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Clinical trials for Developmental disorders

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   44338   clinical trials with a EudraCT protocol, of which   7368   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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    115 result(s) found for: Developmental disorders. Displaying page 2 of 6.
    « Previous 1  2  3  4  5  6  Next»
    EudraCT Number: 2013-001523-39 Sponsor Protocol Number: D1050300 Start Date*: 2013-11-08
    Sponsor Name:Sunovion Pharmaceuticals Inc.
    Full Title: A PHASE 1 OPEN-LABEL, MULTICENTER, SINGLE AND MULTIPLE ASCENDING DOSE STUDY TO EVALUATE PHARMACOKINETICS, SAFETY, AND TOLERABILITY OF LURASIDONE IN SUBJECTS 6 TO 17 YEARS OLD WITH SCHIZOPHRENIA SPE...
    Medical condition: schizophrenia
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004873 10001064 Acute schizophrenia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    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: 2020-003447-28 Sponsor Protocol Number: XPF-009-302 Start Date*: 2021-10-29
    Sponsor Name:Xenon Pharmaceuticals Inc.
    Full Title: An Open-Label Extension Study of 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: FR (Completed) BE (Completed) ES (Prematurely Ended) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2022-000241-32 Sponsor Protocol Number: NL80290.018.22 Start Date*: 2022-08-08
    Sponsor Name:Amsterdam UMC
    Full Title: Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series
    Medical condition: GRIN2B deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027424 Metabolic and nutritional disorders congenital HLGT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    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: 2020-005908-18 Sponsor Protocol Number: 1111_1111 Start Date*: 2021-11-03
    Sponsor Name:AOU FEDERICO II
    Full Title: Functional-genetic stratification as a guide to personalized treatment in developmental and epileptic encephalopathies due to potassium channel mutations
    Medical condition: Epileptic Encephalopathy with developmental delay
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10015039 Epilepsy congenital PT
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2007-006444-21 Sponsor Protocol Number: EC07/90076 Start Date*: 2009-02-13
    Sponsor Name:Fundación para la Investigación Biomédica Hospital Gregorio Marañón
    Full Title: EFFECT OF 8-WEEK FATTY ACIDS OMEGA-3 TREATMENT ON OXIDATIVE METABOLISM IN PATIENTS WITH AUTISM SPECTRUM DISORDER: A RANDOMISED DOUBLE-BLIND CROSSOVER PLACEBO-CONTROLLED TRIAL.
    Medical condition: autism spectrum disorders Trastornos del Espectro Autista
    Disease: Version SOC Term Classification Code Term Level
    9.1 10063844 Autism spectrum disorder LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-000266-29 Sponsor Protocol Number: AVXS-101-CL-302 Start Date*: 2018-06-12
    Sponsor Name:AveXis, Inc.
    Full Title: Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous ...
    Medical condition: Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) SE (Prematurely Ended) BE (Completed) ES (Prematurely Ended) NL (Prematurely Ended) IT (Completed)
    Trial results: View results
    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: 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: 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: 2013-001694-24 Sponsor Protocol Number: D1050302 Start Date*: 2014-03-11
    Sponsor Name:SUNOVION PHARMACEUTICALS INC.
    Full Title: A 104-WEEK, FLEXIBLE-DOSE, OPEN-LABEL, MULTICENTER, EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFECTIVENESS OF LURASIDONE IN PEDIATRIC SUBJECTS WITH SCHIZOPHRENIA AND SUBJECTS WITH IRRIT...
    Medical condition: SCHIZOPHRENIA AND IRRITABILITY ASSOCIATED WITH AUTISTIC DISORDER
    Disease: Version SOC Term Classification Code Term Level
    14.1 10037175 - Psychiatric disorders 10039628 Schizophrenia and other psychotic disorders HLGT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) IT (Prematurely Ended) BG (Completed) Outside EU/EEA HU (Completed) GB (Completed) DE (Completed) PL (Completed) BE (Completed) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2018-002485-39 Sponsor Protocol Number: TAK-935-18-001(OV935)Amendment1 Start Date*: 2019-06-14
    Sponsor Name:Ovid Therapeutics, Inc.
    Full Title: A PHASE 2, PROSPECTIVE, INTERVENTIONAL, OPEN-LABEL, MULTI-SITE, EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY AND TOLERABILITY OF TAK-935 (OV935) AS ADJUNCTIVE THERAPY IN PATIENTS WITH RARE EPILEP...
    Medical condition: developmental and epileptic encephalopathies
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) PL (Trial now transitioned) PT (Trial now transitioned) Outside EU/EEA
    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: 2010-021266-30 Sponsor Protocol Number: A1481283 Start Date*: 2011-12-14
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: A FOLLOW UP INVESTIGATION FOR PATIENTS COMPLETING STUDY A1481276 TO INVESTIGATE DEVELOPMENTAL PROGRESS 12 AND 24 MONTHS FOLLOWING COMPLETION OF SILDENAFIL TREATMENT
    Medical condition: Persistent pulmonary hypertension of the newborn (PPHN)
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10053592 Newborn persistent pulmonary hypertension LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    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: 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: 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: 2021-002550-82 Sponsor Protocol Number: NL76160.018.21 Start Date*: 2022-12-22
    Sponsor Name:Amsterdam UMC
    Full Title: Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series
    Medical condition: Gaucher disease type 3
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027424 Metabolic and nutritional disorders congenital HLGT
    Population Age: Children, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-004532-30 Sponsor Protocol Number: MRZ60201_3091_1 Start Date*: 2014-12-09
    Sponsor Name:Merz Pharmaceuticals GmbH
    Full Title: Prospective, randomized, double-blind, placebo-controlled, parallel-group, multicenter study with an open-label extension period to investigate the efficacy and safety of NT 201 in the treatment of...
    Medical condition: Chronic troublesome sialorrhea associated with neurological disorders (e.g. cerebral palsy, traumatic brain injury) and/or intellectual disability in children and adolescents
    Disease: Version SOC Term Classification Code Term Level
    19.0 10017947 - Gastrointestinal disorders 10039424 Salivary hypersecretion PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) PL (Completed) Outside EU/EEA
    Trial results: View results
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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