Clinical Trials Register

Trials with a EudraCT protocol (1,385)
Paediatric studies in scope of Art45 of the Paediatric Regulation (0)

1,385 result(s) found for: Congenital disorders. Displaying page 6 of 70.

EudraCT Number: 2008-008435-29

Sponsor Protocol Number: 3082B2-4433-WW

Start Date*: Information not available in EudraCT

Sponsor Name:Wyeth Pharmaceuticals Inc. Acting through its division Wyeth Research, a Pfizer Company

Full Title: A Non-Randomized, Open-Label Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of ReFacto AF in Previously Treated Pediatric Subjects Less Than Twelve Years of Age With Severe Hemophilia...

Medical condition: Severe hemophilia A (FVIII:C <1%) in male subjects <12 years of age.

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	14.0	10010331 - Congenital, familial and genetic disorders	10018937	Haemophilia A	LLT

Population Age: Children, Under 18

Gender: Male

Trial protocol: FR (Completed) ES (Completed) IT (Completed) GR (Completed) CZ (Completed) SE (Completed) DK (Completed) FI (Completed) BG (Completed)

Trial results: View results

EudraCT Number: 2016-000898-20

Sponsor Protocol Number: MGT004

Start Date*: 2016-10-04

Sponsor Name:MeiraGTx UK Ltd

Full Title: Long term follow-up study of participants following an open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/5-OPTIRPE65) for gene therapy of adults a...

Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.0	10010331 - Congenital, familial and genetic disorders	10070667	Leber's congenital amaurosis	PT

Population Age: Children, Adolescents, Under 18, Adults

Gender: Male, Female

Trial protocol: GB (GB - no longer in EU/EEA)

Trial results: (No results available)

EudraCT Number: 2011-003072-37

Sponsor Protocol Number: 8HA01EXT

Start Date*: 2012-01-18

Sponsor Name:Biogen Idec Research Ltd

Full Title: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episode...

Medical condition: Hemophilia A

Disease:	Version	SOC Term	Classification Code	Term	Level
	19.0	100000004850	10053753	Hemophilia A without inhibitors	LLT
	19.0	100000004850	10060612	Hemophilia A	LLT

Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly

Gender: Male

Trial protocol: SE (Completed) BE (Completed) GB (Completed) DE (Completed) AT (Completed) ES (Completed) IT (Completed) IE (Completed) PL (Completed) NL (Completed)

Trial results: View results

EudraCT Number: 2018-004652-38

Sponsor Protocol Number: VX18-445-113

Start Date*: 2020-01-15

Sponsor Name:Vertex Pharmaceuticals Incorporated

Full Title: A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis

Medical condition: Cystic Fibrosis

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.0	10010331 - Congenital, familial and genetic disorders	10010331	Congenital, familial and genetic disorders	SOC

Population Age: Adolescents, Under 18, Adults, Elderly

Gender: Male, Female

Trial protocol: IE (Completed) GB (GB - no longer in EU/EEA) DE (Completed) DK (Completed) PL (Completed)

Trial results: View results

EudraCT Number: 2009-011186-88

Sponsor Protocol Number: ABB-09-001

Start Date*: 2009-09-08

Sponsor Name:FONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI

Full Title: Desarrollo de inhibidores en pacientes no tratados previamente (PUP) o en pacientes mínimamente tratados con componentes sanguíneos (MBCTP) al ser expuestos a concentrados de derivados plasmáticos ...

Medical condition: El estudio es un ensayo clínico abierto, aleatorizado, controlado, prospectivo, multicéntrico, internacional e independiente sobre la frecuencia de formación de inhibidores en pacientes no tratados...

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	9		10018937	Haemophilia A	LLT

Population Age: Newborns, Infants and toddlers, Children, Under 18

Gender: Male

Trial protocol: ES (Prematurely Ended) IT (Completed) PT (Completed) FR (Ongoing) DE (Completed) AT (Prematurely Ended) BE (Completed) GB (GB - no longer in EU/EEA) SK (Completed) CZ (Completed)

Trial results: (No results available)

EudraCT Number: 2013-001796-20

Sponsor Protocol Number: RD.03.SPR.40214E

Start Date*: 2013-12-17

Sponsor Name:Galderma R&D SNC

Full Title: Efficacy, safety and pharmacokinetics of 2 concentrations and 2 dosage regimens of CD5789 in subjects with Lamellar Ichthyosis

Medical condition: Lamellar Ichthyosis

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	16.1	100000004850	10023686	Lamellar ichthyosis	LLT

Population Age: Adults, Elderly

Gender: Male, Female

Trial protocol: SE (Prematurely Ended) DE (Completed)

Trial results: View results

EudraCT Number: 2022-001315-44

Sponsor Protocol Number: TAK-935-18-002(OV935)

Start Date*: 2022-05-24

Sponsor Name:Takeda Development Center America, Inc.

