A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
Completed
Censa Pharmaceuticals, Inc.
Phase 1/Phase 2
2018-06-24
This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary
efficacy of CNSA-001 in reducing blood phenylalanine concentrations in participants with
hyperphenylalaninemia due to primary tetrahydrobiopterin (BH4) deficiency.
A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
Completed
PTC Therapeutics
Phase 1/Phase 2
2018-06-24
This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary
efficacy of CNSA-001 in reducing blood phenylalanine concentrations in participants with
hyperphenylalaninemia due to primary tetrahydrobiopterin (BH4) deficiency.
A Study of CNSA-001 in Women With Diabetic Gastroparesis
Completed
Censa Pharmaceuticals, Inc.
Phase 2
2019-02-27
This study evaluates CNSA-001 in the treatment of women with moderate to severe diabetic
gastroparesis. Half the patients will receive CNSA-001, while the other half will receive a
placebo.
A Study of CNSA-001 in Women With Diabetic Gastroparesis
Completed
PTC Therapeutics
Phase 2
2019-02-27
This study evaluates CNSA-001 in the treatment of women with moderate to severe diabetic
gastroparesis. Half the patients will receive CNSA-001, while the other half will receive a
placebo.
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
RECRUITING
PTC Therapeutics
PHASE3
2024-03-04
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
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