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Clinical trials for Developmental abnormalities

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44343   clinical trials with a EudraCT protocol, of which   7372   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    57 result(s) found for: Developmental abnormalities. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2006-005346-37 Sponsor Protocol Number: F1D-MC-HGMR Start Date*: Information not available in EudraCT
    Sponsor Name:Eli Lilly and Company Limited
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Maintenance of Effect Study of Olanzapine in the Treatment of Disruptive Behavioral Symptoms in Children and Adolescents with Pervasive Developmental ...
    Medical condition: children and adolescents with disruptive behavioral symptoms associated with Pervasive Developmental Disorders (PDD)
    Disease: Version SOC Term Classification Code Term Level
    8.1 10061345 Pervasive developmental disorder LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-001109-22 Sponsor Protocol Number: PBGM01-001 Start Date*: Information not available in EudraCT
    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 a Single Dose of PBGM01 Delivered into the Cisterna Magna of Pediatric Su...
    Medical condition: GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the human galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal).
    Disease:
    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: 2018-000504-42 Sponsor Protocol Number: ABT-003 Start Date*: 2019-05-21
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients with Middle and Advanced Phases of MPS IIIA Disease
    Medical condition: MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progress...
    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: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-002979-34 Sponsor Protocol Number: LTFU-ABO-102 Start Date*: 2020-05-18
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
    Medical condition: MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progress...
    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: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-001633-14 Sponsor Protocol Number: CBD_DEE Start Date*: 2020-03-05
    Sponsor Name:IRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA
    Full Title: A single-centre, open-label pilot study to assess the efficacy and safety of CBD oral solution as an adjunctive treatment for pediatric subjects with Developmental and Epileptic Encephalopathy
    Medical condition: Developmental and Epileptic Encephalopathy (DEE)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10077380 Epileptic encephalopathy PT
    20.0 10029205 - Nervous system disorders 10077380 Epileptic encephalopathy PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2019-003032-23 Sponsor Protocol Number: AADC-010 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Phase I/II Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-003072-39 Sponsor Protocol Number: NTUH-AADC-011 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-003904-21 Sponsor Protocol Number: ABT-001 Start Date*: 2016-08-01
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: Phase I/II gene transfer clinical trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
    Medical condition: MPS IIIA is a devastating lysosomal storage disease, caused by a N-sulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progres...
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-002396-35 Sponsor Protocol Number: XPF-009-301 Start Date*: 2021-11-15
    Sponsor Name:Xenon Pharmaceuticals Inc.
    Full Title: A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects with KCNQ2 Developmental and Epileptic Encephalopathy
    Medical condition: KCNQ2 Developmental and Epileptic Encephalopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10077380 Epileptic encephalopathy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) ES (Prematurely Ended) FR (Completed) IT (Prematurely Ended)
    Trial results: View results
    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: 2020-005995-37 Sponsor Protocol Number: COAV101A12306 Start Date*: 2021-08-25
    Sponsor Name:Novartis Pharma AG
    Full Title: A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric pa...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) DE (Completed) BE (Completed) PT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-006781-21 Sponsor Protocol Number: COAV101A12308 Start Date*: 2022-10-31
    Sponsor Name:Novartis Pharma AG
    Full Title: Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) DE (Completed) DK (Trial now transitioned) ES (Ongoing) GR (Prematurely Ended) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-019519-39 Sponsor Protocol Number: RAPIT Start Date*: 2012-03-14
    Sponsor Name:Erasmus MC - Department of Neurology
    Full Title: Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (RAPIT-trial)
    Medical condition: Tuberous Sclerosis Complex
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    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: 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: 2011-003784-30 Sponsor Protocol Number: CHUBX2011/20 Start Date*: Information not available in EudraCT
    Sponsor Name:CHU de bordeaux
    Full Title: Rubinstein-Taybi syndrome: approach to functional imaging and therapeutic trial
    Medical condition: Rubinstein-Taybi Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10039281 Rubinstein-Taybi syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-003031-35 Sponsor Protocol Number: R04049 Start Date*: 2015-11-20
    Sponsor Name:Central Manchester University Hospitals NHS Foundation Trust
    Full Title: A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitigating the Alloimmune Response to Intravenous Laronidase in Infants With Severe Mucopolysaccharido...
    Medical condition: Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10028094 Mucopolysaccharidosis IH LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-002619-24 Sponsor Protocol Number: A1481316 Start Date*: 2013-03-04
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: A MULTI-CENTRE, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND, TWO-ARMED, PARALLEL GROUP STUDY TO EVALUATE EFFICACY AND SAFETY OF IV SILDENAFIL IN THE TREATMENT OF NEONATES WITH PERSISTENT PULMONA...
    Medical condition: Persistent pulmonary hypertension of the newborn
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10053592 Newborn persistent pulmonary hypertension LLT
    Population Age: Preterm newborn infants, Newborns, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) GB (Completed) SE (Completed) ES (Prematurely Ended) AT (Completed) DE (Completed) NO (Completed) IT (Completed) NL (Completed) DK (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2015-001382-10 Sponsor Protocol Number: CS/2015/4775 Start Date*: 2019-01-21
    Sponsor Name:University Hospitals Bristol NHS Foundation Trust
    Full Title: Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service
    Medical condition: Barth Syndrome is a rare, life threatening, genetic disease which affects young males. It is caused by abnormal fats (lipids) in the powerhouses of cells (mitochondria) and those who suffer with it...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2018-004216-22 Sponsor Protocol Number: C602 Start Date*: 2019-09-25
    Sponsor Name:Soleno Therapeutics UK Ltd.
    Full Title: An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients with Prader-Willi Syndrome
    Medical condition: Hyperphagia associated with Prader-Willi Syndrome (PWS)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10027433 - Metabolism and nutrition disorders 10020710 Hyperphagia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
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