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Chemical Structure| 69056-38-8 Chemical Structure| 69056-38-8

Structure of Sapropterin 2HCl
CAS No.: 69056-38-8

Chemical Structure| 69056-38-8

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Sapropterin 2HCl is a synthetic form of BH4 that is approved for the treatment of BH4 responsive PKU.

Synonyms: (6R)-BH4 dihydrochloride; (6R)-Tetrahydro-L-biopterin dihydrochloride; BH4

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Product Details of Sapropterin 2HCl

CAS No. :69056-38-8
Formula : C9H17Cl2N5O3
M.W : 314.17
SMILES Code : O=C1N=C(N)NC2=C1N[C@@H]([C@@H](O)[C@@H](O)C)CN2.[H]Cl.[H]Cl
Synonyms :
(6R)-BH4 dihydrochloride; (6R)-Tetrahydro-L-biopterin dihydrochloride; BH4
MDL No. :MFCD00891665
InChI Key :RKSUYBCOVNCALL-NTVURLEBSA-N
Pubchem ID :135409471

Safety of Sapropterin 2HCl

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H302
Precautionary Statements:P280-P305+P351+P338

Isoform Comparison

Biological Activity

Clinical Trial:

NCT Number Conditions Phases Recruitment Completion Date Locations
NCT02677870 Phenylketonuria Phase 4 Recruiting August 2018 United States, Ohio ... More >> University Hospitals Cleveland Medical Center Recruiting Cleveland, Ohio, United States, 44106 Contact: Lori P Schillaci, MD    216-844-3936    lori-anne.schillaci@uhhospitals.org Less <<
NCT00625820 Kidney Disease ... More >> Albuminuria Less << Phase 2 Completed - United States, Michigan ... More >> University of Michigan Ann Arbor, Michigan, United States, 48109 Less <<
NCT00625820 - Completed - -
NCT01772758 - Completed - -
NCT01772758 Cystic Fibrosis Phase 2 Completed - United States, Georgia ... More >> Georgia Prevention Institute/ Laboratory of Integrative and Exercise Physiology Augusta, Georgia, United States, 30912 Less <<
NCT01869972 - Completed - Canada, Ontario ... More >> McMaster Children's Hospital Hamilton, Ontario, Canada, L8S 4J9 Children's Hospital of Eastern Ontario Ottawa, Ontario, Canada, K1H 8L1 Less <<
NCT02009904 - Completed - Canada, British Columbia ... More >> Child & Family Research Institute Vancouver, British Columbia, Canada, V5Z 4H4 Less <<
NCT01398943 Pulmonary Disease, Chronic Obs... More >>tructive Less << Not Applicable Completed - United States, Georgia ... More >> Augusta University Augusta, Georgia, United States, 30912 Less <<
NCT02947750 Renal Insufficiency, Chronic Phase 2 Recruiting November 2021 United States, Georgia ... More >> Emory University Hospital Midtown Recruiting Atlanta, Georgia, United States, 30308 Contact: Dana DaCosta    404-727-7762    drdacos@emory.edu    Principal Investigator: Jeanie Park, MD          Emory University Hospital Recruiting Atlanta, Georgia, United States, 30322 Contact: Dana DaCosta    404-727-7762    drdacos@emory.edu    Principal Investigator: Jeanie Park, MD          Atlanta VA Health Care System Recruiting Decatur, Georgia, United States, 30033 Contact: Dana DaCosta    404-727-7762    drdacos@emory.edu    Contact: Melanie Kankam    404-321-6111 ext 7040    mljeffe@emory.edu    Principal Investigator: Jeanie Park, MD Less <<
NCT01541397 Hyperphenylalaninemia ... More >> Phenylketonuria Less << Not Applicable Terminated - United States, Texas ... More >> The University of Texas Health Science Center at Houston Houston, Texas, United States, 77030 Less <<
NCT01398943 - Completed - -
NCT00827762 - Terminated(Too few participant... More >>s recruited within timeframe) Less << - United States, Illinois ... More >> Northwestern University/Children's Memorial Hospital Chicago, Illinois, United States, 60614 United States, Missouri University of Missouri Columbia, Missouri, United States, 65211 Washington University St. Louis, Missouri, United States, 63130 United States, Oregon Oregon Health & Science University Portland, Oregon, United States, 97239 Less <<
NCT02774226 Chronic Obstructive Pulmonary ... More >>Disease (COPD) Less << Phase 2 Recruiting September 2018 United States, Georgia ... More >> Georgia Prevention Institute Recruiting Augusta, Georgia, United States, 30912 Contact: Ryan Harris, Ph.D.    706-721-5998    ryharris@augusta.edu    Contact: Nichole Seigler, BA    706-721-5998    maseigler@augusta.edu Less <<
NCT01541397 - Terminated - -
