- Trials with a EudraCT protocol (22)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
22 result(s) found for: Inborn Error of Metabolism.
Displaying page 1 of 2.
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 | |||||||||||||
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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 | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Not Authorised) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2017-002155-28 | Sponsor Protocol Number: P160902J | Start Date*: 2018-08-30 | |||||||||||
Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | |||||||||||||
Full Title: Evaluation of allopurinol treatment for autistic disorders and epilepsy in adenylosuccinate lyase deficiency (ADSL) | |||||||||||||
Medical condition: patient with adenulosuccinate lyase deficiency | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2012-005021-76 | Sponsor Protocol Number: OPBGC&RS_12_003 | Start Date*: 2013-01-31 | |||||||||||
Sponsor Name:EDISON PHARMACEUTICALS INC. | |||||||||||||
Full Title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome | |||||||||||||
Medical condition: Rett Syndrome | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2011-002880-42 | Sponsor Protocol Number: LAL-CL05 | Start Date*: 2011-12-05 | |||||||||||||||||||||
Sponsor Name:Synageva Biopharma Corp. | |||||||||||||||||||||||
Full Title: An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102 | |||||||||||||||||||||||
Medical condition: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). | |||||||||||||||||||||||
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Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: GB (Prematurely Ended) FR (Ongoing) | |||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2011-001513-13 | Sponsor Protocol Number: LAL-CL04 | Start Date*: 2011-10-24 | |||||||||||||||||||||
Sponsor Name:Synageva Biopharma Corp. | |||||||||||||||||||||||
Full Title: AN OPEN LABEL MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF SBC-102 IN ADULT SUBJECTS WITH LIVER DYSFUNCTION DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY WH... | |||||||||||||||||||||||
Medical condition: Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare di... | |||||||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: GB (Completed) CZ (Completed) | |||||||||||||||||||||||
Trial results: View results |
EudraCT Number: 2013-001045-14 | Sponsor Protocol Number: SAF001 | Start Date*: 2013-04-22 | |||||||||||
Sponsor Name:Promethera Biosciences | |||||||||||||
Full Title: SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem. | |||||||||||||
Medical condition: Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The ur... | |||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: BE (Completed) PT (Completed) IT (Completed) GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-000032-28 | Sponsor Protocol Number: LAL-CL03 | Start Date*: 2011-04-18 | |||||||||||||||||||||
Sponsor Name:Synageva Biopharma Corp. | |||||||||||||||||||||||
Full Title: An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal ... | |||||||||||||||||||||||
Medical condition: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease). | |||||||||||||||||||||||
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Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: GB (Completed) FR (Completed) DE (Prematurely Ended) IT (Completed) IE (Completed) | |||||||||||||||||||||||
Trial results: View results |
EudraCT Number: 2011-004074-28 | Sponsor Protocol Number: UKHEP001 | Start Date*: 2012-07-20 | |||||||||||
Sponsor Name:Promethera Biosciences | |||||||||||||
Full Title: A prospective, open label, multicenter, randomized, safety and preliminary efficacy study of one cycle of Promethera HepaStem® in Urea Cycle Disorders and Crigler-Najjar Syndrome patients | |||||||||||||
Medical condition: Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The ur... | |||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) BE (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-003989-27 | Sponsor Protocol Number: PROLONGSTEM | Start Date*: Information not available in EudraCT | ||||||||||||||||||||||||||
Sponsor Name:Promethera Therapeutics | ||||||||||||||||||||||||||||
Full Title: PROLONGSTEM: HepaStem Long-Term Safety Registry Registry for patients who have been administered HepaStem | ||||||||||||||||||||||||||||
Medical condition: This study will include all patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HALPC during a previous interventional clinical study conducted by... | ||||||||||||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |||||||||||||||||||||||||||
Trial protocol: BE (Trial now transitioned) FR (Ongoing) ES (Ongoing) GB (GB - no longer in EU/EEA) BG (Ongoing) PL (Ongoing) | ||||||||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2010-024068-16 | Sponsor Protocol Number: LAL-CL01 | Start Date*: 2011-03-08 | |||||||||||||||||||||
