DDrare: Database of Drug Development for Rare Diseases

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
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.

Drug = 1-deoxynojirimycin; 1-deoxynojirimycin hydrochloride; A-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA); AAV2/8LSPhGAA; ALGLUCOSIDASE ALFA; ALGLUCOSIDASE ALFA (MYOZYME); AT2220; AT2221; AT2221 65 mg Formulated Capsules; AT845; ATB200; Acetaminophen; Albuterol; Aldurazyme (laronidase); Alglucosidase Alfa; Alglucosidase Alfa (Genetical Recombination); Alglucosidase alfa; Alglucosidase alfa (GZ419829); Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]); Alglucosidase alfa GZ419829; Alglucosidase alpha; Alpha-glucosidase; Avalglucosidase Alfa; Avalglucosidase Alfa (NeoGAA); Avalglucosidase alfa; Avalglucosidase alfa GZ402666; Avalglucosidase alfa(GZ402666); BMN 701; Beta-hydroxybuturate esters; Bortezomib; CYCLOPHOSPHAMIDE MONOHYDRATE; Cipaglucosidase alfa; Clenbuterol; Cyclophosphamide; Deoxynojirimycin; Deprakine; Diphenhydramine; Duvoglustat; Duvoglustat HCl; Duvoglustat hydrochloride; Enzyme Replacement Agent; Epilim; Epilim Chrono 200 Controlled Released tablets; Epilim Chrono 300 Controlled Released tablets; Epilim Chrono 500 Controlled Released tablets; Filgrastim; GILT-rhGAA; GZ402666; Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase; Glucosidase alfa; Glycosade; Intravenous immune globulin; Ketocal 4:1 liquid Nutricia (intervention); LMX 4 Topical Cream; Laronidase; Lidocaine; Low carbohydrate ketogenic diet; Lumizyme; Lumizyme®; METHOTREXATE SODIUM; MIGLUSTAT; MYOZYME®; Mannose; Methotrexate; Miglustat; Miglustat (AT2221); Myozyme; Myozyme 50 mg powder for concentrate for solution for infusion; Myozyme®; Myozyme® (alglucosidase alfa); N.a.; NEO-GAA; NeoGAA; NeoGAA or avalglucosidase alfa; Other: No Treatment; Other: RMST; Other: saline; Phosphate; Plecebo; Pyridostigmine; Pyridostigmine Bromide; RAAV1-CMV-GAA (study agent) Administration; RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN; REN001; RITUXIMAB; Rapamycin; ReN001; Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA); Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA); Recombinant human acid alfa-glucosidase; Recombinant human acid alfa-glucosidase (ATB200); Recombinant human acid alfa-glucosidase (rhGAA); Recombinant human acid alpha-glucosidase (rhGAA); Reveglucosidase alfa; Reveglucosidase alpha; RhGAA; RhGAA, acid alpha glucosidase; Rituxan; Rituximab; SODIUM VALPROATE; SPK-3006; Salbutamol; Sodium Valproate; Sodium valproate; Ss-hydroxybuturate esters; TRIHEPTANOIN; Triheptanoin; UX007; VAL-1221; Valproate; Valproic acid; ZAVESCA; Zavesca; Zavesca® Prescription

