Clinical Trials Register

Trials with a EudraCT protocol (1,387)
Paediatric studies in scope of Art45 of the Paediatric Regulation (0)

1,387 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)

Subscribe to this Search
To subscribe to the RSS feed for this search click here

. This will provide an RSS feed for clinical trials matching your search that have been added or updated in the last 7 days.

Download Options:
Number of Trials to download:

Download Content:

Download Format:


Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.

Query did not match any studies.

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials for Congenital disorders