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Also known as 6R-BH4 (sapropterin dihydrochloride), Tetrahydrobiopterin, Sapropterin Dihydrochloride
6R-BH4 · 2 trials · 3 indications
Early morning urine specimens were collected to calculate albumin and creatinine ratio (albuminuria) at 6 and 12 weeks of therapy.
| Arm | Type | Description |
|---|---|---|
| 6R BH4 | EXPERIMENTAL | Subjects 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 . |
| Name | Type | Description |
|---|---|---|
| 6R BH4 | DRUG | 400 mg 6R BH4 oral BID for 6 weeks then 400 mg of 6R BH4 for another 6 weeks in all arms |
| Vitamin C | DIETARY_SUPPLEMENT | 500 mg Vitamin C oral BID for another 6 weeks |
| 6R-BH4 (sapropterin dihydrochloride) | DRUG | - |
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...
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| Mineralys Therapeutics, Inc. | MLYS | 1 | PHASE3 | lorundrostat |
| United Therapeutics Corporation | UTHR | 1 | PHASE3 | Ralinepag |
| Novartis AG Sponsored ADR | NVS | 3 | PHASE2 | QCZ484, HJB647 |
| Vanda Pharmaceuticals Inc. | VNDA | 1 | PHASE2 | iloperidone |
| Opus Genetics, Inc. | IRD | 1 | PHASE1 | OPGx-BEST1 |
| CRISPR Therapeutics AG | CRSP | 1 | PHASE1 | CTX340 |
6R-BH4 has been studied in clinical trials for hypertension, or poorly controlled systemic high blood pressure, and for chronic kidney disease with albuminuria. Both are Phase 2 research programs, and the compound is not approved for these uses. It is a small molecule developed by BioMarin Pharmaceutical Inc.
6R-BH4 is developed by BioMarin Pharmaceutical Inc., which trades on Nasdaq under the ticker BMRN. BioMarin sponsored the Phase 2 clinical trials of the compound in hypertension and in chronic kidney disease with albuminuria.
6R-BH4 is in Phase 2 development. Two Phase 2 trials have been completed: one in chronic kidney disease with albuminuria and one in poorly controlled systemic hypertension. No active trials are currently listed, and the compound is investigational rather than approved.
6R-BH4 has been tested in two completed Phase 2 trials. NCT00325962 studied its effects on blood pressure in 84 subjects with poorly controlled systemic hypertension. NCT00625820 studied it in 17 patients with chronic kidney disease and albuminuria. Both trials were conducted in the United States.
Yes. 6R-BH4 is another name for sapropterin dihydrochloride, also written as 6R BH4. These names refer to the same small molecule, so searches for sapropterin dihydrochloride and 6R-BH4 point to the same BioMarin compound studied in hypertension and kidney disease.