DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	1

告示番号

疾患名（ページ内リンク）

臨床試験数

ライソゾーム病

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	EUCTR2017-002430-23-IT (EUCTR)	14/03/2018	09/03/2020	Gene therapy study with autologous hemapoietic stem cells for patients affected by MPSIH	A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human a-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant - TigetT10_MPS1H	Mucopolysaccharidosis type I Hurler MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Other descriptive name: Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI INN or Proposed INN: BUSULFANO Other descriptive name: BUSULFANO Trade Name: FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML INN or Proposed INN: FLUDARABINA FOSFATO Other descriptive name: FLUDARABINA FOSFATO Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML INN or Proposed INN: RITUXIMAB Other descriptive name: RITUXIMAB Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML INN or Proposed INN: LENOGRASTIM Other descriptive name: LENOGRASTIM Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F	OSPEDALE SAN RAFFAELE	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	6	Phase 1	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

EUCTR2017-002430-23-IT
(EUCTR)

14/03/2018

09/03/2020

Gene therapy study with autologous hemapoietic stem cells for patients affected by MPSIH

A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human a-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant - TigetT10_MPS1H

Mucopolysaccharidosis type I Hurler
MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Other descriptive name: Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi
Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI
INN or Proposed INN: BUSULFANO
Other descriptive name: BUSULFANO
Trade Name: FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML
INN or Proposed INN: FLUDARABINA FOSFATO
Other descriptive name: FLUDARABINA FOSFATO
Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML
INN or Proposed INN: RITUXIMAB
Other descriptive name: RITUXIMAB
Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML
INN or Proposed INN: LENOGRASTIM
Other descriptive name: LENOGRASTIM
Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F

OSPEDALE SAN RAFFAELE

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1

Italy