DDrare: Database of Drug Development for Rare Diseases

Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 5,6,7,8-tetrahydrobiopterin; 6R-BH4; Amino acids; BH4; BMN 165; BMN 165 (rAvPAL-PEG); BMN 307; BMN165 20mg/day; BMN165 40mg/day; Behavioral: Neuro-psychological Assessment; Behavioral: Restricted phenylalanine diet; Behavioral: phenylalanine restricted diet; CDX; CDX 6114; CNSA-001; Cohort 1 0.225g; Cohort 2 0.75g; Cohort 3 2.25 g; Corn; Corn/soy oil; Diagnostic Test: Whole body Phe oxidation testing; Diet; Dietary Supplement for PKU patients; Docosahexaenoic Acid; Docosahexaenoic acid; Free amino acids intake; Glycomacropeptide-based protein substitute; High oleic sunflower oil; Immunosuppression; KUVAN; Kuvan; Kuvan 100mg soluble tablet; KuvanTM Therapy; Kuvan®; Kuvan® (sapropterin dihydrochloride); Large Neutral Amino Acid Therapy; Low calorie protein substitute; Microalgal oil; Moxifloxacin; N.a.; NAP; Other: Diet; Other: Diet treatment; Other: Liver Evaluation; Other: Meal Challenge; Other: Normal (control) diet; Other: Phenylalanine (Phe)-restricted diet; Other: Routine; PEG; PEGVALIASE; PKU Explore; PKU Sphere; PKU Start; PKU sphere; Palynziq; Pegvaliase; Phe; Phenoptin; Phenylalanine; Phenylalanine restricted diet; Phenylalanine-free protein substitute in tablet form (XPhe minis); Phenylalanine-free protein substitute tablets; Phenylketonuria-type diet; Procedure: Hepatocyte Transplant; Procedure: Liver Biopsy; Protein intake; RAvPAL-PEG; RAvPAL-PEG 0.001 mg/kg; RAvPAL-PEG 0.003 mg/kg; RAvPAL-PEG 0.01 mg/kg; RAvPAL-PEG 0.03 mg/kg; RAvPAL-PEG 0.1 mg/kg; RTX; RTX-134; Radiation: Preparative Radiation Therapy; SAPROPTERIN; SAPROPTERIN DIHYDROCHLORIDE; SAPROPTERIN HYDROCHLORIDE; SYNB1618; Sapropterin; Sapropterin (Kuvan); Sapropterin Dihydrochloride; Sapropterin Dihydrocholoride; Sapropterin dihydrochloride; Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin; Sapropterin hydrochloride; Sapropterin, sapropterin dihydrochloride; Sapropterin, sapropterin hydrochloride; Sapropterindihydrochloride; Saproterin dihydrochloride; Sunflower oil; Synergy; T1401; Tetrahydrobiopterin; Tetrahydrobiopterin (BH4)

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01560286 (ClinicalTrials.gov)	May 2012	5/3/2012	A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks	A Phase II, Multi-center, Open-label, Dose-finding Study to Evaluate Safety, Efficacy and Tolerability of Subcutaneously (SC) Administered rAvPAL-PEG in Patients With PKU for 24 Weeks	Phenylketonuria	Biological: BMN 165 (rAvPAL-PEG)	BioMarin Pharmaceutical	NULL	Completed	16 Years	70 Years	All	24	Phase 2	United States
2	NCT01212744 (ClinicalTrials.gov)	March 2011	29/9/2010	Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)	A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria	Phenylketonuria	Drug: rAvPAL-PEG	BioMarin Pharmaceutical	NULL	Completed	16 Years	70 Years	All	16	Phase 2	United States
3	NCT00924703 (ClinicalTrials.gov)	October 8, 2009	18/6/2009	Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)	Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKU	Phenylketonuria	Drug: rAvPAL-PEG	BioMarin Pharmaceutical	NULL	Completed	16 Years	55 Years	All	68	Phase 2	United States
4	NCT00925054 (ClinicalTrials.gov)	September 2009	17/6/2009	Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU	Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU	Phenylketonuria	Drug: rAvPAL-PEG 0.001 mg/kg;Drug: rAvPAL-PEG 0.003 mg/kg;Drug: rAvPAL-PEG 0.01 mg/kg;Drug: rAvPAL-PEG 0.03 mg/kg;Drug: rAvPAL-PEG 0.1 mg/kg	BioMarin Pharmaceutical	NULL	Completed	16 Years	55 Years	All	40	Phase 2	United States
5	NCT00634660 (ClinicalTrials.gov)	May 2008	6/3/2008	Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria	A Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single, Subcutaneous Doses of rAvPAL-PEG in Subjects With Phenylketonuria	Phenylketonuria	Drug: rAvPAL-PEG	BioMarin Pharmaceutical	NULL	Completed	16 Years	50 Years	All	25	Phase 1	United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT01560286
(ClinicalTrials.gov)

May 2012

5/3/2012

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

A Phase II, Multi-center, Open-label, Dose-finding Study to Evaluate Safety, Efficacy and Tolerability of Subcutaneously (SC) Administered rAvPAL-PEG in Patients With PKU for 24 Weeks

Phenylketonuria

Biological: BMN 165 (rAvPAL-PEG)

BioMarin Pharmaceutical

NULL

Completed

16 Years

70 Years

All

Phase 2

United States

NCT01212744
(ClinicalTrials.gov)

March 2011

29/9/2010

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria

Phenylketonuria

Drug: rAvPAL-PEG

BioMarin Pharmaceutical

NULL

Completed

16 Years

70 Years

All

Phase 2

United States

NCT00924703
(ClinicalTrials.gov)

October 8, 2009

18/6/2009

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKU

Phenylketonuria

Drug: rAvPAL-PEG

BioMarin Pharmaceutical

NULL

Completed

16 Years

55 Years

All

Phase 2

United States

NCT00925054
(ClinicalTrials.gov)

September 2009

17/6/2009

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU

Phenylketonuria

Drug: rAvPAL-PEG 0.001 mg/kg;Drug: rAvPAL-PEG 0.003 mg/kg;Drug: rAvPAL-PEG 0.01 mg/kg;Drug: rAvPAL-PEG 0.03 mg/kg;Drug: rAvPAL-PEG 0.1 mg/kg

BioMarin Pharmaceutical

NULL

Completed

16 Years

55 Years

All

Phase 2

United States

NCT00634660
(ClinicalTrials.gov)

May 2008

6/3/2008

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

A Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single, Subcutaneous Doses of rAvPAL-PEG in Subjects With Phenylketonuria

Phenylketonuria

Drug: rAvPAL-PEG

BioMarin Pharmaceutical

NULL

Completed

16 Years

50 Years

All

Phase 1

United States