Full Title: A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder (ARCADE Study)

Medical condition: Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome

Disease:	Version	SOC Term	Classification Code	Term	Level
	20.0	10010331 - Congenital, familial and genetic disorders	10073682	Dravet syndrome	LLT
	20.1	10029205 - Nervous system disorders	10048816	Lennox-Gastaut syndrome	PT
	22.1	10010331 - Congenital, familial and genetic disorders	10083005	CDKL5 deficiency disorder	PT
	23.0	10010331 - Congenital, familial and genetic disorders	10083952	Dup15q syndrome	LLT

Population Age: Children, Adolescents, Under 18, Adults

Gender: Male, Female

Trial protocol: Outside EU/EEA

Trial results: View results

EudraCT Number: 2016-003226-16

Sponsor Protocol Number: BP-404

Start Date*: 2017-02-03

Sponsor Name:Bellicum Pharmaceuticals, Inc.

Full Title: Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders

Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	19.0	10005329 - Blood and lymphatic system disorders	10018849	Haematological disorders NEC	HLGT

Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults

Gender: Male, Female

Trial protocol: IT (Prematurely Ended)

Trial results: (No results available)

EudraCT Number: 2019-003427-38

Sponsor Protocol Number: MOTIVATE

Start Date*: 2020-03-12

Sponsor Name:HZRM – Hämophilie-Zentrum Rhein Main GmbH

Full Title: MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – An International Low-Interventional Pragmatic Investigator Initiated Trial

Medical condition: Inhibitor-Positive patients with Haemophilia A

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.0	10010331 - Congenital, familial and genetic disorders	10060612	Hemophilia A	LLT

Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly

Gender: Male

Trial protocol: DE (Trial now transitioned) NO (Trial now transitioned) SE (Trial now transitioned) FI (Trial now transitioned) HR (Trial now transitioned)

Trial results: (No results available)

EudraCT Number: 2015-000906-20

Sponsor Protocol Number: LUM001-601

Start Date*: 2015-05-13

Sponsor Name:Shire Human Genetic Therapies Inc

Full Title: An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) ...

Medical condition: Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)

Disease:	Version	SOC Term	Classification Code	Term	Level
	17.1	10010331 - Congenital, familial and genetic disorders	10010331	Congenital, familial and genetic disorders	SOC
	17.1	10010331 - Congenital, familial and genetic disorders	10053870	Alagille syndrome	PT

Population Age: Infants and toddlers, Children, Adolescents, Under 18

Gender: Male, Female

Trial protocol: GB (Prematurely Ended)

Trial results: View results

EudraCT Number: 2006-003360-56

Sponsor Protocol Number: 20050183

Start Date*: 2009-08-28

Sponsor Name:Dept. of Pediatrics, The Childrens Hospital, Denver

Full Title: Farmakologisk behandling af CNDI – Et ”proof-of-concept” studie.

Medical condition: Kongenit nefrogen diabetes insipidus

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	14.1	10010331 - Congenital, familial and genetic disorders	10068304	Congenital nephrogenic diabetes insipidus	PT

Population Age: Children, Adolescents, Under 18, Adults

Gender: Male

Trial protocol: DK (Prematurely Ended)

Trial results: (No results available)

EudraCT Number: 2011-001455-37

Sponsor Protocol Number: 1331TMF

Start Date*: 2011-12-21

Sponsor Name:Department of Radiology, Oslo University Hospital, Norway

Full Title: Prospective randomized interventional study. Compare the effect of bleomycin and Tetradecyl Sodium Sulphate (Fibrovein) in the treatment of venous malformations. Clinical neurological and neurophys...

Medical condition: Venous malformation

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	13.1	10010331 - Congenital, familial and genetic disorders	10047229	Venous malformation NOS	LLT

Population Age: Adolescents, Under 18, Adults, Elderly

Gender: Male, Female

Trial protocol: NO (Completed)

Trial results: (No results available)

EudraCT Number: 2022-003877-48

Sponsor Protocol Number: TAK-662-1501

Start Date*: 2022-12-21

Sponsor Name:Takeda

Full Title: An Open-Label, Single-Dose, Phase 1/2 Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Human Protein C (TAK-662) for the Treatment of Congenital Protein C Deficiency in Japanese ...

