Ignite Creation Date:
2025-12-24 @ 11:46 AM
Ignite Modification Date:
2025-12-31 @ 1:29 PM
Study NCT ID:
NCT05166161
Status:
RECRUITING
Last Update Posted:
2025-05-08
First Post:
2021-12-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010661', 'term': 'Phenylketonurias'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C016727', 'term': 'sepiapterin'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 200}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-02-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2026-04-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-05-07', 'studyFirstSubmitDate': '2021-12-08', 'studyFirstSubmitQcDate': '2021-12-08', 'lastUpdatePostDateStruct': {'date': '2025-05-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-12-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-04-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Treatment-Emergent Adverse Events (TEAEs)', 'timeFrame': 'Baseline up to end of study (up to 4 years)', 'description': 'A TEAE is any unfavorable or unintended sign (including an abnormal laboratory finding), symptom or disease in a study participant who is administered study drug in this study'}, {'measure': 'Change From Baseline in Dietary Phe/Protein Consumption at Week 26, Measured During Phe Tolerance Assessment Period', 'timeFrame': 'Baseline, Week 26', 'description': 'Phe tolerance is defined as the total amount of dietary Phe (milligrams \\[mg\\]/kilogram \\[kg\\] per day) ingested while maintaining blood Phe levels within the range of 40 to 360 micromoles (μmol)/liter (L) (defined as ≥40 to \\<360 μmol/L).'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Quality of Life (QOL) Using Phenylketonuria-Quality of Life (PKU-QOL) Questionnaire at Months 8, 14, 20, 26, 32, and 38', 'timeFrame': 'Baseline, Months 8, 14, 20, 26, 32, and 38', 'description': 'QOL using PKU-QOL questionnaire will be assessed in the subset of participants who are able to complete the PKU-QOL (that is, participants whose primary language is English \\[British or American\\], Turkish, Dutch, German, Spanish, Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages ≥18 years Adult PKU-QOL).'}, {'measure': 'Change From Baseline in QOL Using the European Quality of Life - 5 Dimensions (EQ-5D) at Months 8, 14, 20, 26, 32, and 38', 'timeFrame': 'Baseline, Months 8, 14, 20, 26, 32, and 38', 'description': 'QOL will be assessed using the EQ-5D (EQ-5D-Y Proxy Version 1 \\[3 to 7 years\\]; EQ-5D-Y \\[8 to 15 years\\]; EQ-5D-5L (\\[≥16 years\\]).'}, {'measure': 'Palatability of PTC923', 'timeFrame': 'Month 1 Day 1', 'description': 'For participants \\<5 years of age, palatability will be indirectly assessed by the parent(s)/caregiver(s) of participants using the following: On the basis of the reaction/facial expression of your child, do you think that the medication is (pleasant, not sure, unpleasant)?'}, {'measure': 'Acceptability/Ease of Administration of PTC923', 'timeFrame': 'Month 1 Day 1', 'description': 'For all participants \\<12 years of age, parent(s)/caregiver(s) will rate the acceptability/ease of administration, with the following question: Do you sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up? (Yes/No).'}, {'measure': 'Taste/Flavor Assessment Using a Facial Hedonic Scale', 'timeFrame': 'Month 1 Day 1', 'description': 'For participants ≥5 to \\<18 years of age who are able to comply with the instructions, the participant will rate the taste/flavor using a facial hedonic scale (5=really good; 4=good; 3=not sure; 2=bad; 1=really bad).'}, {'measure': 'Plasma Sepiapterin Concentration', 'timeFrame': 'Month 1 Day 1 up to Month 11 Day 1'}, {'measure': 'Plasma BH4 Concentration', 'timeFrame': 'Month 1 Day 1 up to Month 11 Day 1'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Phenylketonuria']}, 'descriptionModule': {'briefSummary': 'The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.', 'detailedDescription': 'Eligible participants are:\n\nFeeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study.\n\nNon-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels \\<360 μmol/L at study entry.\n\nNon-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L.\n* Women of childbearing potential must have a negative pregnancy test at screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for up to 90 days after the last dose of the study drug.\n* Males who are sexually active with women of childbearing potential who have not had a vasectomy must agree to use a barrier method of birth control during the study and for up to 90 days after the last dose of study drug. Males must also refrain from sperm donations during this time period.