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Tetrahydrobiopterin /kg

Phase 1

Claudication, Intermittent | Small molecule | Cardiovascular |BioMarin Pharmaceutical Inc.|Last Updated: Apr 15, 2026

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment10
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent ClaudicationPHASE1 ACTIVE NOT_RECRUITING 10Jun 15, 2021Dec 1, 2027Apr 15, 20261 United States
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Study Endpoints
Primary Endpoints
Nitric Oxide bioavailability
90 days

Nitric Oxide bioavailability as measured by flow mediated dilatation (FMD).

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeOTHER
Treatment Arms
ArmTypeDescription
Tetrahydrobiopterin Dose 1 (Day 0 to 44)EXPERIMENTALAll subjects will receive 3300mg l-Ascorbate , l-Arginine 3400mg and 10mg/kg of Tetrahydrobiopterin once a day.
Tetrahydrobiopterin Dose 2 (Day 45 to 90)EXPERIMENTALAll subjects will receive 3300mg l-Ascorbate, l-Arginine 3400mg and 20mg/kg of Tetrahydrobiopterin once a day.
Interventions
NameTypeDescription
Tetrahydrobiopterin 10 mg/kgDRUG10mg/kg of Tetrahydrobiopterin daily.
Tetrahydrobiopterin 20 mg/kgDRUG20mg/kg of Tetrahydrobiopterin daily.
L-AscorbateDRUG3300 mg of l-ascorbate once daily
L-ArginineDRUG3400mg of l-arginine once daily
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * PAD as demonstrated by an ABI \<0 .9 in one leg or TBI less than \<0.7 in patients with an ABI \>1.3 (non compressible vessels) * Rutherford Classification II, III * Age \>18 years old * Willingness to discontinue phosphodiesterase (PDE) 5 inhibitors such as Viagra, Cialis, Le...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04800692studyFirstPostDate: changed