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Clinical trials for Gene deletion

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   44344   clinical trials with a EudraCT protocol, of which   7373   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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    91 result(s) found for: Gene deletion. Displaying page 1 of 5.
    1  2  3  4  5  Next»
    EudraCT Number: 2018-001762-42 Sponsor Protocol Number: 4658-402 Start Date*: 2020-04-03
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular ...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: IE (Completed) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) ES (Ongoing) SE (Prematurely Ended) DE (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) NO (Trial now transitioned) HU (Trial now transitioned) CZ (Trial now transitioned) SI (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-001235-27 Sponsor Protocol Number: AVXS-101-CL-101 Start Date*: 2020-04-06
    Sponsor Name:AveXis, Inc.
    Full Title: Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101
    Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-000095-28 Sponsor Protocol Number: H3E-MC-JMHW Start Date*: 2017-03-02
    Sponsor Name:Eli Lilly and Company
    Full Title: A Phase II Study of Pemetrexed in Children with Recurrent Malignancies
    Medical condition: Recurrent Malignancies (recurrent solid tumors): Target tumor types were osteosarcoma, Ewing sarcoma/peripheral PNET, rhabdomyosarcoma, neuroblastoma (measurable disease), neuroblastoma (metaiodob...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    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: 2009-011513-24 Sponsor Protocol Number: CC-5013-MDS-005 Start Date*: 2009-12-14
    Sponsor Name:Celgene Corporation
    Full Title: A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO COMPARE THE EFFICACY AND SAFETY OF LENALIDOMIDE (REVLIMID®) VERSUS PLACEBO IN SUBJECTS WITH TRANSFUSION...
    Medical condition: TRANSFUSION-DEPENDENT ANEMIA DUE TO IPSS LOW OR INTERMEDIATE-1 RISK MYELODYSPLASTIC SYNDROMES WITHOUT DELETION 5Q [31] AND UNRESPONSIVE OR REFRACTORY TO ERYTHROPOIESIS-STIMULATING AGENT
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004864 10068361 MDS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) ES (Completed) DE (Completed) FR (Completed) CZ (Completed) AT (Completed) IT (Completed) PT (Completed) GB (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2017-004087-35 Sponsor Protocol Number: AVXS-101-CL-304 Start Date*: 2018-12-21
    Sponsor Name:AveXis, Inc.
    Full Title: A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2
    Medical condition: Spinal Muscular Atrophy
    Disease:
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) IT (Completed) GB (GB - no longer in EU/EEA) BE (Completed) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-002873-24 Sponsor Protocol Number: RM-493-035 Start Date*: 2022-05-16
    Sponsor Name:Rhythm Pharmaceuticals Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melano...
    Medical condition: Specific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Ongoing) DE (Trial now transitioned) GR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-022509-17 Sponsor Protocol Number: ML25434 Start Date*: 2010-12-13
    Sponsor Name:Roche Farmacêutica Química, Lda
    Full Title: Phase II, open-label study of erlotinib (Tarceva®) treatment in patients with locally advanced or metastatic non-small cell lung cancer who present activating mutations in the tyrosine kinase domai...
    Medical condition: Patients with histologically or cytologically confirmed locally advanced or metastatic NSCLC who have not received previous chemotherapy for their disease and who present activating mutations in th...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Completed)
    Trial results: View results
    EudraCT Number: 2010-019983-35 Sponsor Protocol Number: VPA-CdA Start Date*: 2010-11-24
    Sponsor Name:Cliniques universitaires Saint Luc
    Full Title: Phase I-II study of low dose CdA combined with valproic acid (VPA) in previously treated B-cell chronic lymphocytic leukemia (CLL) patients.
