DDrare: Database of Drug Development for Rare Diseases

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	2

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	NCT02678689 (ClinicalTrials.gov)	February 2016	15/1/2016	A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease	A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease	Jansky-Bielschowsky Disease;Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;CLN2 Disease;CLN2 Disorder	Biological: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1);Device: Intraventricular access device	BioMarin Pharmaceutical	NULL	Active, not recruiting	N/A	17 Years	All	14	Phase 2	United States;Germany;Italy;United Kingdom
2	EUCTR2014-003480-37-IT (EUCTR)	06/05/2015	07/01/2015	An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease	A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease	Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2). MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)	BioMarin Pharmaceutical Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	22		United States;Germany;United Kingdom;Japan;Italy

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

NCT02678689
(ClinicalTrials.gov)

February 2016

15/1/2016

A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Jansky-Bielschowsky Disease;Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;CLN2 Disease;CLN2 Disorder

Biological: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1);Device: Intraventricular access device

BioMarin Pharmaceutical

NULL

Active, not recruiting

N/A

17 Years

All

Phase 2

United States;Germany;Italy;United Kingdom

EUCTR2014-003480-37-IT
(EUCTR)

06/05/2015

07/01/2015

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease

A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease

Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2).
MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)

BioMarin Pharmaceutical Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

United States;Germany;United Kingdom;Japan;Italy