NCT00841100 Phenylketonuria Not Applicable Unknown February 2010 United States, Florida ... More >> Department of Biochemistry & Molecular Biology Recruiting Miami, Florida, United States, 33136 Contact: Louis J Elsas, MD    305-243-7126    lelsas@med.miami.edu    Contact: Andrea M Wierenga, PhD    305 243 8122    awierenga@med.miami.edu    Sub-Investigator: Andrea M Wierenga, PhD Less <<
NCT00532844 Endothelial Dysfunction Phase 2 Completed - United States, New Jersey ... More >> Hackensack, New Jersey, United States Less <<
NCT00778206 - Recruiting September 2023 -
NCT01425528 GTP Cyclohydrolase Deficiency Phase 1 Phase 2 Completed - United States, Utah ... More >> University of Utah Salt Lake City, Utah, United States, 84132 Less <<
NCT01706965 Schizophrenia ... More >> Schizoaffective Disorder Less << Phase 1 Phase 2 Completed - United States, New York ... More >> New York State Psychiatric Institute New York, New York, United States, 10032 Less <<
NCT01706965 - Completed - -
NCT03493412 Peripheral Artery Disease ... More >> Peripheral Arterial Disease Less << Phase 4 Not yet recruiting December 2018 -
NCT00423280 Coronary Artery Disease Phase 2 Unknown February 2009 United Kingdom ... More >> Department of Cardiovascular Medicine, University of Oxford Recruiting Oxford, United Kingdom, OX3 9DU Less <<
NCT00688844 - Completed - United States, Georgia ... More >> Emory Hospital, CIN/ACTSI Atlanta, Georgia, United States, 30322 Emory University Genetics Clinic Decatur, Georgia, United States, 30033 Less <<
NCT00688844 - Completed - -
NCT01141595 - Completed - -
NCT01141595 Autism Spectrum Disorder Not Applicable Completed - United States, Arkansas ... More >> Arkansas Children's Hospital Research Institute Little Rock, Arkansas, United States, 72202 Less <<
NCT00445978 Sickle Cell Disease Phase 2 Completed - United States, District of Col... More >>umbia Washington, District of Columbia, United States United States, Georgia Augusta, Georgia, United States Savannah, Georgia, United States United States, Indiana Indianapolis, Indiana, United States United States, Michigan Detroit, Michigan, United States Flint, Michigan, United States United States, New Jersey Hackensack, New Jersey, United States United States, North Carolina Chapel Hill, North Carolina, United States United States, Pennsylvania Philadelphia, Pennsylvania, United States United States, Texas Galveston, Texas, United States United States, Virginia Norfolk, Virginia, United States Richmond, Virginia, United States Less <<
NCT00935753 Behavioral Manifestations of L... More >>esch-Nyhan Disease Less << Phase 2 Phase 3 Withdrawn(Contractual issues) - United States, California ... More >> UCSD Mitochondrial and Metabolic Disease Center San Diego, California, United States, 92103 Less <<
NCT00403494 Intermittent Claudication Phase 2 Completed - United States, Arizona ... More >> Scottsdale, Arizona, United States United States, California Sacramento, California, United States San Diego, California, United States Santa Ana, California, United States Santa Rosa, California, United States United States, Florida Clearwater, Florida, United States Jacksonville, Florida, United States United States, Georgia Conyers, Georgia, United States United States, Indiana Indianapolis, Indiana, United States United States, Maine Auburn, Maine, United States United States, North Carolina Charlotte, North Carolina, United States United States, Oregon Portland, Oregon, United States United States, Tennessee Knoxville, Tennessee, United States United States, Texas Dallas, Texas, United States San Antonio, Texas, United States United States, Virginia Norfolk, Virginia, United States Richmond, Virginia, United States Argentina Buenos Aires, Argentina Corrientes, Argentina Santa Fe, Argentina Less <<
NCT01425528 - Completed - -
NCT00325962 Hypertension Phase 2 Completed - United States, Alabama ... More >> Hoover, Alabama, United States Huntsville, Alabama, United States Mobile, Alabama, United States Muscle Shoals, Alabama, United States United States, Arizona Gilbert, Arizona, United States Phoenix, Arizona, United States United States, California Anaheim, California, United States Burbank, California, United States Cudahy, California, United States Long Beach, California, United States Roseville, California, United States San Francisco, California, United States United States, Colorado Denver, Colorado, United States United States, Florida Pembroke Pines, Florida, United States United States, Georgia Atlanta, Georgia, United States Decatur, Georgia, United States United States, Indiana Indianapolis, Indiana, United States United States, Maine Auburn, Maine, United States Scarborough, Maine, United States United States, Maryland Baltimore, Maryland, United States United States, North Carolina Charlotte, North Carolina, United States United States, Ohio Cincinnati, Ohio, United States United States, Tennessee Knoxville, Tennessee, United States United States, Texas Carrollton, Texas, United States Less <<