Medical condition: Congenital Protein C Deficiency

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.1	10010331 - Congenital, familial and genetic disorders	10051298	Protein C deficiency	PT

Population Age: Children, Adolescents, Under 18, Adults

Gender: Male, Female

Trial protocol: Outside EU/EEA

Trial results: View results

EudraCT Number: 2009-017978-21

Sponsor Protocol Number: EMR700568-012

Start Date*: 2010-12-20

Sponsor Name:Merck Serono S.A. - Geneva

Full Title: Prospective observational long-term safety registry of Multiple Sclerosis patients who have participated in cladribine clinical trials (PREMIERE)

Medical condition: Multiple Sclerosis patients who have participated in cladribine tablets clinical trials

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.1	10029205 - Nervous system disorders	10028245	Multiple sclerosis	PT

Population Age: Adults, Elderly

Gender: Male, Female

Trial protocol: DE (Completed) FI (Completed) LT (Completed) AT (Completed) GR (Completed) CZ (Completed) GB (Completed) EE (Completed) LV (Completed) BE (Completed) SE (Completed) PT (Completed) DK (Completed) ES (Completed) IT (Completed) PL (Completed) BG (Completed)

Trial results: View results

EudraCT Number: 2008-008436-93

Sponsor Protocol Number: B1831006(3082B2-4434-WW)

Start Date*: 2009-12-16

Sponsor Name:Wyeth Pharmaceutical Inc. acting through its division Wyeth Research, a Pfizer Company

Full Title: An Open-Label Study of the Safety and Efficacy of ReFacto AF in Previously Untreated Patients in Usual Care Settings

Medical condition: Severe hemophilia A (FVIII:C <1%) in male subjects <6 years of age.

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	17.0	100000004850	10018937	Haemophilia A	LLT

Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18

Gender: Male

Trial protocol: DE (Completed) FR (Completed) AT (Completed) ES (Completed) IT (Completed) SE (Completed) NL (Completed) GR (Completed)

Trial results: View results

EudraCT Number: 2016-004623-23

Sponsor Protocol Number: AMO-02-MD-2-003

Start Date*: 2018-11-19

Sponsor Name:AMO Pharma Ltd

Full Title: A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy

Medical condition: Treatment of child and adolescent congenital myotonic dystrophy.

Disease:	Version	SOC Term	Classification Code	Term	Level
	20.0	10010331 - Congenital, familial and genetic disorders	10068871	Myotonic dystrophy	PT
	20.0	10010331 - Congenital, familial and genetic disorders	10068871	Myotonic dystrophy	PT

Population Age: Children, Adolescents, Under 18

Gender: Male, Female

Trial protocol: GB (GB - no longer in EU/EEA)

Trial results: View results

EudraCT Number: 2019-003213-34

Sponsor Protocol Number: 270-205

Start Date*: 2020-09-25

Sponsor Name:BioMarin Pharmaceutical Inc.

Full Title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Active or Prior Inhibitors

Medical condition: Hemophilia A

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.0	100000004850	10060612	Hemophilia A	LLT

Population Age: Adults, Elderly

Gender: Male

Trial protocol: GB (GB - no longer in EU/EEA) IT (Prematurely Ended) DE (Prematurely Ended)

Trial results: (No results available)

EudraCT Number: 2012-001394-87

Sponsor Protocol Number: EBSTEM001

Start Date*: 2012-11-16

Sponsor Name:King's College London

Full Title: A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa.

Medical condition: Recessive Dystrophic Epidermolysis Bullosa

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	17.0	10010331 - Congenital, familial and genetic disorders	10010331	Congenital, familial and genetic disorders	SOC

Population Age: Infants and toddlers, Children, Adolescents, Under 18

Gender: Male, Female

Trial protocol: GB (Completed)

Trial results: View results

EudraCT Number: 2011-004800-40

Sponsor Protocol Number: AT1001-041

Start Date*: 2012-01-25

Sponsor Name:Amicus Therapeutics, Inc.

Full Title: An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease.

Medical condition: Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme α-galactosidase A.

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	17.0	10010331 - Congenital, familial and genetic disorders	10010331	Congenital, familial and genetic disorders	SOC

Population Age: Adults

Gender: Male, Female

Trial protocol: GB (Completed) ES (Completed) IT (Completed) DK (Completed) BE (Completed) AT (Completed)

Trial results: View results

EudraCT Number: 2021-001959-13

Sponsor Protocol Number: EPICDKL5

Start Date*: 2021-12-02

Sponsor Name:FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE

Full Title: Adjunctive cannabidiol therapy in patients with CDKL5 deficiency disorder. Interventional drug study on efficacy and safety with focus on seizure effects

Medical condition: patients with CDKL5

Disease:	Version	SOC Term	Classification Code	Term	Level
Disease:	20.0	10010331 - Congenital, familial and genetic disorders	10015039	Epilepsy congenital	PT

Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults

Gender: Male, Female

Trial protocol: IT (Completed)

Trial results: (No results available)

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Clinical trials for Congenital disorders