteria is met in response to oral galactose therapy at week 16: Reduction in FSPF to \<0.5 Palb or decrease in FSPF by \> 0.3 Palb.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

7 participants

Primary outcome timeframe

16 weeks

Results posted on

2014-09-15

Participant Flow

Participant milestones

Participant milestones
Measure	Galactose Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks. D-Galactose: Oral galactose will be initiated at a dose of 0.2gm/kg/dose twice daily to a maximum of 15 gm BID for a period of 4 months. The prescribed dose of D-galactose powder will be dispensed to subjects in packets, mixed with 4 ounces of water, and consumed orally.
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Galactose n=7 Participants Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks. D-Galactose: Oral galactose will be initiated at a dose of 0.2gm/kg/dose twice daily to a maximum of 15 gm BID for a period of 4 months. The prescribed dose of D-galactose powder will be dispensed to subjects in packets, mixed with 4 ounces of water, and consumed orally.
Age, Continuous	7.7 years STANDARD_DEVIATION 4 • n=5 Participants
Age, Categorical <=18 years	7 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants

PRIMARY outcome

Timeframe: 16 weeks

FSPF is reported in relation to its induction of glomerular albumin permeability (Palb) of isolated glomeruli on a range from 0 to 1, with 0 indicative of normal glomeruli and 1 indicative of injury to the permeability barrier. Results will be considered clinically significant if the following criteria is met in response to oral galactose therapy at week 16: Reduction in FSPF to \<0.5 Palb or decrease in FSPF by \> 0.3 Palb.

Outcome measures

Outcome measures
Measure	Galactose n=7 Participants Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks. D-Galactose: Oral galactose will be initiated at a dose of 0.2gm/kg/dose twice daily to a maximum of 15 gm BID for a period of 4 months. The prescribed dose of D-galactose powder will be dispensed to subjects in packets, mixed with 4 ounces of water, and consumed orally.
Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) Baseline FSPF	0.69 Palb Standard Deviation 0.11
Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) 16 Week FSPF	0.35 Palb Standard Deviation 0.21

SECONDARY outcome

Timeframe: 16 weeks

Results will be considered clinically significant if the following criteria is met in response to oral galactose therapy at week 16. Complete remission is defined as (Urine Protein:Creatinine ratio \[UPC\] \<0.2 g/g). Partial remission is defined as UPC 0.2-2 g/g.

Outcome measures

Outcome measures
Measure	Galactose n=7 Participants Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks. D-Galactose: Oral galactose will be initiated at a dose of 0.2gm/kg/dose twice daily to a maximum of 15 gm BID for a period of 4 months. The prescribed dose of D-galactose powder will be dispensed to subjects in packets, mixed with 4 ounces of water, and consumed orally.
Number of Participants Achieving Complete or Partial Remission at 16 Weeks	0 participants in remission at 16 weeks

Adverse Events

Galactose

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Galactose n=7 participants at risk Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks. D-Galactose: Oral galactose will be initiated at a dose of 0.2gm/kg/dose twice daily to a maximum of 15 gm BID for a period of 4 months. The prescribed dose of D-galactose powder will be dispensed to subjects in packets, mixed with 4 ounces of water, and consumed orally.
Gastrointestinal disorders Diarrhea	14.3% 1/7 • Number of events 1 • Monthly for duration of galactose therapy (4 months)

Additional Information

Asha Moudgil MD, Professor of Pediatrics

Children's National

Phone: 202-476-5058

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place