NCT02353312 Heart Failure ... More >> Cardiovascular Disease Less << Not Applicable Recruiting December 2018 United States, Rhode Island ... More >> Providence VAMC Recruiting Providence, Rhode Island, United States, 02908 Contact: Wen-Chih Wu, MD    401-273-7100 ext 6237    wen-chih.wu@va.gov    Contact: Melanie Parent, BS    4012737100 ext 6293    melanie.parent@va.gov Less <<
NCT01439555 Alzheimer's Disease Phase 2 Completed - United States, Massachusetts ... More >> UMass Medical School/ UMass Memorial Medical Center Worcester, Massachusetts, United States, 01655 Less <<
NCT00435331 Pulmonary Arterial Hypertensio... More >>n Less << Phase 1 Completed - United States, Tennessee ... More >> Vanderbilt Medical Center Nashville, Tennessee, United States, 37232 Less <<
NCT00838435 Phenylketonuria Phase 3 Active, not recruiting January 2020 United States, California ... More >> La Jolla, California, United States Orange, California, United States United States, Florida Tampa, Florida, United States United States, Illinois Chicago, Illinois, United States United States, Massachusetts Boston, Massachusetts, United States United States, Ohio Cleveland, Ohio, United States Columbus, Ohio, United States United States, Pennsylvania Hershey, Pennsylvania, United States United States, Tennessee Nashville, Tennessee, United States United States, Utah Salt Lake City, Utah, United States United States, Wisconsin Milwaukee, Wisconsin, United States Canada, Alberta Edmonton, Alberta, Canada Canada, British Columbia Vancouver, British Columbia, Canada Canada, Manitoba Winnipeg, Manitoba, Canada Canada, Ontario Hamilton, Ontario, Canada Toronto, Ontario, Canada Canada, Quebec Montreal, Quebec, Canada Sainte-Fo, Quebec, Canada Less <<
NCT01395394 - Terminated(Difficulty with rec... More >>ruitment and agreed with Sponsor to report completed data as case reports) Less << - -
NCT01082328 Phenylketonuria Phase 4 Completed - Norway ... More >> Department of Paediatric Research, Division of Paediatrics, Oslo University Hospital, Rikshospitalet Oslo, Norway Less <<
NCT01395394 Phenylketonuria Phase 2 Terminated(Difficulty with rec... More >>ruitment and agreed with Sponsor to report completed data as case reports) Less << - United States, Georgia ... More >> Emory University Decatur, Georgia, United States, 30033 Less <<
NCT00964236 - Completed - United States, Missouri ... More >> University of Missouri Columbia, Missouri, United States, 65211 Less <<
NCT00943579 Autistic Disorder Phase 2 Phase 3 Completed - United States, California ... More >> The Children's Health Council Palo Alto, California, United States, 94304 Less <<
NCT01965912 Phenylketonuria Phase 4 Active, not recruiting June 2023 Germany ... More >> Research Site Düsseldorf, Germany Research site Munich, Germany Research site Münster, Germany Italy Research site Bologna, Italy Research site Milano, Italy Research site Padova, Italy Research site Roma, Italy Research site Rome, Italy Spain Research site Baracaldo Bilbao, Spain Research Site Barcelona, Spain Research site Murcia, Spain Research site Santiago de Compostela, Spain United Kingdom Research Site Belfast, United Kingdom Research site Birmingham, United Kingdom Research site Bristol, United Kingdom Research Site - Evelina London, United Kingdom Research site - GOSH London, United Kingdom Less <<
NCT00986973 Phenylketonuria Not Applicable Completed - United States, Pennsylvania ... More >> Children's Hospital of Philadelphia, Section of Metabolism,PKU program Philadelphia, Pennsylvania, United States, 19106 Less <<
NCT02297347 - Completed - United States, Massachusetts ... More >> Boston Children's Hospital Boston, Massachusetts, United States, 02115 Less <<
NCT01082328 - Completed - -