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2013-001768-48-AT (EUCTR)	21/02/2016	12/01/2016	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	70	Phase 3	Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland
2	EUCTR2013-001768-48-PT (EUCTR)	30/01/2015	02/07/2014	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
3	EUCTR2013-001768-48-DE (EUCTR)	23/09/2014	22/05/2014	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom
4	EUCTR2013-001768-48-IT (EUCTR)	22/09/2014	01/07/2014	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	Pompe disease MedDRA version: 17.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;United Kingdom;Italy
5	EUCTR2013-001768-48-BE (EUCTR)	08/09/2014	12/05/2014	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
6	NCT01924845 (ClinicalTrials.gov)	April 2014	13/8/2013	BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease	Late-onset Pompe Disease	Drug: BMN 701	BioMarin Pharmaceutical	NULL	Terminated	18 Years	N/A	All	24	Phase 3	United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom
7	EUCTR2013-001768-48-GB (EUCTR)	27/11/2013	22/10/2013	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease	Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alfa Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	70	Phase 3	Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
8	EUCTR2011-001805-28-DE (EUCTR)	14/02/2013	28/06/2012	A long term study of the safety of BMN 701 in patients with POMPE disease	A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study	POMPE disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	21	Phase 2	United States;Australia;Germany;New Zealand;United Kingdom
9	EUCTR2010-023561-22-DE (EUCTR)	04/07/2012	07/02/2012	A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease	A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease	Pompe disease MedDRA version: 16.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 1;Phase 2	France;United States;Netherlands;Germany;United Kingdom
10	EUCTR2011-001805-28-GB (EUCTR)	03/07/2012	26/04/2012	A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study	A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study	Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 2	United States;France;Australia;Germany;United Kingdom
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
11	NCT01435772 (ClinicalTrials.gov)	August 15, 2011	8/9/2011	Extension Study for Patients Who Have Participated in a BMN 701 Study	A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study	Pompe Disease	Biological: BMN 701	BioMarin Pharmaceutical	NULL	Terminated	13 Years	N/A	All	21	Phase 2	United States;Australia;France;Germany;New Zealand;United Kingdom
12	EUCTR2010-023561-22-GB (EUCTR)	15/08/2011	11/04/2011	A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease	A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease	Pompe disease MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	28	Phase 1;Phase 2	United States;Germany;United Kingdom
13	NCT01230801 (ClinicalTrials.gov)	January 17, 2011	27/10/2010	Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease	A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease	Pompe Disease	Biological: BMN 701	BioMarin Pharmaceutical	NULL	Completed	13 Years	N/A	All	22	Phase 1;Phase 2	United States;Australia;France;Germany;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2013-001768-48-AT
(EUCTR)

21/02/2016

12/01/2016

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland

EUCTR2013-001768-48-PT
(EUCTR)

30/01/2015

02/07/2014

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom

EUCTR2013-001768-48-DE
(EUCTR)

23/09/2014

22/05/2014

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom

EUCTR2013-001768-48-IT
(EUCTR)

22/09/2014

01/07/2014

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Pompe disease
MedDRA version: 17.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;United Kingdom;Italy

EUCTR2013-001768-48-BE
(EUCTR)

08/09/2014

12/05/2014

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT01924845
(ClinicalTrials.gov)

April 2014

13/8/2013

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease

Late-onset Pompe Disease

Drug: BMN 701

BioMarin Pharmaceutical

NULL

Terminated

18 Years

N/A

All

Phase 3

United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom

EUCTR2013-001768-48-GB
(EUCTR)

27/11/2013

22/10/2013

A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alfa
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom

EUCTR2011-001805-28-DE
(EUCTR)

14/02/2013

28/06/2012

A long term study of the safety of BMN 701 in patients with POMPE disease

A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study

POMPE disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;Australia;Germany;New Zealand;United Kingdom

EUCTR2010-023561-22-DE
(EUCTR)

04/07/2012

07/02/2012

A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease

A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease

Pompe disease
MedDRA version: 16.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

France;United States;Netherlands;Germany;United Kingdom

EUCTR2011-001805-28-GB
(EUCTR)

03/07/2012

26/04/2012

A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study

Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;France;Australia;Germany;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT01435772
(ClinicalTrials.gov)

August 15, 2011

8/9/2011

Extension Study for Patients Who Have Participated in a BMN 701 Study

A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study

Pompe Disease

Biological: BMN 701

BioMarin Pharmaceutical

NULL

Terminated

13 Years

N/A

All

Phase 2

United States;Australia;France;Germany;New Zealand;United Kingdom

EUCTR2010-023561-22-GB
(EUCTR)

15/08/2011

11/04/2011

A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease

Pompe disease
MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA

BioMarin Pharmaceutical Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

United States;Germany;United Kingdom

NCT01230801
(ClinicalTrials.gov)

January 17, 2011

27/10/2010

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

Pompe Disease

Biological: BMN 701

BioMarin Pharmaceutical

NULL

Completed

13 Years

N/A

All

Phase 1;Phase 2

United States;Australia;France;Germany;United Kingdom