IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease2

19. Lysosomal storage disease


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-004909-27-NL
(EUCTR)
11/05/202103/09/2020STUDY TO EVALUATE THE SAFETY AND ESTABLISH A SAFE DOSE OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROMEA PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II])
MedDRA version: 20.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: DNL310
Product Code: DNL310 Drug Substance
INN or Proposed INN: Not applicable
Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR
Denali Therapeutics Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
16Phase 1;Phase 2United States;Germany;Netherlands;United Kingdom;Italy
2EUCTR2019-004909-27-GB
(EUCTR)
22/07/202001/05/2020STUDY TO EVALUATE THE SAFETY AND ESTABLISH A SAFE DOSE OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROMEA PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II])
MedDRA version: 20.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: DNL310
Product Code: DNL310 Drug Substance
INN or Proposed INN: Not applicable
Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR
Denali Therapeutics Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
16Phase 1;Phase 2United States;Netherlands;Germany;Italy;United Kingdom