NCT00104247 Phenylketonurias Phase 3 Completed - United States, California ... More >> Los Angeles, California, United States Oakland, California, United States United States, Connecticut New Haven, Connecticut, United States United States, Illinois Chicago, Illinois, United States United States, Massachusetts Boston, Massachusetts, United States United States, Minnesota Minneapolis, Minnesota, United States United States, Missouri St. Louis, Missouri, United States United States, New York New York, New York, United States United States, Oregon Portland, Oregon, United States United States, Pennsylvania Pittsburgh, Pennsylvania, United States United States, Texas Dallas, Texas, United States United States, Utah Salt Lake City, Utah, United States United States, Wisconsin Madison, Wisconsin, United States Less <<
NCT00355264 Tetrahydrobiopterin Deficienci... More >>es Hyperphenylalaninemia, Non-Phenylketonuric Less << Phase 2 Completed - United States, California ... More >> Los Angeles, California, United States, 90095 United States, Illinois Chicago, Illinois, United States, 60614 United States, Minnesota Minneapolis, Minnesota, United States, 55455 United States, New York New York, New York, United States, 10029 United States, North Carolina Chapel Hill, North Carolina, United States, 27599 United States, Oregon Portland, Oregon, United States, 97239 United States, Utah Salt Lake City, Utah, United States, 84132 United States, Washington Seattle, Washington, United States, 98195 United States, Wisconsin Madison, Wisconsin, United States, 53705 Less <<
NCT00986973 - Completed - -
NCT01650909 - Withdrawn(Withdrawn due to no ... More >>funding) Less << - -
NCT00104247 - Completed - -
NCT01376908 Phenylketonuria Phase 3 Completed - Austria ... More >> Research Site Graz, Austria Research site Innsbruck, Austria Belgium Research Site Bruxelles, Belgium Research Site Edegem, Belgium Czechia Research Site Praha 10, Czechia Germany Research site Heidelberg, Germany Research Site Munich, Germany Research Site Münster, Germany Research Site Reutlingen, Germany Italy Research Site Bologna, Italy Research Site Milano, Italy Research Site Padova, Italy Research Site Roma, Italy Research site Rome, Italy Netherlands Research Site Amsterdam, Netherlands Research Site Maastricht, Netherlands Slovakia Research Site Banska Bystrica, Slovakia Research Site Bratislava, Slovakia Research Site Kosice, Slovakia Turkey Research Site Ankara, Turkey United Kingdom Research Site Birmingham, United Kingdom Research site London, United Kingdom Less <<
NCT00332189 - Completed - -
NCT00943579 - Completed - -
NCT00850070 Autistic Disorder Phase 2 Completed - United States, California ... More >> The Children's Health Council Palo Alto, California, United States, 94304 Less <<
NCT00225615 Phenylketonurias Phase 3 Completed - United States, California ... More >> Los Angeles, California, United States Oakland, California, United States Sacramento, California, United States San Jose, California, United States United States, Connecticut New Haven, Connecticut, United States United States, Georgia Atlanta, Georgia, United States United States, Illinois Chicago, Illinois, United States United States, Minnesota Minneapolis, Minnesota, United States United States, Missouri St. Louis, Missouri, United States United States, New York New York, New York, United States United States, Oregon Portland, Oregon, United States United States, Texas Dallas, Texas, United States United States, Utah Salt Lake City, Utah, United States United States, Wisconsin Madison, Wisconsin, United States Less <<
NCT01376908 - Completed - -
NCT01456286 Liver Cirrhosis ... More >> Portal Hypertension Less << Phase 2 Phase 3 Completed - Spain ... More >> Hospital Ramón y Cajal Madrid, Madrid Community, Spain, 28034 Hospital Clínic de Barcelona Barcelona, Spain, 08036 Less <<
NCT00332189 Phenylketonuria Phase 3 Completed - United States, California ... More >> Los Angeles, California, United States Sacramento, California, United States San Jose, California, United States United States, Connecticut New Haven, Connecticut, United States United States, Georgia Atlanta, Georgia, United States United States, Illinois Chicago, Illinois, United States United States, Minnesota Minneapolis, Minnesota, United States United States, Missouri St. Louis, Missouri, United States United States, New York New York, New York, United States United States, Oregon Portland, Oregon, United States United States, Texas Dallas, Texas, United States United States, Utah Salt Lake City, Utah, United States United States, Washington Seattle, Washington, United States United States, Wisconsin Madison, Wisconsin, United States Less <<
