Therapeutic area

Congenital, familial and genetic disorders – page 10

796 clinical trials
308 recruiting
374 conditions
27 countries

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

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796 clinical trials

Primary hyperoxaluria

Recruiting

InvestigationalNo placebo

Study on the Safety and Effects of ABO-101 with mRNA-002 and gRNA-001 for Patients with Primary Hyperoxaluria Type 1

InvestigationalNo placebo

France

Participants:0–17 years · 18–64 years
Substances:GRNA-001
Sponsor:Arbor Biotechnologies Inc.

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Duchenne muscular dystrophy+1

Recruiting

Registered drug

Study of ENTR-601-45 Safety and Effectiveness in Patients with Duchenne Muscular Dystrophy Who Can Skip Exon 45

Registered drug

Belgium

Participants:0–17 years · 18–64 years
Substances:Sodium Chloride
Sponsor:Entrada Therapeutics Inc.

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Trisomy 21+1

Recruiting

Registered drug

Study of levetiracetam to prevent seizures in adults who have both Down syndrome and Alzheimer's disease

Registered drug

Spain

Participants:18–64 years · 65+ years
Substances:Levetiracetam
Sponsor:Fundacio Institut De Recerca De L'Hospital De La Santa Creu I Sant Pau

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Von Willebrand's disease

Recruiting

Registered drugNo placebo

Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

Registered drugNo placebo

Belgium

Participants:0–17 years · 18–64 years · 65+ years
Substances:Efmoroctocog Alfa
Sponsor:F. Hoffmann-La Roche AG

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Duchenne muscular dystrophy+1

Recruiting

Registered drug

Study of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment

Registered drug

Belgium

Participants:0–17 years · 18–64 years
Substances:Sodium Chloride
Sponsor:Entrada Therapeutics Inc.

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Alagille syndrome+1

Recruiting

Registered drugNo placebo

Study on Long-Term Safety and Effects of Maralixibat for Patients with Alagille Syndrome

Registered drugNo placebo

Belgium

Participants:0–17 years · 18–64 years · 65+ years
Substances:MARALIXIBAT
Sponsor:Mirum Pharmaceuticals Inc.

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Heritable pulmonary arterial hypertension+1

Recruiting

Registered drugNo placebo

Study on Sotatercept for Improving Heart Function in Patients with Pulmonary Arterial Hypertension

Registered drugNo placebo

The Netherlands

Participants:18–64 years
Substances:Sotatercept
Sponsor:Amsterdam UMC Stichting

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Turner's syndrome

Recruiting

Registered drug

Study on Testosterone and Isopropyl Myristate for Women with Turner Syndrome

Registered drug

Denmark

Participants:18–64 years
Substances:CARBOMER 980
Sponsor:Region Midtjylland

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Pyruvate dehydrogenase complex deficiency

Recruiting

Registered drugNo placebo

Study of glycerol phenylbutyrate (oral liquid) for treatment of fatigue in patients with pyruvate dehydrogenase deficiency

Registered drugNo placebo

France

Participants:0–17 years · 18–64 years
Substances:Glycerol Phenylbutyrate
Sponsor:Assistance Publique Hopitaux De Paris

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Wiskott-Aldrich syndrome

Recruiting

InvestigationalNo placebo

Long-term Safety and Efficacy Study of Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

InvestigationalNo placebo

Italy

Participants:0–17 years · 18–64 years · 65+ years
Substances:Etuvetidigene Autotemcel
Sponsor:Fondazione Telethon Ets

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Leber's congenital amaurosis

Recruiting

Investigational

Study on the Effectiveness and Safety of Sepofarsen for Patients with Leber Congenital Amaurosis Due to a Specific Genetic Mutation

Investigational

Belgium

Participants:0–17 years · 18–64 years · 65+ years
Substances:SEPOFARSEN
Sponsor:Laboratoires Thea

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Type IIa hyperlipidaemia

Recruiting

Registered drugNo placebo

Study on Inclisiran, Alirocumab, and Rosuvastatin for Children with Familial Hypercholesterolemia

Registered drugNo placebo

Poland

Participants:0–17 years
Substances:Alirocumab
Sponsor:Medical University Of Lodz

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Substances studied in this area

The therapies tested most often in Congenital, familial and genetic disorders trials.

Sirolimus21 clinical trials Sodium Chloride21 clinical trials Prednisolone12 clinical trials Plerixafor11 clinical trials Busulfan10 clinical trials Ivacaftor9 clinical trials Mitapivat9 clinical trials Paracetamol8 clinical trials Tezacaftor8 clinical trials

Congenital, familial and genetic disorders – page 10

Conditions in this area

Clinical trials in Congenital, familial and genetic disorders

796 clinical trials

Primary hyperoxaluria

Study on the Safety and Effects of ABO-101 with mRNA-002 and gRNA-001 for Patients with Primary Hyperoxaluria Type 1

Duchenne muscular dystrophy+1

Study of ENTR-601-45 Safety and Effectiveness in Patients with Duchenne Muscular Dystrophy Who Can Skip Exon 45

Trisomy 21+1

Study of levetiracetam to prevent seizures in adults who have both Down syndrome and Alzheimer's disease

Von Willebrand's disease

Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

Duchenne muscular dystrophy+1

Study of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment

Alagille syndrome+1

Study on Long-Term Safety and Effects of Maralixibat for Patients with Alagille Syndrome

Heritable pulmonary arterial hypertension+1

Study on Sotatercept for Improving Heart Function in Patients with Pulmonary Arterial Hypertension

Turner's syndrome

Study on Testosterone and Isopropyl Myristate for Women with Turner Syndrome

Pyruvate dehydrogenase complex deficiency

Study of glycerol phenylbutyrate (oral liquid) for treatment of fatigue in patients with pyruvate dehydrogenase deficiency

Wiskott-Aldrich syndrome

Long-term Safety and Efficacy Study of Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

Leber's congenital amaurosis

Study on the Effectiveness and Safety of Sepofarsen for Patients with Leber Congenital Amaurosis Due to a Specific Genetic Mutation

Type IIa hyperlipidaemia

Study on Inclisiran, Alirocumab, and Rosuvastatin for Children with Familial Hypercholesterolemia

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Substances studied in this area

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