251. Urea cycle disorder
42 clinical trials,   45 drugs   (DrugBank: 10 drugs),   4 drug target genes,   29 drug target pathways

Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
6 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-004842-40-FR
(EUCTR)
14/01/202018/11/2019International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs.A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Kaleido BiosciencesNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
29Phase 2United States;France;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
2EUCTR2018-004842-40-DE
(EUCTR)
25/11/201904/06/2019International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs. A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Kaleido BiosciencesNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
29Phase 2France;United States;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
3EUCTR2018-004842-40-ES
(EUCTR)
06/11/201911/06/2019International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs.A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: KB195
Product Code: KB195
INN or Proposed INN: KB195
Kaleido BiosciencesNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
29Phase 2France;United States;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
4NCT03933410
(ClinicalTrials.gov)
September 17, 201929/4/2019UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle DisorderA Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A Urea Cycle Disorder With Inadequate Control on Standard of CareUrea Cycle DisorderDrug: KB195Kaleido BiosciencesNULLRecruiting12 Years70 YearsAll24Phase 2United States;Belgium;Germany;Spain;Switzerland;Turkey;United Kingdom;Mexico
5EUCTR2018-004842-40-GB
(EUCTR)
30/08/201922/10/2020International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs.A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha Urea Cycle Disorder with Inadequate Control on Standard of Care - Kaleido K020-218 Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: KB195
Product Code: KB195
INN or Proposed INN: KB195
Kaleido BiosciencesNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
29Phase 1;Phase 2United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;Switzerland;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2018-004842-40-BE
(EUCTR)
21/08/201928/05/2019International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs.A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha Urea Cycle Disorder with Inadequate Control on Standard of Care Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
MedDRA version: 20.1;Level: PT;Classification code 10080020;Term: Urea cycle disorder;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: KB195
Product Code: KB195
INN or Proposed INN: KB195
Kaleido BiosciencesNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
29Phase 2United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Germany;United Kingdom;Switzerland