NCT01977820 Phenylketonuria Phase 2 Terminated(Recruitment challen... More >>ges) Less << - Italy ... More >> Research Site Bologna, Italy Switzerland Research site Lausanne, Switzerland Less <<
NCT00104260 Phenylketonurias Phase 2 Completed - United States, California ... More >> Los Angeles, California, United States Oakland, California, United States United States, Connecticut New Haven, Connecticut, United States United States, Illinois Chicago, Illinois, United States United States, Massachusetts Boston, Massachusetts, United States United States, Minnesota Minneapolis, Minnesota, United States United States, Missouri St. Louis, Missouri, United States United States, New York New York, New York, United States United States, Oregon Portland, Oregon, United States United States, Pennsylvania Pittsburgh, Pennsylvania, United States United States, Texas Dallas, Texas, United States United States, Utah Salt Lake City, Utah, United States United States, Wisconsin Madison, Wisconsin, United States Less <<
NCT01977820 - Terminated(Recruitment challen... More >>ges) Less << - -
NCT00272792 - Completed - -
NCT00850070 - Completed - -
NCT00484991 Phenylketonuria APPROVED_FOR_MARKETING - Los Angeles, California, Unite... More >>d States|Orange, California, United States|San Diego, California, United States|Stanford, California, United States|Denver, Colorado, United States|Gainseville, Florida, United States|Tampa, Florida, United States|Chicago, Illinois, United States|Indianapolis, Indiana, United States|Louisville, Kentucky, United States|New Orleans, Louisiana, United States|Bangor, Maine, United States|Boston, Massachusetts, United States|Minneapolis, Minnesota, United States|Columbia, Missouri, United States|Kansas City, Missouri, United States|St. Louis, Missouri, United States|Newark, New Jersey, United States|Manhasset, New York, United States|Rochester, New York, United States|Stony Brook, New York, United States|Valhalla, New York, United States|Akron, Ohio, United States|Columbus, Ohio, United States|Hershey, Pennsylvania, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Florence, South Carolina, United States|Sioux Falls, South Dakota, United States|Galveston, Texas, United States|Houston, Texas, United States|Salt Lake City, Utah, United States|Charlottesville, Virginia, United States|Norfolk, Virginia, United States|Madison, Wisconsin, United States|Milwakee, Wisconsin, United States Less <<
NCT01274026 - Completed - United States, Louisiana ... More >> Tulane University Health Science Center New Orleans, Louisiana, United States, 70112 Less <<
NCT01412437 PKU Not Applicable Withdrawn(poor recruitment) - United States, District of Col... More >>umbia Children's Research Institute Washington, District of Columbia, United States, 20010 Less <<
NCT01135186 Gastroparesis Not Applicable Completed - United States, California ... More >> Stanford University School of Medicine Stanford, California, United States, 94305 Less <<
NCT00272792 Phenylketonurias Phase 3 Completed - United States, California ... More >> Los Angeles, California, United States Oakland, California, United States Sacramento, California, United States San Jose, California, United States United States, Illinois Chicago, Illinois, United States United States, Massachusetts Boston, Massachusetts, United States United States, Minnesota Minneapolis, Minnesota, United States United States, Missouri St. Louis, Missouri, United States United States, Utah Salt Lake City, Utah, United States Less <<
NCT01114737 Phenylketonuria Phase 3 Completed - -
NCT00789568 Phenylketonuria Phase 1 Completed - United States, North Dakota ... More >> PRACS Institute, Ltd. Fargo, North Dakota, United States, 58104 Less <<
NCT00730080 - Completed - United States, Missouri ... More >> Washington University Saint Louis, Missouri, United States, 63130 Less <<
NCT01732471 Phenylketonuria Phase 3 Completed - Germany ... More >> Please contact Merck KGaA Communication Center Darmstadt, Germany Less <<
NCT01732471 - Completed - -
NCT01114737 - Completed - -
NCT01135186 - Completed - -
NCT04227080 Pku Phenylketonuria|PAH Defici... More >>ency Less << PHASE4 UNKNOWN 2025-06-22 -