Sponsor Name:Synageva Biopharma Corp. | |||||||||||||||||||||||
Full Title: An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency. | |||||||||||||||||||||||
Medical condition: Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare di... | |||||||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: GB (Completed) CZ (Completed) | |||||||||||||||||||||||
Trial results: View results |
EudraCT Number: 2011-004259-38 | Sponsor Protocol Number: CAC-91-10-10 | Start Date*: 2011-10-06 | |||||||||||
Sponsor Name:Asklepion Pharmaceuticals, LLC | |||||||||||||
Full Title: Investigation in the Pathogenesis of Liver Disease in Patients with Inborn Errors of bile Acid Metabolism | |||||||||||||
Medical condition: Patients with inborn errors of bile acid synthesis and metabolism | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2011-004491-10 | Sponsor Protocol Number: CAC-001-01 | Start Date*: 2011-10-06 | |||||||||||
Sponsor Name:Asklepion Pharmaceuticals, LLC | |||||||||||||
Full Title: An Open-Label, Single-Center, Nonrandomized Study to Compare the Therapeutic Efficacy of To Be Marketed (TBM) Cholic Acid Capsules with that of the Currently Used (CU) Formulation of Cholic Acid Ca... | |||||||||||||
Medical condition: Patients with inborn errors of bile acid synthesis and metabolism | |||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-004528-36 | Sponsor Protocol Number: CAC-002-01 | Start Date*: 2011-10-06 | |||||||||||
Sponsor Name:Asklepion Pharmaceuticals, LLC | |||||||||||||
Full Title: An open-label, single center, non-randomized, continuation study of cholic acid capsules in subjects with inborn errors of bile acid synthesis. | |||||||||||||
Medical condition: Patients with inborn errors of bile acid synthesis and metabolism | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2008-000071-25 | Sponsor Protocol Number: JE049-3101 | Start Date*: 2009-09-10 | |||||||||||
Sponsor Name:Jerini AG | |||||||||||||
Full Title: Open-Label, Multicenter Study to Evaluate Safety, Local Tolerability, Convenience, and Efficacy of a Self-Administered Subcutaneous Formulation of Icatibant for the Treatment of Acute Attacks of He... | |||||||||||||
Medical condition: hereditary angioedema, HAE (in adult patients with C1-esterase-inhibitor deficiency) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) AT (Completed) GB (Completed) FR (Completed) ES (Completed) IT (Completed) DK (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2014-003950-15 | Sponsor Protocol Number: rhLAMAN-10 | Start Date*: 2015-01-22 | |||||||||||
Sponsor Name:Zymenex A/S | |||||||||||||
Full Title: A single center, open label clinical trial investigating the long-term efficacy of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) treatment in subjects with alpha-Mannosidosis who previo... | |||||||||||||
Medical condition: alpha-Mannosidosis | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2013-000336-97 | Sponsor Protocol Number: rhLAMAN-07 | Start Date*: 2015-01-30 | |||||||||||
Sponsor Name:Chiesi Farmaceutici S.p.A. | |||||||||||||
Full Title: A multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis | |||||||||||||
Medical condition: Alpha-Mannosidosis | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Completed) FR (Ongoing) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-003131-35 | Sponsor Protocol Number: AA-HPP-208 | Start Date*: 2016-04-19 | |||||||||||
Sponsor Name:Alexion Pharma GmbH | |||||||||||||
Full Title: A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia | |||||||||||||
Medical condition: Hypophosphatasia is a rare inborn error of bone metabolism caused by inactivating mutations in the gene encoding the Tissue-nonspecific alkaline phosphatase isoenzyme. With deficiency of Tissue-no... | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-000474-29 | Sponsor Protocol Number: PTC923-MD-003-PKU | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:PTC Therapeutics, Inc. | |||||||||||||
Full Title: A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | |||||||||||||
Medical condition: Metabolic Disorders - Phenylketonuria | |||||||||||||
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Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) ES (Ongoing) PT (Completed) DK (Completed) NL (Completed) IT (Ongoing) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-000497-28 | Sponsor Protocol Number: PTC923-MD-004-PKU | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:PTC Therapeutics, Inc. | |||||||||||||
Full Title: Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria | |||||||||||||
Medical condition: Metabolic Disorders - Phenylketonuria | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Ongoing) ES (Ongoing) PT (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Ongoing) | |||||||||||||
Trial results: (No results available) |
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