19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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"
31 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2010-020199-45-ES
(EUCTR)
23/07/201309/04/2013A Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)Extensión de un estudio multicéntrico, multinacional para evaluar la eficacia y seguridad a largo plazo de BMN 110 en pacientes con mucopolisacaridosis IVA (síndrome de Morquio A) Mucopolysaccharidosis Type IVA
MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
Product Code: BMN 110
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;France;Canada;Argentina;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of
2NCT01966029
(ClinicalTrials.gov)
July 201310/9/2013BMN 110 Phase 3B in Australian PatientsA Multicenter Open-Label, Phase 3B Study to Evaluate the Efficacy and Safety of BMN 110 in Australian Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Mucopolysaccharidosis IVA (Morquio A Syndrome)Drug: BMN 110BioMarin PharmaceuticalNULLCompletedN/AN/AAll13Phase 3Australia
3NCT01697319
(ClinicalTrials.gov)
August 201217/9/2012Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited AmbulationA Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited AmbulationMucopolysaccharidosis IVA;Morquio A Syndrome;MPS IVADrug: BMN 110BioMarin PharmaceuticalNULLTerminated5 YearsN/AAll16Phase 2United States;Germany;United Kingdom
4EUCTR2010-020199-45-DK
(EUCTR)
04/07/201224/05/2012A Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
Product Code: BMN 110
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;France;Canada;Argentina;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of
5EUCTR2011-005703-33-DE
(EUCTR)
19/06/201231/01/2012A Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited AmbulationA Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation Mucopolysaccharidosis Type IVA
MedDRA version: 16.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
Product Code: BMN 110
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2United States;Germany;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2011-005682-20-DE
(EUCTR)
19/06/201231/01/2012A Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA PatientsA Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
25United States;Canada;Germany;United Kingdom
7EUCTR2010-020199-45-DE
(EUCTR)
14/06/201212/09/2011A phase 3 extension study to evaluate the long term efficacy and safety of BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio A syndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - NA Mucopolysaccharidosis type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: recombinant human Nacetylgalactosamine-
Product Code: BMN 110
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Turkey;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Brazil;Poland;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of
8EUCTR2011-005703-33-GB
(EUCTR)
29/05/201201/03/2012A Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited AmbulationA Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation Mucopolysaccharidosis Type IVA
MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
Product Code: BMN 110
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2United States;Germany;Italy;United Kingdom
9NCT01609062
(ClinicalTrials.gov)
April 201224/5/2012Safety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeA Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Mucopolysaccharidosis IVA;Morquio A Syndrome;MPS IVADrug: BMN 110BioMarin PharmaceuticalNULLTerminated7 YearsN/AAll25Phase 2United States;Canada;Germany;United Kingdom
10EUCTR2011-005682-20-GB
(EUCTR)
19/03/201221/03/2012A Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA PatientsA Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
25United States;Canada;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2011-003197-84-IT
(EUCTR)
13/03/201212/03/2012A Study to Evaluate the Safety and Efficacy of BMN 110 in MPS IVA Patients Less Than 5 Years of AgeA Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: NA
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BIOMARIN PHARMACEUTICAL INC.NULLNot RecruitingFemale: yes
Male: yes
15Phase 2United States;United Kingdom;Italy
12EUCTR2010-020199-45-PT
(EUCTR)
02/03/201229/12/2011A phase 3 extension study to evaluate the long term efficacy and safety of BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio A syndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - NA Mucopolysaccharidosis type IVA
MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: recombinant human Nacetylgalactosamine-
Product Code: BMN 110
INN or Proposed INN: Not Available
Other descriptive name: recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
162Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Turkey;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Brazil;Poland;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of
13EUCTR2010-020198-18-NO
(EUCTR)
03/01/201219/09/2011A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVAA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
162Phase 3Portugal;Germany;Colombia;Switzerland;Netherlands;France;Norway;Italy;Australia;Brazil;United Kingdom;Canada;Saudi Arabia;Denmark;Taiwan;Argentina;Korea, Republic of;Qatar;Japan;United States;Poland
14EUCTR2010-020199-45-IT
(EUCTR)
20/12/201127/12/2011A phase 3 extension study to evaluate the long term efficacy and safetyof BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio Asyndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with MucopolysaccharidosisIVA (Morquio A Syndrome) Mucopolysaccharidosis type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BM110
Other descriptive name: recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
BIOMARIN PHARMACEUTICAL INC.NULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Korea, Democratic People's Republic of;Turkey;Colombia;United Kingdom;Italy;Switzerland;Canada;Argentina;Brazil;Denmark;Australia;Germany;Japan
15EUCTR2010-020198-18-DK
(EUCTR)
