337. Homocystinuria Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 23 / Drugs : 31 - (DrugBank : 11) / Drug target genes : 3 - Drug target pathways : 26
Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
5-mthf
Zambon Group S.p.A
2007 - EUCTR2006-000614-21-AT Austria;Hungary;Italy;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-SK Austria;Hungary;Slovakia;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-HU Austria;Hungary;Slovakia;United Kingdom
2006 - EUCTR2006-000614-21-GB Austria;Hungary;Italy;United Kingdom
Acetaminophen
Hospital de Clinicas de Porto Alegre
2022 Phase 1/Phase 2 NCT04015557 Brazil
ACN00177
Aeglea Biotherapeutics, Inc.
2020 Phase 1 EUCTR2019-004791-19-GB United Kingdom
Betaine
Assistance Publique - Hôpitaux de Paris
2015 Phase 2 NCT02404337 France
Creatine
University of British Columbia
2024 - NCT06495567 Canada
Cysteine
Hamad Medical Corporation
2024 - NCT06622577 Qatar
Detoxifying agents FOR antineoplastic treatment
ZAMBON GROUP
2006 - EUCTR2006-000614-21-IT Austria;Hungary;Italy;United Kingdom
Dietary methionine tracer and protein intake
University of British Columbia
2024 - NCT06495567 Canada
EPI-743
Bambino Gesù Hospital and Research Institute
2013 Phase 2 NCT01793090 Italy
Hydroxocobalamin chloride injection
CSPC ZhongQi Pharmaceutical Technology Co., Ltd.
2025 Phase 3 NCT07163364 -
Iupac name: 2-[4-[(2-amino-5-methyl-4-OXO-1,6,7,8-tetrahydropteridin-6-YL)methylam INO]benzoyl]amino
Zambon Group S.p.A
2007 - EUCTR2006-000614-21-AT Austria;Hungary;Italy;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-SK Austria;Hungary;Slovakia;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-HU Austria;Hungary;Slovakia;United Kingdom
2006 - EUCTR2006-000614-21-GB Austria;Hungary;Italy;United Kingdom
Methylenetetrahydrofolate reductase (mthfr) deficiency
Istanbul Medeniyet University
2019 - NCT04287881 Turkey
N-acetylcysteine
Hospital de Clinicas de Porto Alegre
2022 Phase 1/Phase 2 NCT04015557 Brazil
McGill University Health Center
2007 Phase 2 NCT00483314 Canada
Obtaining DRY blood spots from healthy children aged 8 days - 7 years
West Kazakhstan Medical University
2022 - NCT05910151 Kazakhstan
Obtaining DRY blood spots from healthy children aged 8-18 years
West Kazakhstan Medical University
2022 - NCT05910151 Kazakhstan
Pegtarviliase IV
Aeglea Biotherapeutics
2021 Phase 1/Phase 2 NCT05154890 Australia;United Kingdom;United States
Pegtarviliase SC
Aeglea Biotherapeutics
2021 Phase 1/Phase 2 NCT05154890 Australia;United Kingdom;United States
Pegtibatinase
Travere Therapeutics, Inc.
2024 Phase 3 NCT06431893 Australia;Belgium;France;Germany;Ireland;Italy;Poland;Portugal;Qatar;Saudi Arabia;Spain;Turkey (Türkiye);United Kingdom;United States
2023 Phase 3 NCT06247085 Australia;Belgium;France;Germany;Ireland;Italy;Poland;Portugal;Qatar;Saudi Arabia;Spain;Turkey (Türkiye);United Kingdom;United States
2019 Phase 1/Phase 2 NCT03406611 United States
PKU express plus
Vitaflo International, Ltd
2021 - NCT05051657 United Kingdom
Prefolic
Zambon Group S.p.A
2007 - EUCTR2006-000614-21-AT Austria;Hungary;Italy;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-SK Austria;Hungary;Slovakia;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-HU Austria;Hungary;Slovakia;United Kingdom
2006 - EUCTR2006-000614-21-GB Austria;Hungary;Italy;United Kingdom
Prefolic 15*30CPR gastror 15MG
ZAMBON GROUP
2006 - EUCTR2006-000614-21-IT Austria;Hungary;Italy;United Kingdom
SYNB1353
Synlogic
2022 Phase 1 NCT05462132 United States
Taurine
University of Colorado, Denver
2010 Phase 1/Phase 2 NCT01192828 United States
Zambon Group S.p.A
2007 - EUCTR2006-000614-21-AT Austria;Hungary;Italy;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-SK Austria;Hungary;Slovakia;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-HU Austria;Hungary;Slovakia;United Kingdom
2006 - EUCTR2006-000614-21-GB Austria;Hungary;Italy;United Kingdom
Acetaminophen
Hospital de Clinicas de Porto Alegre
2022 Phase 1/Phase 2 NCT04015557 Brazil
ACN00177
Aeglea Biotherapeutics, Inc.