\n* Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator).\n\nExclusion Criteria:\n\n* Inability to tolerate oral medication.\n* A female who is pregnant or breastfeeding, or considering pregnancy.\n* Serious neuropsychiatric illness (for example, major depression) not currently under medical control, that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.\n* Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate \\[GFR\\] \\<60 milliliters \\[mL\\]/minute \\[min\\] min as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist.\n* Any other condition that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.\n* Requirement for concomitant treatment with any drug known to inhibit folate synthesis (for example, methotrexate).\n* Concomitant treatment with tetrahydrobiopterin (BH4) supplementation (for example, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ).\n\nAdditional criteria for non-feeder participants who did not participate in a feeder study:\n\n* Gastrointestinal disease (such as irritable bowel syndrome, inflammatory bowel disease, chronic gastritis, and peptic ulcer disease, etc) that could affect the absorption of study drug.\n* History of gastric surgery, including Roux-en-Y gastric bypass surgery or an antrectomy with vagotomy, or gastrectomy.\n* History of allergies or adverse reactions to synthetic BH4 or sepiapterin.\n* Any clinically significant laboratory abnormality as determined by the investigator.\n* Any abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated GFR \\<60 milliliters (mL)/minute/1.73 square meter (m\\^2).\n\nConfirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive GTP cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin-4-alphacarbinolamine dehydratase genes."}, 'identificationModule': {'nctId': 'NCT05166161', 'briefTitle': 'A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria', 'organization': {'class': 'INDUSTRY', 'fullName': 'PTC Therapeutics'}, 'officialTitle': 'A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria', 'orgStudyIdInfo': {'id': 'PTC923-MD-004-PKU'}, 'secondaryIdInfos': [{'id': '2021-000497-28', 'type': 'EUDRACT_NUMBER'}, {'id': '2023-509229-31-00', 'type': 'OTHER', 'domain': 'CTIS Number'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PTC923', 'description': 'Participants will receive PTC923 7.5 mg/kg (participants 0 to \\<6 months of age), 15 mg/kg (participants 6 to \\<12 months of age), 30 mg/kg (participants 12 months to \\<2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for a minimum of 12 months or until participant experiences lack of efficacy, adverse events (AEs) that lead to discontinuation, withdraws from treatment, or PTC923 is authorized and commercially available in the specific country.', 'interventionNames': ['Drug: PTC923']}], 'interventions': [{'name': 'PTC923', 'type': 'DRUG', 'otherNames': ['Sepiapterin'], 'description': 'PTC923 powder for oral use will be suspended in water or apple juice prior to administration.', 'armGroupLabels': ['PTC923']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Janet Thomas', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Roberto Zori', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Melissa Lah', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Indiana University School of Medicine Department of Medical and Molecular Genetics', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10009', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Dr. Margot Breilyn', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Icahn School of Medicine at Mount Sinai (ISMMS)', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Gerard Vockley', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "UPMC Children's Hospital of Pittsburgh", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Ashley Andrews', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Michel Tchan', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Westmead Hospital', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': 'SA 5000', 'city': 'Adelaide', 'state': 'South Australia', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Drago Bratkovic', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'PARC Clinical Research', 'geoPoint': {'lat': -34.92866, 'lon': 138.59863}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Dr. Heidi Peters', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Royal Children's