    Medical condition: •B-CLL, as defined by the NCIWG criteria •Patients must have intermediate or high-risk categories of the modified 3-stage Rai and Binet stagings
    Disease: Version SOC Term Classification Code Term Level
    14.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008977 Chronic lymphocytic leukemia recurrent LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-003667-11 Sponsor Protocol Number: M15-550 Start Date*: 2016-08-01
    Sponsor Name:AbbVie Deutschland GmbH & Co. KG
    Full Title: Open-Label, Single Arm, Phase 3B, Multi-Center Study Evaluating the Efficacy of Venetoclax (ABT 199) in Relapsed/Refractory Subjects with Chronic Lymphocytic Leukemia (CLL) (VENICE I)
    Medical condition: Chronic Lymphocytic Leukemia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008958 Chronic lymphocytic leukaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GR (Completed) SE (Completed) BE (Completed) NL (Completed) DE (Completed) AT (Completed) IE (Completed) PT (Completed) FI (Completed) DK (Completed) ES (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2010-024551-82 Sponsor Protocol Number: ATOM_22qDS/2011 Start Date*: 2012-03-07
    Sponsor Name:King's College London [...]
    1. King's College London
    2. South London and Maudsley NHS Foundation Trust
    Full Title: OPEN LABEL TRIAL OF ATOMOXETINE FOR ATTENTION DEFICIT HYPERACTIVITY DISORDER (ADHD) IN CHILDREN WITH 22q11.2 DELETION SYNDROME (22qDS)
    Medical condition: Attention Deficit Hyperactivity Disorder (ADHD) in children with 22qDS deletion syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10037175 - Psychiatric disorders 10064104 ADHD LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-000327-27 Sponsor Protocol Number: ISIS396443-CS2 Start Date*: 2017-01-26
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2005-002520-33 Sponsor Protocol Number: SNT-II-1 Start Date*: 2005-09-06
    Sponsor Name:Santhera Pharmaceuticals LLC
    Full Title: A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac...
    Medical condition: Duchenne Muscular Dystrophy
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2016-000784-16 Sponsor Protocol Number: NCPACI Start Date*: 2016-04-15
    Sponsor Name:Akershus University Hospital
    Full Title: Focal cartilage defects in the knee – A randomized controlled trial comparing Autologous Chondrocyte Implantation with arthroscopic debridement
    Medical condition: Symptomatic focal cartilage defects in the knee
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NO (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-002069-52 Sponsor Protocol Number: 4045-301 Start Date*: 2017-01-23
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
    Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Trial now transitioned) SE (Completed) DE (Completed) FR (Ongoing) ES (Trial now transitioned) CZ (Trial now transitioned) NL (Ongoing) IT (Trial now transitioned) BG (Trial now transitioned) PL (Trial now transitioned) IE (Trial now transitioned) DK (Trial now transitioned) GR (Completed) NO (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-001335-12 Sponsor Protocol Number: D5160C00030 Start Date*: 2016-05-12
    Sponsor Name:Hellenic Oncology Research Group
    Full Title: A longitudinal study evaluating molecular changes associated with resistance to first and third (AZD9291) generation EGFR TKIs in patients with EGFR mutant NSCLC using "liquid biopsy"
    Medical condition: Patients with NSCLC
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: GR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000601-77 Sponsor Protocol Number: 5051-201 Start Date*: 2019-12-11
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IE (Prematurely Ended) ES (Temporarily Halted) GB (GB - no longer in EU/EEA) BE (Trial now transitioned) NL (Restarted) DE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-000814-23 Sponsor Protocol Number: CLDK378A2407 Start Date*: 2015-10-26
    Sponsor Name:Novartis Farmacéutica, S.A.
    Full Title: A Phase II, open label, multi-center, multi-arm study of ceritinib in patients with advanced solid tumors and hematological malignancies characterized by genetic abnormalities of anaplastic lymphom...
    Medical condition: Advanced solid tumors and hematological malignancies with ALK genetic alteration and/or overexpression
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) DE (Temporarily Halted) CZ (Completed) FR (Prematurely Ended) DK (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-004369-34 Sponsor Protocol Number: UMC-NMZ-SMA2011 Start Date*: 2015-04-22
    Sponsor Name:Universtiy Medical Center Utrecht
    Full Title: SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients ...
    Medical condition: Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene...
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10068209 Spinal muscular atrophy adult onset LLT
    17.1 100000004850 10051203 Spinal muscular atrophy congenital LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
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