19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
4 trials found
No.TrialIDDate_
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PhaseCountries
1EUCTR2017-002806-10-NL
(EUCTR)
29/11/201908/11/2018An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patientsAn open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome
MedDRA version: 20.1;Level: LLT;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Chemically modified recombinant human sulfamidase
Product Code: SOBI003
INN or Proposed INN: not applicable
Other descriptive name: CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE
Swedish Orphan Biovitrum AB (publ)NULLNot RecruitingFemale: yes
Male: yes
9Phase 1United States;Turkey;Germany;Netherlands
2NCT03811028
(ClinicalTrials.gov)
January 19, 201917/12/2018A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA PatientsAn Open, Single-arm, Multicenter Extension Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA PatientsSanfilippo Syndrome Type A (MPS IIIA)Drug: SOBI003Swedish Orphan BiovitrumNULLCompleted18 Months78 MonthsAll6Phase 1/Phase 2United States;Turkey
3NCT03423186
(ClinicalTrials.gov)
June 19, 201829/1/2018A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA PatientsAn Open, Non-controlled, Parallel, Ascending Multiple-dose, Multicenter Study to Assess Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of SOBI003 in Pediatric MPS IIIA PatientsSanfilippo Syndrome Type A (MPS IIIA)Drug: SOBI003Swedish Orphan BiovitrumNULLCompleted12 Months72 MonthsAll6Phase 1/Phase 2United States;Germany;Turkey
4EUCTR2017-002806-10-DE
(EUCTR)
01/10/2018An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patientsAn open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome
MedDRA version: 20.1;Level: LLT;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Chemically modified recombinant human sulfamidase
Product Code: SOBI003
Other descriptive name: CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE
Swedish Orphan Biovitrum AB (publ)NULLNot RecruitingFemale: yes
Male: yes
9Phase 1United States;Turkey;Netherlands;Germany