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

23 trials found

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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	NCT05634512 (ClinicalTrials.gov)	November 17, 2022	22/11/2022	Evaluation of Intravenous LaronidasePharmacokinetics Before and After Hematopoietic Cell Transplanta ...	Evaluation of Intravenous LaronidasePharmacokinetics Before and After Hematopoietic Cell Transplanta ...	Hematopoietic Cell Transplantation;Mucopolysaccharidosis Type I	Drug: Laronidase therapy and a stem cell transplant	Masonic Cancer Center, University of Minnesota	NULL	Enrolling by invitation	0 Years	3 Years	All	24		United States
2	NCT05134571 (ClinicalTrials.gov)	October 28, 2021	21/10/2021	China Post-marketing Surveillance (PMS) Study of Aldurazyme®	A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Re ...	Mucopolysaccharidosis I	Drug: Laronidase	Genzyme, a Sanofi Company	NULL	Active, not recruiting	5 Years	N/A	All	12	Phase 4	China
3	NCT04532047 (ClinicalTrials.gov)	July 1, 2021	19/8/2020	In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases	In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs ...	MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease In ...	Drug: Aldurazyme (laronidase)	University of California, San Francisco	Duke University	Recruiting	18 Years	50 Years	Female	10	Phase 1	United States
4	EUCTR2015-003031-35-GB (EUCTR)	20/11/2015	27/10/2015	Immune Tolerance Induction with Methotrexate in Hurler Syndrome	A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitig ...	Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH) MedDRA version: 18.1;Level: LLT;Classi ...	Product Name: Methotrexate INN or Proposed INN: Methotrexate	Central Manchester University Hospitals NHS Foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	4	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United Kingdom
5	NCT01173016 (ClinicalTrials.gov)	May 29, 2012	28/7/2010	Administration of IV Laronidase Post Bone Marrow Transplant in Hurler	Pilot Study of Administration of Intravenous LaronidaseFollowing Allogeneic Transplantation for Hurl ...	Hurler Syndrome	Drug: Laronidase	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	14 Years	All	11	Phase 1	United States
6	NCT01572636 (ClinicalTrials.gov)	March 28, 2012	4/4/2012	Laronidase(Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hur ...	MT2011-21C Laronidase(Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell T ...	Mucopolysaccharidosis Type IH;MPS I;Hurler Syndrome	Drug: Laronidase	Masonic Cancer Center, University of Minnesota	NULL	Terminated	N/A	N/A	All	20		United States
7	NCT00418821 (ClinicalTrials.gov)	October 22, 2010	3/1/2007	A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Muc ...	A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme (Laronidase) Treatment o ...	Mucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie	Biological: Aldurazyme (laronidase)	Genzyme, a Sanofi Company	BioMarin/Genzyme LLC	Recruiting	N/A	N/A	Female	2	Phase 4	Italy;United States
8	EUCTR2007-007003-33-IT (EUCTR)	16/09/2010	27/12/2010	A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme(laronidase) Treatment on ...	A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme(laronidase) Treatment on ...	mucocopolysaccharidoses I MedDRA version: 9.1;Level: LLT;Classification code 10056886	Trade Name: ALDURAZYME *INFUS 1FL 5ML 500U INN or Proposed INN: Laronidase	Genzyme Europe BV	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: no	10		Italy
9	NCT00852358 (ClinicalTrials.gov)	June 2009	25/2/2009	A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I	A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I	Cognitive Decline;Mucopolysaccharidosis I;Hurler-Scheie Syndrome;Scheie Syndrome;Lysosomal Storage D ...	Drug: laronidase	Patricia I. Dickson, M.D.	The Ryan Foundation;BioMarin Pharmaceutical;Rare Diseases Clinical Research Network;National Center ...	Completed	6 Years	N/A	Both	9	N/A	United States
10	NCT00741338 (ClinicalTrials.gov)	September 2008	13/8/2008	Immune Tolerance Study With Aldurazyme® (Laronidase)	A Trial of Antigen-specific Immune Tolerance Induction in Mucopolysaccharidosis I (MPS I) Patients I ...	Mucopolysaccharidosis I	Biological: Laronidase;Drug: Cyclosporine A (CsA);Drug: Azathioprine (Aza)	Genzyme, a Sanofi Company	BioMarin/Genzyme LLC	Completed	N/A	5 Years	All	7	Phase 1/Phase 2	Brazil;Russian Federation;Ukraine

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