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Clinical trials for leukodystrophy

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   44156   clinical trials with a EudraCT protocol, of which   7326   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    22 result(s) found for: leukodystrophy. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2020-005229-95 Sponsor Protocol Number: PBKR03-001 Start Date*: 2021-08-23
    Sponsor Name:Passage Bio, Inc.
    Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Dis...
    Medical condition: Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LSD) caused by mutations in the gene encoding the hydrolytic enzyme galactosylceramidase (galactoce...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10023492 Krabbe's disease PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2007-006345-40 Sponsor Protocol Number: rhASA-03 Start Date*: 2008-08-26
    Sponsor Name:Shire Pharmaceutical Ireland Limited
    Full Title: A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and long-term safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of pati...
    Medical condition: Metachromatic Leukodystrophy (MLD) in late infantile patients
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 Leukodystrophy LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2008-000084-41 Sponsor Protocol Number: HGT-MLD-049 Start Date*: 2008-05-15
    Sponsor Name:Shire Human Genetic Therapies Inc
    Full Title: A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD)
    Medical condition: Metachromatic Leukodystrophy (MLD) in late infantile patients
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 Leukodystrophy LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) FR (Prematurely Ended) IT (Prematurely Ended) BE (Completed)
    Trial results: View results
    EudraCT Number: 2011-002044-28 Sponsor Protocol Number: HGT-MLD-070 Start Date*: 2012-06-14
    Sponsor Name:Shire Human Genetics Therapies Inc
    Full Title: A Phase I/II, Multicenter, Open-label, Dose Escalation Study of HGT-1110 Administered Intrathecally in Children with Metachromatic Leukodystrophy
    Medical condition: Treatment of Metachromatic Leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2007-007165-20 Sponsor Protocol Number: rhASA-04 Start Date*: 2008-02-26
    Sponsor Name:Shire Pharmaceuticals Ireland Limited
    Full Title: A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients w...
    Medical condition: Metachromatic Leukodystrophy (MLD) in late infantile patients
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 Leukodystrophy LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2017-001730-26 Sponsor Protocol Number: 205756 Start Date*: 2017-11-30
    Sponsor Name:Orchard Therapeutics (Europe) Ltd
    Full Title: A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA OTL-200, for the treatment of early onset Metachromatic...
    Medical condition: Metachromatic Leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-003775-20 Sponsor Protocol Number: HGT-MLD-071 Start Date*: 2013-09-11
    Sponsor Name:Shire Human Genetics Therapies Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
    Full Title: An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
    Medical condition: Treatment of Metachromatic Leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Ongoing) DK (Trial now transitioned) FR (Ongoing) CZ (Ongoing) GB (GB - no longer in EU/EEA) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-003291-12 Sponsor Protocol Number: SHP611-201 Start Date*: 2019-04-11
    Sponsor Name:Shire Human Genetic Therapies, Inc.
    Full Title: A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
    Medical condition: Late Metachromatic Leukodystrophy (MLD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) NL (Ongoing) ES (Ongoing) BE (Trial now transitioned) DE (Trial now transitioned) GB (GB - no longer in EU/EEA) IT (Ongoing) GR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-005341-11 Sponsor Protocol Number: rhASA-01 Start Date*: 2006-12-28
    Sponsor Name:Shire Pharmaceuticals Ireland Limited
    Full Title: A single center, open-label, non-randomized, uncontrolled, multiple-dose, dose escalation study of the safety, pharmacokinetics and efficacy of Metazym (recombinant human arylsulfatase A or rhASA) ...
    Medical condition: Late infantile metachromatic leukodystrophy (MLD)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 Leukodystrophy LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2009-017349-77 Sponsor Protocol Number: TIGET-MLD Start Date*: 2010-03-15
    Sponsor Name:FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
    Full Title: A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy
    Medical condition: metachromatic leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10024381 PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-004410-42 Sponsor Protocol Number: C11-09 Start Date*: 2012-11-26
    Sponsor Name:Inserm
    Full Title: A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA c...
    Medical condition: Early onset forms of MLD
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    14.1 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    14.1 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-002636-82 Sponsor Protocol Number: OTL-200-07 Start Date*: 2019-12-02
    Sponsor Name:Orchard Therapeutics (Europe) Limited
    Full Title: An open label, non-randomised trial to evaluate the safety and efficacy of a single infusion of OTL-200 in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD)
    Medical condition: Metachromatic Leukodystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-000976-40 Sponsor Protocol Number: ION373-CS1 Start Date*: Information not available in EudraCT
    Sponsor Name:Ionis Pharmaceuticals, Inc
    Full Title: A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexand...
    Medical condition: Alexander Disease
    Disease: Version SOC Term Classification Code Term Level
    22.1 10010331 - Congenital, familial and genetic disorders 10083059 Alexander disease PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Trial now transitioned) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-002805-13 Sponsor Protocol Number: LTF-304 Start Date*: 2015-11-27
    Sponsor Name:bluebird bio, Inc
    Full Title: Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned) DE (Trial now transitioned) NL (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-001438-25 Sponsor Protocol Number: VWM1 Start Date*: 2020-04-21
    Sponsor Name:VU University Medical Center
    Full Title: A Study to Explore the Safety, Tolerability, Pharmacokinetic Profile, and Potential Efficacy of Guanabenz in Patients With Early Childhood Onset Vanishing White Matter (VWM)
    Medical condition: Vanishing white matter
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-001953-10 Sponsor Protocol Number: ALD-102 Start Date*: 2013-12-13
    Sponsor Name:bluebird bio, Inc.
    Full Title: A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD)
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) DE (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-024084-40 Sponsor Protocol Number: XAMNANTIOXAP2010 Start Date*: 2011-06-30
    Sponsor Name:Aurora Pujol Onofre
    Full Title: Ensayo clínico en adrenomieloneuropatía (AMN): validación de biomarcadores de estrés oxidativo, eficacia y tolerancia de la combinación de antioxidantes N-acetilcisteína, ácido lipoico y vitamina E
    Medical condition: Adrenoleucodistrofia ligada al X (XALD), fenotipo Adrenomieloneuropatía (AMN).
    Disease: Version SOC Term Classification Code Term Level
    13 10051260 Adrenoleucodistrofia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-001145-14 Sponsor Protocol Number: ALD-104 Start Date*: 2019-04-17
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Ongoing) DE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    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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