- Trials with a EudraCT protocol (89)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
89 result(s) found for: Gene Deletion.
Displaying page 1 of 5.
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 | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: IE (Restarted) GB (GB - no longer in EU/EEA) FR (Ongoing) ES (Ongoing) SE (Prematurely Ended) DE (Restarted) DK (Restarted) NL (Ongoing) PL (Ongoing) GR (Ongoing) NO (Ongoing) HU (Ongoing) CZ (Ongoing) SI (Ongoing) RO (Ongoing) | |||||||||||||
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 | |||||||||||||
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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*: Information not available in EudraCT |
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 (Ongoing) ES (Ongoing) DE (Ongoing) GR (Ongoing) NL (Ongoing) | ||
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 | |||||||||||||
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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 | |||||||||||||
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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 [...] | |||||||||||||
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 | |||||||||||||
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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 (Ongoing) | ||
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 | ||||||||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | Gender: Male | |||||||||||||||||
Trial protocol: ES (Ongoing) DE (Ongoing) IT (Ongoing) FR (Ongoing) | ||||||||||||||||||
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 | |||||||||||||
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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 (Restarted) | |||||||||||||
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 | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) BE (Ongoing) SE (Completed) DE (Completed) FR (Ongoing) ES (Ongoing) CZ (Ongoing) NL (Ongoing) IT (Ongoing) BG (Ongoing) PL (Ongoing) IE (Ongoing) DK (Ongoing) GR (Ongoing) 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: 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... | ||||||||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: NL (Completed) | ||||||||||||||||||
Trial results: View results |
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