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6R BH4

Phase 2

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

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment17

FDA Designations

No designations recorded

Clinical trial landscape

6R BH4 · 1 trial · 2 indications

Phase 2 1
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and AlbuminuriaKidney Disease
COMPLETED17 Analytics
PHASE2COMPLETED
Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
Kidney DiseaseUnlock trial analytics

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 to 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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Frequently asked questions about 6R BH4

What is 6R BH4 used for?

6R BH4 is an investigational small molecule being studied for kidney disease and hypertension. In clinical trials, it has been evaluated for its effects on blood pressure in patients with poorly controlled systemic hypertension and in patients with chronic kidney disease and albuminuria. It is not approved and remains in clinical development.

Who makes 6R BH4?

6R BH4 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker BMRN. The company has sponsored Phase 2 clinical trials of the drug in the United States.

What phase is 6R BH4 in?

6R BH4 is in Phase 2 clinical development. Two Phase 2 trials have been completed, one in hypertension and one in kidney disease with albuminuria. The drug is investigational and has not been approved by regulatory authorities.

What clinical trials is 6R BH4 in?

6R BH4 has been studied in two completed Phase 2 trials. NCT00325962 examined its effects on blood pressure in 84 adults with poorly controlled systemic hypertension. NCT00625820 studied the drug in 17 adults with chronic kidney disease and albuminuria. Both trials were conducted in the United States.

Is 6R BH4 the same as tetrahydrobiopterin?

6R BH4 is also known as tetrahydrobiopterin, and the clinical trial NCT00625820 refers to the drug by that name. It is a small molecule being investigated for its potential effects on blood pressure and kidney function in patients with hypertension and chronic kidney disease.