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Clinical trials for Sanfilippo Syndrome (MPS III)

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    The EU Clinical Trials Register currently displays   42585   clinical trials with a EudraCT protocol, of which   7011   are clinical trials conducted with subjects less than 18 years old.
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    20 result(s) found for: Sanfilippo Syndrome (MPS III). Displaying page 1 of 1.
    EudraCT Number: 2010-021348-16 Sponsor Protocol Number: HGT-SAN-067 Start Date*: 2011-02-09
    Sponsor Name:Shire Human Genetic Therapies, Inc
    Full Title: An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients with Sanfilippo Syndrome Type A (MPS IIIA)
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) NL (Completed)
    Trial results: View results
    EudraCT Number: 2015-001983-20 Sponsor Protocol Number: NGLU-CL01-T Start Date*: 2015-08-06
    Sponsor Name:Alexion Pharmaceuticals, Inc
    Full Title: A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Int...
    Medical condition: Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-000753-20 Sponsor Protocol Number: NGLU-CL06 Start Date*: 2016-08-16
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A PHASE I/II OPEN LABEL STUDY IN MPS IIIB SUBJECTS TO INVESTIGATE THE SAFETY, BIODISTRIBUTION, PHARMACOKINETICS, AND PHARMACODYNAMICS/EFFICACY OF SBC-103 ADMINISTERED INTRAVENOUSLY
    Medical condition: Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2014-003960-20 Sponsor Protocol Number: SHP-610-201 Start Date*: 2015-02-18
    Sponsor Name:Shire human Genetic Therapies, Inc.
    Full Title: A Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in...
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    17.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) DE (Prematurely Ended) NL (Completed) GB (Prematurely Ended) FR (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-003450-24 Sponsor Protocol Number: HGT-SAN-093 Start Date*: 2014-04-09
    Sponsor Name:Shire Human Genetic Therapies, Inc
    Full Title: A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in ...
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    17.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) ES (Completed) NL (Completed) IT (Completed) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2009-015984-15 Sponsor Protocol Number: HGT-SAN-055 Start Date*: 2010-03-25
    Sponsor Name:Shire Human Genetic Therapies, Inc. (Shire HGT)
    Full Title: A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan-N-sulfatase (rhHNS) Intrathecal Administration via an Intrathecal Drug Delivery Device in Pat...
    Medical condition: Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    14.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-001479-18 Sponsor Protocol Number: GENiSIS2013 Start Date*: 2014-06-09
    Sponsor Name:Manchester University NHS Foundation Trust
    Full Title: A Phase III, Double Blinded, Randomised, Placebo Controlled Clinical Trial of High Dose Oral Genistein Aglycone in Patients with Sanfilippo Syndrome (Mucopolysaccharidosis III)
    Medical condition: Sanfilippo syndrome (Mucopolysaccharidosis III)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-003083-13 Sponsor Protocol Number: 250-202 Start Date*: Information not available in EudraCT
    Sponsor Name:Allievex Corporation
    Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo ...
    Medical condition: Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    20.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Ongoing) ES (Prematurely Ended) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2013-003400-39 Sponsor Protocol Number: NGLU-CL02 Start Date*: 2014-07-15
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
    Medical condition: Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) ES (Temporarily Halted)
    Trial results: View results
    EudraCT Number: 2010-019962-10 Sponsor Protocol Number: P1-SAF301 Start Date*: Information not available in EudraCT
    Sponsor Name:SANFILIPPO Therapeutics SAS
    Full Title: 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 TREAT...
    Medical condition: Sanfilippo type A syndrome (also named Mucopolysaccharidosis Type A)
    Disease: Version SOC Term Classification Code Term Level
    12.1 10056918 Sanfilippo's syndrome LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-000195-15 Sponsor Protocol Number: P4-SAF-302 Start Date*: 2018-12-27
    Sponsor Name:Lysogene SA
    Full Title: An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA for the...
    Medical condition: Mucopolysaccharidosis Type IIIA
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing) GB (GB - no longer in EU/EEA) NL (Temporarily Halted) DE (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2012-000856-33 Sponsor Protocol Number: 2012-40 Start Date*: Information not available in EudraCT
    Sponsor Name:Institut Pasteur
    Full Title: A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase cDNA in children with Sanfilippo type B syndrome
    Medical condition: Mucopolysaccharidosis III B
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2006-004661-34 Sponsor Protocol Number: 2006.426/19 Start Date*: 2006-12-08
    Sponsor Name:HOSPICES CIVILS DE LYON
    Full Title: Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo). Essai thérapeutique de phase IIb randomisé en aveugle con...
    Medical condition: Mucopolysaccharidose de type III
    Disease: Version SOC Term Classification Code Term Level
    8.1 10056890 Mucopolysaccharidosis III LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-001985-25 Sponsor Protocol Number: 250-201 Start Date*: 2015-11-06
    Sponsor Name:Allievex Corporation
    Full Title: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB ...
    Medical condition: Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-002936-97 Sponsor Protocol Number: LTFU-ABO-101 Start Date*: Information not available in EudraCT
    Sponsor Name:Abeona Therapeutics Europe SL.
    Full Title: A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU)
    Medical condition: MPS IIIB is a devastating lysosomal storage disease, caused by a N-α-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB appear normal at birth, but the disease is relentlessly progres...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-001411-39 Sponsor Protocol Number: ABT-002 Start Date*: 2018-09-04
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: Phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB
    Medical condition: MPS IIIB is a devastating lysosomal storage disease, caused by a N-α-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB appear normal at birth, but the disease is relentlessly progre...
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) DE (Prematurely Ended) FR (Prematurely Ended) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-002051-42 Sponsor Protocol Number: R119861 Start Date*: 2019-10-25
    Sponsor Name:University of Manchester
    Full Title: A phase I-II, study of autologous CD34+ haematopoietic stem cells transduced ex vivo with CD11b lentiviral vector encoding for human SGSH in patients with mucopolysaccharidosis type IIIA (MPS IIIA,...
    Medical condition: MPS IIIA, mucopolysaccharidosis type IIIA
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10028093 Mucopolysaccharidosis PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-002806-10 Sponsor Protocol Number: SOBI003-001 Start Date*: Information not available in EudraCT
    Sponsor Name:Swedish Orphan Biovitrum AB (publ)
    Full Title: An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients
    Medical condition: Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10028094 Mucopolysaccharidosis IH LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) NL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2009-017026-39 Sponsor Protocol Number: UCBT-002 Start Date*: 2009-12-28
    Sponsor Name:Aldagen, Inc
    Full Title: A PHASE III TRIAL OF ALD-101 ADUVANT THERAPY of UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN PATIENTS WITH INBORN ERRORS OF METABOLISM
    Medical condition: Inborn errors of metabolism
    Disease: Version SOC Term Classification Code Term Level
    9.1 10062018 Inborn error of metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-011921-15 Sponsor Protocol Number: UCBT-002 Start Date*: 2009-06-22
    Sponsor Name:Aldagen, Inc
    Full Title: A PHASE III TRIAL OF ALD-101 ADUVANT THERAPY of UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN PATIENTS WITH INBORN ERRORS OF METABOLISM
    Medical condition: Inborn errors of metabolism
    Disease: Version SOC Term Classification Code Term Level
    9.1 10062018 Inborn error of metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Not Authorised)
    Trial results: (No results available)
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