01/12/201117/10/2011A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVAA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;United States;Qatar;Saudi Arabia;Taiwan;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Poland;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2011-003197-84-GB
(EUCTR)
22/11/201113/09/2011A Study to Evaluate the Safety and Efficacy of BMN 110 in MPS IVA Patients Less Than 5 Years of AgeA Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
15Phase 2United States;Italy;United Kingdom
17NCT01515956
(ClinicalTrials.gov)
October 201122/12/2011Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)Mucopolysaccharidosis IVA;Morquio A Syndrome;MPS IVADrug: BMN 110BioMarin PharmaceuticalNULLCompletedN/A5 YearsAll15Phase 2United States;Italy;Taiwan;United Kingdom
18EUCTR2010-020198-18-DE
(EUCTR)
12/08/201114/02/2011A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVAA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
162Phase 3United States;Portugal;Qatar;Taiwan;Saudi Arabia;Colombia;United Kingdom;Switzerland;Italy;France;Canada;Argentina;Poland;Brazil;Australia;Denmark;Germany;Netherlands;Norway;Japan;Korea, Republic of
19EUCTR2010-020198-18-IT
(EUCTR)
19/07/201114/06/2011A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - NDA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - ND Mucopolysaccharidosis Type IVA
MedDRA version: 13.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Name: BMN 110
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BIOMARIN PHARMACEUTICAL INC.NULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;Germany;United Kingdom;Denmark;Norway;Italy
20EUCTR2010-020198-18-PT
(EUCTR)
01/07/201119/04/2011A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVAA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
162Phase 3United Kingdom;France;Canada;Argentina;Poland;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of;Portugal;United States;Qatar;Saudi Arabia;Taiwan;Colombia;Italy;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT01415427
(ClinicalTrials.gov)
July 20118/8/2011Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)Mucopolysaccharidosis IV A;Morquio A Syndrome;MPS IVADrug: BMN 110 - Weekly;Drug: BMN 110 - Every Other WeekBioMarin PharmaceuticalNULLCompleted5 YearsN/AAll173Phase 3United States;Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom
22EUCTR2010-020199-45-GB
(EUCTR)
08/06/201106/05/2011A Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
162Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;Switzerland;France;Canada;Argentina;Poland;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of
23NCT01275066
(ClinicalTrials.gov)
February 201110/1/2011A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)MPS IV ADrug: BMN 110 Weekly;Drug: Placebo;Drug: BMN 110 Every Other WeekBioMarin PharmaceuticalNULLCompleted5 YearsN/AAll177Phase 3United States;Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Qatar;Saudi Arabia;Taiwan;United Kingdom;Australia;Norway;Poland;Switzerland;Turkey
24EUCTR2010-020198-18-GB
(EUCTR)
30/12/201015/11/2010A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVAA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 14.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
162Phase 3United States;Portugal;Qatar;Taiwan;Saudi Arabia;Spain;Turkey;Colombia;Switzerland;United Kingdom;Italy;France;Canada;Argentina;Poland;Brazil;Australia;Denmark;Norway;Germany;Netherlands;Japan;Korea, Republic of
25NCT01242111
(ClinicalTrials.gov)
November 201028/10/2010A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)MPS IV A;Mucopolysaccharidosis IVA;Morquio A SyndromeDrug: BMN 110BioMarin PharmaceuticalNULLTerminatedN/AN/AAll20Phase 1;Phase 2United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2010-021048-16-GB
(EUCTR)
07/10/201017/06/2010A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 16.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
20United Kingdom
27NCT00884949
(ClinicalTrials.gov)
April 200910/4/2009A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVAA Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)MPS IV ADrug: BMN 110BioMarin PharmaceuticalNULLCompleted5 Years18 YearsAll20Phase 1;Phase 2United Kingdom
28EUCTR2008-007365-23-GB
(EUCTR)
12/03/200914/04/2009A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome)A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome) Mucopolysaccharidosis Type IV A
MedDRA version: 9.1;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV
Product Name: BMN 110
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
Phase 1;Phase 2United Kingdom
29NCT01858103
(ClinicalTrials.gov)
January 200915/5/2013BMN 110 US Expanded Access ProgramA Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVAMucopolysaccharidosis IVA;Morquio A Syndrome;MPS IVADrug: BMN 110BioMarin PharmaceuticalNULLApproved for marketingN/AN/ABothN/AUnited States;Puerto Rico
30EUCTR2010-020198-18-NL
(EUCTR)
31/01/2011A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 12.1;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV
MedDRA version: 12.1;Level: PT;Term: Mucopolysaccharidosis IV
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
162Phase 3Portugal;France;Denmark;Norway;Germany;Netherlands;Italy;United Kingdom
No.TrialIDDate_
enrollment
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registration
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sponsor
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sponsor
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agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
31EUCTR2010-020198-18-FR
(EUCTR)
01/02/2011A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Mucopolysaccharidosis Type IVA
MedDRA version: 12.1;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV
MedDRA version: 12.1;Level: PT;Term: Mucopolysaccharidosis IV
Product Name: BMN 110
Product Code: N/A
INN or Proposed INN: N/A
Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical IncNULLNAFemale: yes
Male: yes
162Phase 3Portugal;France;Denmark;Norway;Germany;Netherlands;Italy;United Kingdom