- Trials with a EudraCT protocol (800)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (2)
800 result(s) found for: Linked.
Displaying page 1 of 40.
EudraCT Number: 2016-000600-29 | Sponsor Protocol Number: UX023-CL301 | Start Date*: 2016-09-15 | |||||||||||
Sponsor Name:Ultragenyx Pharmaceuticals Inc | |||||||||||||
Full Title: A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients with X-linked Hypophosphatemia (XLH) | |||||||||||||
Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, defective bone mineralisation, and impaired growth plate or endochondral ossification caused by inactivating mutations in t... | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Prematurely Ended) IE (Completed) DE (Completed) ES (Prematurely Ended) SE (Completed) GB (Completed) IT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-005015-82 | Sponsor Protocol Number: NGAM-05 | Start Date*: 2012-01-10 | ||||||||||||||||
Sponsor Name:Octapharma AG | ||||||||||||||||||
Full Title: Clinical study to evaluate the safety and tolerability of immunoglobulin intravenous (human) 10% (NewGam) administered at high infusion rates to patients with primary immunodeficiency diseases (ext... | ||||||||||||||||||
Medical condition: primary immunodeficiency diseases | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: Outside EU/EEA | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2014-000406-35 | Sponsor Protocol Number: UX023-CL201 | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:Ultragenyx Pharmaceuticals Inc. | |||||||||||||
Full Title: A Randomized, open Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the anti-FGF23 antibody, KRN23, in Pediatric Patients with X-linked Hypophosphatemia (XLH) | |||||||||||||
Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, and the most common heritable form of rickets. In XLH patients, high circulating levels of fibroblast growth factor 23 (FG... | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) NL (Completed) FR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-002532-23 | Sponsor Protocol Number: ER004-CLIN01/F60082AI201 | Start Date*: 2021-10-25 | |||||||||||
Sponsor Name:EspeRare Foundation [...] | |||||||||||||
Full Title: A prospective, open-label, genotype-match controlled, multicenter clinical trial to investigate the efficacy and safety of intra-amniotic ER004 as a prenatal treatment for male subjects with X-link... | |||||||||||||
Medical condition: X-linked hypohidrotic ectodermal dysplasia (XLHED) | |||||||||||||
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Population Age: In utero, Under 18 | Gender: Male | ||||||||||||
Trial protocol: DE (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2013-004565-14 | Sponsor Protocol Number: ECP-002e | Start Date*: 2014-02-13 | |||||||||||
Sponsor Name:Edimer Pharmaceuticals, Inc. | |||||||||||||
Full Title: Extension Study of XLHED-Affected Male Subjects treated with EDI200 in Protocol ECP-002 | |||||||||||||
Medical condition: X-linked hypohidrotic ectodermal dysplasia (XLHED) is caused by inherited defects in the ectodysplasin (EDA) gene that disrupt synthesis and/or function of the primary translational product EDA-A1.... | |||||||||||||
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Population Age: Infants and toddlers, Under 18 | Gender: Male | ||||||||||||
Trial protocol: DE (Completed) FR (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2018-000202-37 | Sponsor Protocol Number: BUR02 | Start Date*: 2018-10-09 | |||||||||||
Sponsor Name:Kyowa Kirin Pharmaceutical Development Ltd | |||||||||||||
Full Title: A Phase 3b Open-label Study of the Anti-FGF23 Antibody, Burosumab (KRN23) in Adult Patients with X-linked Hypophosphatemia (XLH) | |||||||||||||
Medical condition: XLH is a rare, genetic disorder that is serious, chronically debilitating and represents an unmet medical need. This genetic deficiency is estimated to occur in about 1:20,000 live births (Burn... | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Completed) GB (GB - no longer in EU/EEA) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2009-011434-10 | Sponsor Protocol Number: NGAM-01 | Start Date*: 2010-03-02 | ||||||||||||||||
Sponsor Name:OCTAPHARMA AG | ||||||||||||||||||
Full Title: CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES | ||||||||||||||||||
Medical condition: Primary immunodeficiency diseases | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: DE (Completed) Outside EU/EEA | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2012-000792-16 | Sponsor Protocol Number: GAMr-29 | Start Date*: 2012-06-14 | ||||||||||||||||
Sponsor Name:OCTAPHARMA AG | ||||||||||||||||||
Full Title: “CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES” | ||||||||||||||||||
Medical condition: primary immunodeficiency disease (PID) | ||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: HU (Completed) CZ (Completed) DE (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2014-005529-11 | Sponsor Protocol Number: UX023-CL303 | Start Date*: 2016-02-01 | |||||||||||
Sponsor Name:Ultragenyx Pharmaceutical Inc. | |||||||||||||
Full Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study with Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH) | |||||||||||||
Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phospha... | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) IE (Completed) FR (Completed) DK (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-000469-19 | Sponsor Protocol Number: BUR-CL207 | Start Date*: 2020-11-03 | |||||||||||
Sponsor Name:Kyowa Kirin Pharmaceutical Development Ltd. | |||||||||||||
Full Title: A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Pediatric Patients from Birth to Less than 1 Year of Ag... | |||||||||||||
Medical condition: XLH is a rare, genetic disorder that is serious, chronically debilitating and represents an unmet medical need. This genetic deficiency is estimated to occur in about 1:20,000 live births (Burnett ... | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) DE (Prematurely Ended) SE (Completed) AT (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-001775-41 | Sponsor Protocol Number: UX023-CL304 | Start Date*: 2015-12-16 | |||||||||||
Sponsor Name:Ultragenyx Pharmaceutical Inc. | |||||||||||||
Full Title: An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults with X-linked Hypophosphatemia (XLH) | |||||||||||||
Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phospha... | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DK (Completed) FR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2009-010609-35 | Sponsor Protocol Number: FLG090205 | Start Date*: 2009-09-10 |
Sponsor Name:Department of Medical Sciences, Dermatology and venerology, Uppsala University, Uppsala Sweden | ||
Full Title: Is treatment with emollients always beneficial? Comparative biophysical and molecularbiological studies of the skinbarrier function in patients with atopic dermatitis, ichtyosis vulgaris and x-li... | ||
Medical condition: Patients with Atopic Dermatitis, Ichtyosis vulgaris and X-linked Ichtyosis | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: SE (Completed) | ||
Trial results: (No results available) |
EudraCT Number: 2012-003561-17 | Sponsor Protocol Number: ECP-002 | Start Date*: 2013-08-09 | |||||||||||
Sponsor Name:Edimer Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 2 open-label, dose-escalation study to evaluate the safety, pharmacokinetics, immunogeniticy and pharmacodynamics/efficacy of EDI200, an EDA-A1 replacement protein, administered to male inf... | |||||||||||||
Medical condition: X-linked hypohidrotic ectodermal dysplasia (XLHED) is caused by inherited defects in the ectodysplasin (EDA) gene that disrupt synthesis and/or function of the primary translational product EDA-A1.... | |||||||||||||
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Population Age: Newborns, Under 18 | Gender: Male | ||||||||||||
Trial protocol: DE (Completed) GB (Completed) IT (Completed) FR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-002805-13 | Sponsor Protocol Number: LTF-304 | Start Date*: 2015-11-27 | |||||||||||
Sponsor Name:bluebird bio, Inc | |||||||||||||
Full Title: Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | |||||||||||||
Medical condition: Cerebral Adrenoleukodystrophy (CALD) | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-000122-10 | Sponsor Protocol Number: NS-065/NCNP-01-302 | Start Date*: 2021-06-30 | |||||||||||
Sponsor Name:NS Pharma, Inc. | |||||||||||||
Full Title: A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy (DMD) | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male | ||||||||||||
Trial protocol: NL (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) GR (Trial now transitioned) NO (Trial now transitioned) CZ (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2010-024566-22 | Sponsor Protocol Number: DMD114118 | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:GlaxoSmithKline Research and Development LTD | |||||||||||||
Full Title: A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subject... | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: FR (Ongoing) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-000654-59 | Sponsor Protocol Number: MT-2-02 | Start Date*: 2019-06-25 | |||||||||||
Sponsor Name:Minoryx Therapeutics S.L. | |||||||||||||
Full Title: An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral... | |||||||||||||
Medical condition: Cerebral X-linked Adrenoleukodystrophy (cALD) | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: ES (Ongoing) DE (Trial now transitioned) FR (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2016-005023-92 | Sponsor Protocol Number: 4658-203 | Start Date*: 2017-03-28 | |||||||||||
Sponsor Name:Sarepta Therapeutics, Inc. | |||||||||||||
Full Title: An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy | |||||||||||||
Medical condition: Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2016-005002-19 | Sponsor Protocol Number: 4658-301 | Start Date*: 2017-03-28 | |||||||||||
Sponsor Name:Sarepta Therapeutics, Inc. | |||||||||||||
Full Title: An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy | |||||||||||||
Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2013-003605-26 | Sponsor Protocol Number: PRO044-CLIN-02 | Start Date*: 2014-12-02 | |||||||||||
Sponsor Name:Prosensa Therapeutics B.V. | |||||||||||||
Full Title: A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy | |||||||||||||
Medical condition: Duchenne muscular dystrophy | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: IT (Completed) SE (Prematurely Ended) NL (Prematurely Ended) BE (Completed) | |||||||||||||
Trial results: View results |
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