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

3.18mL

0.64mL

0.32mL

15.91mL

3.18mL

1.59mL

31.83mL

6.37mL

3.18mL

Dissolving Methods
The prepared working fluid is recommended to be prepared now and used up as soon as possible in a short period of time. The percentage shown in front of the following solvent refers to the volume ratio of the solvent in the final solution; If precipitation or precipitation occurs in the preparation process, it can be assisted by heating and/or ultrasound.
Protocol 1

References

[1]Yildiz Y, Dursun A, et al. Partial hydatidiform mole in a phenylketonuria patient treated with sapropterin dihydrochloride. Gynecol Endocrinol. 2017 Jan;33(1):19-20.

[2]unal o, Gokmen-ozel H, et al. Sapropterin dihydrochloride treatment in Turkish hyperphenylalaninemic patients under age four. Turk J Pediatr. 2015 May-Jun;57(3):213-8.

[3]Harding CO. New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride. Biologics. 2010 Aug 9;4:231-6

[4]Muntau AC, Adams DJ, Bélanger-Quintana A, Bushueva TV, Cerone R, Chien YH, Chiesa A, Coşkun T, de Las Heras J, Feillet F, Katz R, Lagler F, Piazzon F, Rohr F, van Spronsen FJ, Vargas P, Wilcox G, Bhattacharya K. International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria. Mol Genet Metab. 2019 May;127(1):1-11

[5]Clinical Review Report: Sapropterin dihydrochloride (Kuvan) [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2017 Sep

[6]Hawks Z, Shimony J, Rutlin J, Grange DK, Christ SE, White DA. Pretreatment cognitive and neural differences between sapropterin dihydrochloride responders and non-responders with phenylketonuria. Mol Genet Metab Rep. 2017 Feb 23;12:8-13

[7]Dikalova A, Aschner JL, Kaplowitz MR, Summar M, Fike CD. Tetrahydrobiopterin oral therapy recouples eNOS and ameliorates chronic hypoxia-induced pulmonary hypertension in newborn pigs. Am J Physiol Lung Cell Mol Physiol. 2016 Oct 1;311(4):L743-L753

 

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