Trial Outcomes & Findings for Podocyte Retinoids (NCT NCT00098020)
NCT ID: NCT00098020
Last Updated: 2017-12-13
Results Overview
Change of proteinuria at Week 24 compared to the baseline using protein/creatinine ratio (PCR)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
Baseline and Week 24
Results posted on
2017-12-13
Participant Flow
Participant milestones
| Measure |
Isotretinoin
Subjects will be treated with isotretinoin initially at 1mg/kg for the first 4 weeks and then dose will escalate to a higher dose only if subjects have tolerated the lower dose.
In younger patients of 16 and 17 years of age, the dose will be started at 1 mg/kg and will remain at this dose without escalation.
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
Started Isotretinoin
|
8
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Isotretinoin
Subjects will be treated with isotretinoin initially at 1mg/kg for the first 4 weeks and then dose will escalate to a higher dose only if subjects have tolerated the lower dose.
In younger patients of 16 and 17 years of age, the dose will be started at 1 mg/kg and will remain at this dose without escalation.
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
elevated liver enzymes
|
1
|
|
Overall Study
Low urine PCR
|
1
|
|
Overall Study
Low epidermal growth factor receptor
|
1
|
Baseline Characteristics
Podocyte Retinoids
Baseline characteristics by cohort
| Measure |
Isotretinoin
n=12 Participants
subjects will be treated with iIsotretinoin.
|
|---|---|
|
Age, Continuous
|
48 years
STANDARD_DEVIATION 12.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Protein, Urine
|
626.42 mg/dL
STANDARD_DEVIATION 642.58 • n=5 Participants
|
|
Creatinine, Urine
|
71.37 mg/dL
STANDARD_DEVIATION 43.36 • n=5 Participants
|
|
Protein/Creatinine Ratio, Urine
|
8.42 g/g
STANDARD_DEVIATION 4.69 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 24Population: A total of 7 patients is analyzed due to 1 withdrawal.
Change of proteinuria at Week 24 compared to the baseline using protein/creatinine ratio (PCR)
Outcome measures
| Measure |
Isotretinoin
n=7 Participants
oral isotretinoin
|
|---|---|
|
Change in Proteinuria at Week 24 From Baseline
Baseline
|
8.42 g/g
Standard Deviation 4.69
|
|
Change in Proteinuria at Week 24 From Baseline
Week 24
|
7.67 g/g
Standard Deviation 5.36
|
SECONDARY outcome
Timeframe: End of one year from baselinePopulation: Seven out of 8 subjects who were treated with isotretinoin completed Week 24. One out of the 7 subjected who completed Week 24 did not complete the whole study.
Based on 24hour proteinuria, response outcomes are defined as CR (complete remission): \<0.3 g/g PR (partial remission): 50% fall from baseline and \<2.0 g/g
Outcome measures
| Measure |
Isotretinoin
n=7 Participants
oral isotretinoin
|
|---|---|
|
Number of Patients Who Are in Complete Remission (CR) or Partial Remission (PR) at 6 Months or at the End of One Year.
|
0 Participants
|
Adverse Events
Isotretinoin
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Isotretinoin
n=8 participants at risk
Subjects will be treated with isotretinoin .
|
|---|---|
|
Nervous system disorders
Agitation
|
12.5%
1/8 • Number of events 3 • 18 months
|
|
Renal and urinary disorders
Alanine aminotransferase increased
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Blood and lymphatic system disorders
Alopecia
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Blood and lymphatic system disorders
Anemia
|
25.0%
2/8 • Number of events 3 • 18 months
|
|
Gastrointestinal disorders
Anorexia
|
25.0%
2/8 • Number of events 2 • 18 months
|
|
Immune system disorders
Arthralgia (knee/ankle)
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Blood and lymphatic system disorders
Ascites
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Hepatobiliary disorders
Aspartate aminotransferase increased
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Hepatobiliary disorders
Cholesterol high
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
General disorders
Conduction disorder
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Renal and urinary disorders
Creatinine Increased CTCAE
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Renal and urinary disorders
Creatinine increased
|
12.5%
1/8 • Number of events 2 • 18 months
|
|
General disorders
Dexamethasone x4 doses: start MMF and CSA
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Eye disorders
Dry eye
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
12.5%
1/8 • Number of events 2 • 18 months
|
|
Skin and subcutaneous tissue disorders
Dry skin (lips)
|
25.0%
2/8 • Number of events 4 • 18 months
|
|
Gastrointestinal disorders
Dyspepsia
|
25.0%
2/8 • Number of events 2 • 18 months
|
|
Blood and lymphatic system disorders
Edema Limbs
|
37.5%
3/8 • Number of events 4 • 18 months
|
|
Blood and lymphatic system disorders
Edema Trunk
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Blood and lymphatic system disorders
Epistaxis
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Nervous system disorders
Facial pain
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
General disorders
Fatigue
|
50.0%
4/8 • Number of events 4 • 18 months
|
|
General disorders
Headache
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Gastrointestinal disorders
Hemorrhoidal hemorrhage
|
12.5%
1/8 • Number of events 2 • 18 months
|
|
General disorders
Hoarseness
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Renal and urinary disorders
Hyperkalemia
|
25.0%
2/8 • Number of events 6 • 18 months
|
|
Cardiac disorders
Hypertension
|
37.5%
3/8 • Number of events 3 • 18 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Gastrointestinal disorders
Nausea
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Eye disorders
Night Blindness
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Cardiac disorders
Pacemaker placed
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Blood and lymphatic system disorders
Periorbital edema
|
12.5%
1/8 • Number of events 2 • 18 months
|
|
Infections and infestations
Postnasal drip
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Infections and infestations
Productive cough
|
25.0%
2/8 • Number of events 2 • 18 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
12.5%
1/8 • Number of events 3 • 18 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
General disorders
Scalp pain
|
25.0%
2/8 • Number of events 2 • 18 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Reproductive system and breast disorders
Vaginal hemorrhage
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Cardiac disorders
Vasovagal reaction
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
General disorders
Watering eyes
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Metabolism and nutrition disorders
Weight loss
|
12.5%
1/8 • Number of events 1 • 18 months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
12.5%
1/8 • Number of events 1 • 18 months
|
Additional Information
Dr. Jeffery Kopp
National Institutes of Diabetes, Digestive, and Kidney Disorders
Phone: 301-594-3403
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place