2020 Phase 1 EUCTR2019-004791-19-GB United Kingdom
Betaine
Assistance Publique - Hôpitaux de Paris
2015 Phase 2 NCT02404337 France
Creatine
University of British Columbia
2024 - NCT06495567 Canada
Cysteine
Hamad Medical Corporation
2024 - NCT06622577 Qatar
Detoxifying agents FOR antineoplastic treatment
ZAMBON GROUP
2006 - EUCTR2006-000614-21-IT Austria;Hungary;Italy;United Kingdom
Dietary methionine tracer and protein intake
University of British Columbia
2024 - NCT06495567 Canada
EPI-743
Bambino Gesù Hospital and Research Institute
2013 Phase 2 NCT01793090 Italy
Hydroxocobalamin chloride injection
CSPC ZhongQi Pharmaceutical Technology Co., Ltd.
2025 Phase 3 NCT07163364 -
Iupac name: 2-[4-[(2-amino-5-methyl-4-OXO-1,6,7,8-tetrahydropteridin-6-YL)methylam INO]benzoyl]amino
Zambon Group S.p.A
2007 - EUCTR2006-000614-21-AT Austria;Hungary;Italy;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-SK Austria;Hungary;Slovakia;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-HU Austria;Hungary;Slovakia;United Kingdom
2006 - EUCTR2006-000614-21-GB Austria;Hungary;Italy;United Kingdom
Methylenetetrahydrofolate reductase (mthfr) deficiency
Istanbul Medeniyet University
2019 - NCT04287881 Turkey
N-acetylcysteine
Hospital de Clinicas de Porto Alegre
2022 Phase 1/Phase 2 NCT04015557 Brazil
McGill University Health Center
2007 Phase 2 NCT00483314 Canada
Obtaining DRY blood spots from healthy children aged 8 days - 7 years
West Kazakhstan Medical University
2022 - NCT05910151 Kazakhstan
Obtaining DRY blood spots from healthy children aged 8-18 years
West Kazakhstan Medical University
2022 - NCT05910151 Kazakhstan
Pegtarviliase IV
Aeglea Biotherapeutics
2021 Phase 1/Phase 2 NCT05154890 Australia;United Kingdom;United States
Pegtarviliase SC
Aeglea Biotherapeutics
2021 Phase 1/Phase 2 NCT05154890 Australia;United Kingdom;United States
Pegtibatinase
Travere Therapeutics, Inc.
2024 Phase 3 NCT06431893 Australia;Belgium;France;Germany;Ireland;Italy;Poland;Portugal;Qatar;Saudi Arabia;Spain;Turkey (Türkiye);United Kingdom;United States
2023 Phase 3 NCT06247085 Australia;Belgium;France;Germany;Ireland;Italy;Poland;Portugal;Qatar;Saudi Arabia;Spain;Turkey (Türkiye);United Kingdom;United States
2019 Phase 1/Phase 2 NCT03406611 United States
PKU express plus
Vitaflo International, Ltd
2021 - NCT05051657 United Kingdom
Prefolic
Zambon Group S.p.A
2007 - EUCTR2006-000614-21-AT Austria;Hungary;Italy;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-SK Austria;Hungary;Slovakia;United Kingdom
2006 Phase 2 EUCTR2006-000614-21-HU Austria;Hungary;Slovakia;United Kingdom
2006 - EUCTR2006-000614-21-GB Austria;Hungary;Italy;United Kingdom
Prefolic 15*30CPR gastror 15MG
ZAMBON GROUP
2006 - EUCTR2006-000614-21-IT Austria;Hungary;Italy;United Kingdom
SYNB1353
Synlogic
2022 Phase 1 NCT05462132 United States
Taurine
University of Colorado, Denver
2010 Phase 1/Phase 2 NCT01192828 United States
