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Clinical trials for Developmental milestones

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   44347   clinical trials with a EudraCT protocol, of which   7375   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    13 result(s) found for: Developmental milestones. Displaying page 1 of 1.
    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: 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: 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: 2021-003214-40 Sponsor Protocol Number: MVX0004 Start Date*: 2022-01-13
    Sponsor Name:MinervaX Aps
    Full Title: A multicentre, multinational, parallel group, observer-blind, randomised, placebo-controlled study on the Group B Streptococcus vaccine (GBS-NN/NN2), investigating the immunogenicity and safety of ...
    Medical condition: Prevention of Group B Streptococcus infection in neonates.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021881 - Infections and infestations 10053588 Group B streptococcus neonatal sepsis PT
    Population Age: Newborns, Infants and toddlers, Under 18, Adults Gender: Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2020-001055-40 Sponsor Protocol Number: 111-209 Start Date*: 2020-07-29
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia at risk of requiring cervicom...
    Medical condition: achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2014-002053-19 Sponsor Protocol Number: CLMI070X2201 Start Date*: 2014-12-16
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10051203 Spinal muscular atrophy congenital LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) IT (Completed) DE (Completed) BE (Completed) NL (Ongoing) CZ (Completed) PL (Completed) HU (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2023-000864-67 Sponsor Protocol Number: COAV101A1IC01 Start Date*: 2024-02-02
    Sponsor Name:NOVARTIS BIOCIÊNCIAS S.A
    Full Title: A Phase IV Open-label, single-arm, single-dose, multicenter study to evaluate the saFEty, toLerability and effIcacy of gene replacement therapy with intravenous OAV101(AVXS101) in pediatric patien...
    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, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-002611-26 Sponsor Protocol Number: AVXS-101-LT-002 Start Date*: 2020-03-30
    Sponsor Name:AveXis, Inc.
    Full Title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
    Medical condition: Spinal Muscular Atrophy
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Trial now transitioned) ES (Prematurely Ended) FR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-002702-30 Sponsor Protocol Number: ALXN1101-MCD-202 Start Date*: Information not available in EudraCT
    Sponsor Name:Origin Biosciences, Inc.
    Full Title: A PHASE 2/3, MULTICENTER, MULTINATIONAL, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALXN1101 IN NEONATES WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A
    Medical condition: Molydenum CoFactor Deficiency (MoCD) Type A
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10069687 Molybdenum cofactor deficiency PT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) ES (Completed) FR (Completed) IT (Prematurely Ended) NO (Completed)
    Trial results: View results
    EudraCT Number: 2023-001104-33 Sponsor Protocol Number: AT0071002 Start Date*: 2023-12-22
    Sponsor Name:Applied Therapeutics Inc.
    Full Title: A Sequential, Two-Part Study to Evaluate the Clinical Benefit, Safety, Pharmacokinetics, and Pharmacodynamics of AT-007 in Pediatric Patients with Classic Galactosemia (CG)
    Medical condition: Galactosemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017610 Galactosemia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2011-002379-40 Sponsor Protocol Number: CAFQ056B2278 Start Date*: 2012-01-09
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome
    Medical condition: Fragile X syndrome
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Prematurely Ended) DE (Prematurely Ended) Outside EU/EEA GB (Prematurely Ended) ES (Temporarily Halted) DK (Prematurely Ended) IT (Completed) FR (Completed) NL (Prematurely Ended) BE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-015768-33 Sponsor Protocol Number: EMR700773-003/BMN162-503 Start Date*: 2011-01-04
    Sponsor Name:BioMarin International Ltd.
    Full Title: A Phase IIIb, Multicentre, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan®) in Phenylketonuria (PKU) Patient...
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) SK (Completed) DE (Completed) AT (Completed) CZ (Completed) PT (Completed) BE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-002667-34 Sponsor Protocol Number: WA39085 Start Date*: 2020-03-06
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: AN OPEN-LABEL, PARALLEL-GROUP STUDY TO EVALUATE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMIC EFFECTS OF OCRELIZUMAB IN CHILDREN AND ADOLESCENTS WITH RELAPSING-REMITTING MULTIPLE SCLE...
    Medical condition: Relapsing-remitting multiple sclerosis (MS)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10063399 Relapsing-remitting multiple sclerosis PT
    20.1 10029205 - Nervous system disorders 10039720 Sclerosis multiple LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
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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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