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6R BH4

Phase 2

Kidney Disease | Small molecule | Nephrology |BioMarin Pharmaceutical Inc.|Last Updated: Oct 18, 2016

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment17
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and AlbuminuriaPHASE2 COMPLETED 17May 1, 2008Sep 1, 2008Oct 18, 20161 United States
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Study Endpoints
Primary Endpoints
The Primary Outcome Measure is Level of Albuminuria.
12 weeks

Early morning urine specimens were collected to calculate albumin and creatinine ratio (albuminuria) at 6 and 12 weeks of therapy.

Secondary Endpoints
Systolic Blood Pressure Measured at 6 and 12 Weeks of Therapy.
12 weeks
Estimated Glomerular Filtration Rate (eGFR) Measured at 6 and 12 Weeks of Therapy.
12 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
6R BH4EXPERIMENTALSubjects will receive 6R-BH4 400mg bid for 6 weeks, sequentially followed by 6R-BH4 plus Vitamin C 500mg bid for another 6 weeks. Patients will have scheduled visits at Weeks 0,3,6,9 and 12, with an exit-visit at week 16. Albuminuria will be assessed in 24-hour urine collections as well as early morning spot urine samples for albumin:creatinine ratio. Blood and urine will be tested for routine clinical laboratory tests, blood nitric oxide (NO), and also archived for later assays for special biomarkers. The primary outcome will be level of albuminuria as measured in a 24-hour urine collection at 6 and 12 weeks of therapy. Secondary outcomes will include urine albumin/creatinine ratio, estimated glomerular filtration rate (eGFR), and blood pressure .
Interventions
NameTypeDescription
6R BH4DRUG400 mg 6R BH4 oral BID for 6 weeks then 400 mg of 6R BH4 for another 6 weeks in all arms
Vitamin CDIETARY_SUPPLEMENT500 mg Vitamin C oral BID for another 6 weeks
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Patients with controlled hypertension (blood pressure (BP) less than 150/90 mmHg) using standard antihypertensive medications. * Stable chronic kidney disease (CKD) with estimated glomerular filtration rate (eGFR) 40-90 ml/min/173m2 by the abbreviated Modification of Diet in R...

Countries:United States
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