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_ gender	Target_ size	Phase	Countries
1	EUCTR2009-018168-81-DK (EUCTR)	16/04/2010	03/03/2010	ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE	ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE	Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)). MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders	Trade Name: Kuvan INN or Proposed INN: Sapropterindihydrochloride Other descriptive name: SAPROPTERIN HYDROCHLORIDE	Merck Serono Norway	NULL	Not Recruiting	Female: yes Male: yes	70	Phase 4	Denmark
2	EUCTR2004-002071-16-IT (EUCTR)	17/03/2005	27/04/2005	A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels	A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels	Treatment of Phenylketonuria MedDRA version: 6.1;Level: SOC;Classification code 10027433	Product Name: Phenoptin Product Code: NA INN or Proposed INN: sapropterin hydrochloride Other descriptive name: NA	BIOMARIN	NULL	Not Recruiting	Female: yes Male: yes		Phase 2	United Kingdom;Germany;Italy
3	EUCTR2004-002071-16-DE (EUCTR)	28/01/2005	14/10/2004	A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA	A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA	Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872	Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin , sapropterin hydrochloride	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting	Female: yes Male: yes	700	Phase 2	United Kingdom;Germany;Italy
4	EUCTR2009-018168-81-NO (EUCTR)		10/03/2010	ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE	ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE	Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)). MedDRA version: 12.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU)	Trade Name: Kuvan INN or Proposed INN: Sapropterindihydrochloride Other descriptive name: SAPROPTERIN HYDROCHLORIDE	Merck Serono Norway	NULL	NA	Female: yes Male: yes	150	Phase 4	Denmark;Norway
5	EUCTR2004-004512-23-DE (EUCTR)		17/03/2005	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA	Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872	Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin , sapropterin hydrochloride	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting	Female: yes Male: yes	100	Phase 3	United Kingdom;Germany;Ireland;Italy

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

EUCTR2009-018168-81-DK
(EUCTR)

16/04/2010

03/03/2010

ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE

Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders

Trade Name: Kuvan
INN or Proposed INN: Sapropterindihydrochloride
Other descriptive name: SAPROPTERIN HYDROCHLORIDE

Merck Serono Norway

NULL

Not Recruiting

Female: yes
Male: yes

Phase 4

Denmark

EUCTR2004-002071-16-IT
(EUCTR)

17/03/2005

27/04/2005

A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels

Treatment of Phenylketonuria
MedDRA version: 6.1;Level: SOC;Classification code 10027433

Product Name: Phenoptin
Product Code: NA
INN or Proposed INN: sapropterin hydrochloride
Other descriptive name: NA

BIOMARIN

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United Kingdom;Germany;Italy

EUCTR2004-002071-16-DE
(EUCTR)

28/01/2005

14/10/2004

A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA

Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872

Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin , sapropterin hydrochloride

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: yes
Male: yes

700

Phase 2

United Kingdom;Germany;Italy

EUCTR2009-018168-81-NO
(EUCTR)

10/03/2010

Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
MedDRA version: 12.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU)

Trade Name: Kuvan
INN or Proposed INN: Sapropterindihydrochloride
Other descriptive name: SAPROPTERIN HYDROCHLORIDE

Merck Serono Norway

NULL

Female: yes
Male: yes

150

Phase 4

Denmark;Norway

EUCTR2004-004512-23-DE
(EUCTR)

17/03/2005

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA

Phenylketonuria (PKU)
MedDRA version: 6.1;Classification code 10034872

Product Name: Phenoptin
Product Code: T1401
INN or Proposed INN: Sapropterin , sapropterin hydrochloride

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 3

United Kingdom;Germany;Ireland;Italy