19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | NCT02053064 (ClinicalTrials.gov) | May 2013 | 16/1/2014 | Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy | Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301. | Mucopolysaccharidosis Type III A;Sanfilippo Disease Type A | Genetic: SAF-301 | LYSOGENE | NULL | Completed | N/A | N/A | All | 4 | Phase 1/Phase 2 | France |
| 2 | NCT01474343 (ClinicalTrials.gov) | August 2011 | 10/11/2011 | Intracerebral Gene Therapy for Sanfilippo Type A Syndrome | An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome. | Mucopolysaccharidosis Type III A;Sanfilippo Disease Type A | Genetic: SAF-301 | LYSOGENE | NULL | Completed | 18 Months | 6 Years | Both | 4 | Phase 1/Phase 2 | France |
| 3 | EUCTR2010-019962-10-FR (EUCTR) | 19/11/2010 | AN OPEN-LABEL, SINGLE ARM, MONOCENTRIC, PHASE I/II CLINICAL STUDY OF INTRACEREBRAL ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS SEROTYPE 10 CARRYING THE HUMAN SGSH AND SUMF1 cDNAS FOR THE TREATMENT OF SANFILIPPO TYPE A SYNDROME - Intracerebral Gene therapy for Sanfilippo type A syndrome | AN OPEN-LABEL, SINGLE ARM, MONOCENTRIC, PHASE I/II CLINICAL STUDY OF INTRACEREBRAL ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS SEROTYPE 10 CARRYING THE HUMAN SGSH AND SUMF1 cDNAS FOR THE TREATMENT OF SANFILIPPO TYPE A SYNDROME - Intracerebral Gene therapy for Sanfilippo type A syndrome | Sanfilippo type A syndrome (also named Mucopolysaccharidosis Type A) MedDRA version: 12.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome | Product Name: AAVrh.10-hMPSIIIA Product Code: SAF-301 | SANFILIPPO Therapeutics SAS | NULL | Not Recruiting | Female: yes Male: yes | 4 | Phase 1/2 | France |