Hospital", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '90035-903', 'city': 'Porto Alegre', 'state': 'Rio Grande do Sul', 'status': 'RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Ida Schwartz', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital de clinicas de Porto Alegre', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}, {'zip': '14051-140', 'city': 'Ribeirão Preto', 'state': 'São Paulo', 'status': 'RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Maria Avanise Yumi Minami', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo', 'geoPoint': {'lat': -21.1775, 'lon': -47.81028}}, {'zip': 'T2E 7Z4', 'city': 'Calgary', 'state': 'Alberta', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Aneal Khan', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd.', 'geoPoint': {'lat': 51.05011, 'lon': -114.08529}}, {'zip': 'L8N 3Z5', 'city': 'Hamilton', 'state': 'Ontario', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Shaymaa Shurrab', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "McMaster Children's Hospital Hamilton Health Sciences", 'geoPoint': {'lat': 43.25011, 'lon': -79.84963}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Dr. Laura Guilder', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'The Hospital for Sick Children University of Toronto Adult Clinic: The Fred A Litwin Family Centre in Genetic Medicine University Health Network & Mt. Sinai Hospital', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': '100 34', 'city': 'Prague', 'status': 'RECRUITING', 'country': 'Czechia', 'contacts': [{'name': 'Renáta Tycová', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Fakultní nemocnice Královské Vinohrady Klinika dětí a dorostu Ambulance pro léčbu PKU a HPA', 'geoPoint': {'lat': 50.08804, 'lon': 14.42076}}, {'zip': 'DK-2100', 'city': 'Copenhagen', 'status': 'RECRUITING', 'country': 'Denmark', 'contacts': [{'name': 'Allan Lund', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Copenhagen University Hospital, Rigshospitalet', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '75015', 'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Jean-Baptiste Arnoux', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hôpital Necker-Enfants Malades - Centre de Référence des Maladies Héréditaires du Métabolisme', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '0159', 'city': 'Tbilisi', 'status': 'RECRUITING', 'country': 'Georgia', 'contacts': [{'name': 'Lali Margvelashvili', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Medical Genetics and Laboratory Diagnostics Center', 'geoPoint': {'lat': 41.69143, 'lon': 44.83412}}, {'zip': '20246', 'city': 'Hamburg', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Ania Carolina Muntau', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE)", 'geoPoint': {'lat': 53.55073, 'lon': 9.99302}}, {'zip': '69120', 'city': 'Heidelberg', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Thomas Opladen', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin', 'geoPoint': {'lat': 49.40768, 'lon': 8.69079}}, {'zip': '48149', 'city': 'Münster', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Frank Rutsch', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Universitätsklinikum Münster', 'geoPoint': {'lat': 51.96236, 'lon': 7.62571}}, {'zip': '35129', 'city': 'Padua', 'state': 'Veneto', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Alberto Burlina', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Azienda Ospedaliera-Universita Padova', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'zip': '00185', 'city': 'Rome', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Filippo Manti', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Department of Human Neuroscience, Child and Adolescent Neuropsychiatry, Policlinico Umberto I', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'city': 'Multiple Locations', 'status': 'RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Tetsuya Ito', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'PTC Clinical Site 1'}, {'city': 'Multiple Locations', 'status': 'RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Mika Ishige', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'PTC Clinical Site 2'}, {'city': 'Multiple Locations', 'status': 'RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Takashi Hamazaki', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'PTC Clinical Site 3'}, {'zip': '44670', 'city': 'Guadalajara', 'state': 'Jalisco', 'status': 'RECRUITING', 'country': 'Mexico', 'contacts': [{'name': 