Drug · CHEMBL1201774
SAPROPTERIN
7 sourcespipelines-2026-09-18-0.3.1
Cite this record
ConductScience Library. SAPROPTERIN. Drugs and targets, record CHEMBL1201774. Release pipelines-2026-09-18-0.3.1, 18 Sept 2026. https://conductscience.org/library/drugs/CHEMBL1201774
On this page
Drug
Names and IDs as ChEMBL gives them, through Open Targets.
- Open Targets' highest stage
- Approval, for 2 of its 28 diseases, from EMA Human Drugs and TTD
- Open Targets
Targets1
The targets ChEMBL's mechanism records give for the drug, through Open Targets. A mechanism record says what the drug acts on, not how strongly.
| Target | Protein | Mechanism | Action | Gene in Genes |
|---|---|---|---|---|
| PAH | Phenylalanine-4-hydroxylase | Phenylalanine-4-hydroxylase activator | ACTIVATOR | https://conductscience.org/library/genes/5053 |
Source ChEMBL
Diseases28
The diseases Open Targets' clinical reports study the drug for, the most trial reports first. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
1 to 10 of 28Source Open Targets
See all 28 diseasesTrials32
Trials whose record names the drug by a name ChEMBL states, posted results first, then the largest. Open Targets read each trial's text to find its drugs. 1 more rests on that reading alone and is shown because the trial's posted result groups or NLM's indexing of its papers name the drug. 1 more that Open Targets linked to it is not shown, since the release's checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Named as | Linked by | Source |
|---|---|---|---|---|---|---|---|
| NCT01356966 | Exercise Intolerance in Renal Failure | Phase 2 | 74 | 2015-06-01 | tetrahydrobiopterin | Its name | ClinicalTrials.gov |
| NCT01772758 | Cystic Fibrosis and Endothelial Function: At Rest and During Exercise | Phase 2 | 64 | 2018-01-10 | tetrahydrobiopterin | Its name | ClinicalTrials.gov |
| NCT01398943 | Nitric Oxide Bioavailability in Chronic Obstructive Pulmonary Disease (COPD) | 60 | 2017-07-25 | tetrahydrobiopterin | Its name | ClinicalTrials.gov | |
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | Phase 2 | 46 | 2014-02-28 | sapropterin | Its name | ClinicalTrials.gov |
| NCT00943579 | Open-Label Extension Study of Kuvan for Autism | Phase 2/3 | 41 | 2013-07-04 | sapropterin | Its name | ClinicalTrials.gov |
| NCT02353312 | Rhode Island Diastolic Dysfunction - Heart Failure | 28 | 2025-07-03 | tetrahydrobiopterin | Its name | ClinicalTrials.gov | |
| NCT01566084 | Efficacy of Dietary Sodium Restriction of Improving Vascular Endothelial Function in Middle Aged and Older Adults | 17 | 2014-06-20 | BH4 | Open Targets' reading, confirmed by the trial's result groups | ClinicalTrials.gov | |
| NCT02774226 | Long Term Nitric Oxide Bioavailability on Vascular Health in Chronic Obstructive Pulmonary Disease | Phase 2 | 15 | 2021-02-23 | tetrahydrobiopterin | Its name | ClinicalTrials.gov |
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | Phase 1/2 | 12 | 2014-04-08 | sapropterin | Its name | ClinicalTrials.gov |
| NCT02530996 | Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment | Phase 1/2 | 12 | 2021-04-05 | tetrahydrobiopterin | Its name | ClinicalTrials.gov |
1 to 10 of 32
See all 32 trialsComparisons in its trials14
Comparisons Trial comparisons calculated in the trials whose record names the drug, most participants first. A comparison in one of its trials does not always compare the drug, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| COPD Patients vs Controls: serious adverse events | 0 of 30 | 0 of 30 | 60 | NCT01398943 | Trial comparisons |
| On Kuvan vs Off Kuvan: deaths from any cause | 0 of 28 | 0 of 28 | 56 | NCT02353312 | Trial comparisons |
| On Kuvan vs Off Kuvan: serious adverse events | 4 of 28 | 6 of 28 | 56 | NCT02353312 | Trial comparisons |
| Sapropterin vs Placebo: Clinical Global Impression -- Severity (CGI-S) Scale | 3 of 23 | 1 of 23 | 46 | NCT00850070 | Trial comparisons |
| Sapropterin vs Placebo: Clinical Global Impression -- Improvement (CGI-I) Scale | 5 of 23 | 3 of 23 | 46 | NCT00850070 | Trial comparisons |
| Sapropterin vs Placebo: serious adverse events | 2 of 23 | 1 of 23 | 46 | NCT00850070 | Trial comparisons |
| Kuvan Following Active Treatment vs Kuvan Following Placebo: serious adverse events | 0 of 20 | 0 of 21 | 41 | NCT00943579 | Trial comparisons |
| Tetrahydrobiopterin + Folate vs Placebo + Folate: serious adverse events | 0 of 18 | 0 of 14 | 32 | NCT01356966 | Trial comparisons |
| 12 SSc Participants BH4 vs 12 SSc Participants Placebo: deaths from any cause | 0 of 12 | 0 of 12 | 24 | NCT02530996 | Trial comparisons |
| 12 SSc Participants BH4 vs 12 SSc Participants Placebo: serious adverse events | 0 of 12 | 0 of 12 | 24 | NCT02530996 | Trial comparisons |
1 to 10 of 14
See all 14 comparisons in its trialsPapers from its trials14
The papers the trials whose record names the drug list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Tests in its trials9
The tests ConductCognition runs that the trials whose record names the drug name in their registered outcomes, as ScienceGraph matched them. ConductCognition's list names the tests it runs, not every test a trial used.
| Test | Trials naming it as an outcome | As a primary outcome | Source |
|---|---|---|---|
| Verbal Fluency | 2 | 0 | ConductCognition |
| Category Fluency | 1 | 0 | ConductCognition |
| Digit Span Test | 1 | 0 | ConductCognition |
| Go/No-Go Task | 1 | 0 | ConductCognition |
| 1-Back Task | 1 | 0 | ConductCognition |
| 2-Back Task | 1 | 0 | ConductCognition |
| Paced Visual Serial Addition Test | 1 | 0 | ConductCognition |
| Symbol Coding | 1 | 0 | ConductCognition |
| Verbal Learning | 1 | 0 | ConductCognition |
Data dates
The date of each source this release read.
Sources 7
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ConductCognition's list of its testsOur own data
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ConductCognition's list of its testsOur own data: ConductCognition's list of its tests, https://conductcognition.com
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this drug from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/drugs/v1/CHEMBL1201774
GET https://conductscience.org/api/drugs/v1/CHEMBL1201774/diseases?page=2