'Ixiu del Carmen Cabrales Guerra', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'PanAmerican Clinical Research', 'geoPoint': {'lat': 20.67738, 'lon': -103.34749}}, {'zip': '9713 GZ', 'city': 'Groningen', 'status': 'RECRUITING', 'country': 'Netherlands', 'contacts': [{'name': 'Francjan Van Spronsen', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "UMCG Beatrix Children's Hospital", 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}, {'zip': '71-252', 'city': 'Szczecin', 'status': 'RECRUITING', 'country': 'Poland', 'contacts': [{'name': 'Maria Gizewska', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Pomorski Uniwersytet Medyczny w Szczecinie Centrum Wsparcia Badań Klinicznych', 'geoPoint': {'lat': 53.42894, 'lon': 14.55302}}, {'zip': '1649-035', 'city': 'Lisbon', 'state': 'Estremadura', 'status': 'RECRUITING', 'country': 'Portugal', 'contacts': [{'name': 'Patricia Janeiro', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,', 'geoPoint': {'lat': 38.72509, 'lon': -9.1498}}, {'zip': '1649-035', 'city': 'Lisbon', 'state': 'Estremadura', 'status': 'RECRUITING', 'country': 'Portugal', 'contacts': [{'name': 'Anabela Oliveira', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria', 'geoPoint': {'lat': 38.72509, 'lon': -9.1498}}, {'zip': '4099-001', 'city': 'Porto', 'status': 'RECRUITING', 'country': 'Portugal', 'contacts': [{'name': 'Arlindo Guimas', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Centro Hospitalar Universitário Do Porto, Epe', 'geoPoint': {'lat': 41.1485, 'lon': -8.61097}}, {'zip': '1000', 'city': 'Ljubljana', 'status': 'RECRUITING', 'country': 'Slovenia', 'contacts': [{'name': 'Urh Groselj', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Univerzitetni klinicni center Ljubljana', 'geoPoint': {'lat': 46.05108, 'lon': 14.50513}}, {'zip': '08950', 'city': 'Barcelona', 'state': 'Esplugues de Llobregat', 'status': 'RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Jaume Campistol', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28034', 'city': 'Madrid', 'status': 'RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Amaya Belanger-Quintana', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital Universitario Ramón y Cajal', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '41013', 'city': 'Seville', 'status': 'RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Eva Venegas Moreno', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital Universitario Virgen del Rocío', 'geoPoint': {'lat': 37.38283, 'lon': -5.97317}}, {'zip': '06230', 'city': 'Altındağ', 'state': 'Ankara', 'status': 'RECRUITING', 'country': 'Turkey (Türkiye)', 'contacts': [{'name': 'Serap Sivri', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hacettepe University Medical Faculty', 'geoPoint': {'lat': 39.96673, 'lon': 32.92254}}, {'zip': '06500', 'city': 'Yenimahalle', 'state': 'Ankara', 'status': 'RECRUITING', 'country': 'Turkey (Türkiye)', 'contacts': [{'name': 'Fatih Ezgu', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Gazi Üniversitesi Tıp Fakültesi', 'geoPoint': {'lat': 39.97787, 'lon': 32.80147}}, {'zip': '34098', 'city': 'Fatih', 'state': 'Istanbul', 'status': 'RECRUITING', 'country': 'Turkey (Türkiye)', 'contacts': [{'name': 'Ertugrul Kiykim', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi', 'geoPoint': {'lat': 41.0225, 'lon': 28.94083}}, {'zip': '35100', 'city': 'Bornova', 'state': 'İzmir', 'status': 'RECRUITING', 'country': 'Turkey (Türkiye)', 'contacts': [{'name': 'Ebru Canda', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Ege University Faculty of Medicine Children Hospital', 'geoPoint': {'lat': 38.47921, 'lon': 27.2399}}, {'zip': '01330', 'city': 'Adana', 'status': 'RECRUITING', 'country': 'Turkey (Türkiye)', 'contacts': [{'name': 'Neslihan Mungan', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi', 'geoPoint': {'lat': 36.98615, 'lon': 35.32531}}, {'zip': 'B4 6NH', 'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Suresh Vijay', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Birmingham Children's Hospital NHS Foundation Trust", 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Tarekegn Hiwot', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'WC1N 3JH', 'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Anupam Chakrapani', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'Patient Advocacy', 'role': 'CONTACT', 'email': 'medinfo@ptcbio.com', 'phone': '1-866-562-4620'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'PTC Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}