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Congenital, familial and genetic disorders – page 60

  • 803 clinical trials
  • 311 recruiting
  • 375 conditions
  • 27 countries

Conditions in this area

375 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Hypertrophic cardiomyopathy17Spinal muscular atrophy17Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Huntington's disease13Sickle cell anaemia13Congenital cystic kidney disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Epidermolysis bullosa10Fabry's disease10+351

Clinical trials in Congenital, familial and genetic disorders

803 trials across Europe, recruiting first

Type of trial
Recruitment status
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803 clinical trials

Primary immunodeficiency syndrome

Not recruiting

Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Octapharma Pharmazeutika Produktionsgesellschaft mbH

Fabry's disease

Not recruiting

Study on the Safety and Tolerability of ST-920 Gene Therapy for Patients with Fabry Disease

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years
  • Substances:Isaralgagene Civaparvovec
  • Sponsor:Sangamo Therapeutics Inc.

Short stature+3

Not recruiting

Study Comparing Somapacitan and Somatropin for Growth in Children with Short Stature Due to Small for Gestational Age, Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Somapacitan
  • Sponsor:Novo Nordisk A/S

Phenylketonuria

Not recruiting

Study to Evaluate mRNA-3210 for Safety and Tolerability in Patients with Phenylketonuria

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Codeine Phosphate
  • Sponsor:Moderna Inc.

Hereditary angioedema with C1 esterase inhibitor deficiency

Not recruiting

Study on Deucrictibant and Icatibant for Treating Hereditary Angioedema Attacks in Adolescents and Adults

Registered drug
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Deucrictibant
  • Sponsor:Pharvaris Netherlands B.V.

Sickle cell disease

Not recruiting

Study on the Effects of Etavopivat for Patients Aged 12-65 with Sickle Cell Disease

Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Etavopivat
  • Sponsor:Novo Nordisk A/S

Colour blindness

Not recruiting

Study on the Safety and Effectiveness of rAAV.hCNGA3 for Adults and Minors with CNGA3-Linked Achromatopsia

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Adeno-Associated Viral Vector Serotype 8 Containing The Human Cnga3 Gene Under The Control Of A Cone Arrestin Promoter
  • Sponsor:Universitaetsklinikum Tuebingen AöR

Huntington's disease

Not recruiting

Study on Long-Term Safety and Effects of PTC518 for Patients with Huntington's Disease

Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Votoplam
  • Sponsor:Novartis Pharma AG

Huntington's disease

Not recruiting

Study on the Safety and Effects of MBF-015 for Patients with Huntington's Disease

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years
  • Substances:MBF-015
  • Sponsor:Medibiofarma S.L.

Facioscapulohumeral muscular dystrophy

Not recruiting

Study on the Effectiveness and Safety of Satralizumab for Patients with Type 1 Facioscapulohumeral Muscular Dystrophy

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Satralizumab
  • Sponsor:Centre Hospitalier Universitaire De Nice

Severe myoclonic epilepsy of infancy

Not recruiting

Study on the Safety and Effects of Fenfluramine in Infants Aged 1 to Less Than 2 Years with Dravet Syndrome

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Fenfluramine Hydrochloride
  • Sponsor:UCB Biosciences Inc.

Osteochondrodysplasia+1

Not recruiting

Study on the Safety and Effects of SAR442501 for Children with Achondroplasia

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Substances studied in this area

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

Sirolimus21 clinical trialsSodium Chloride21 clinical trialsPrednisolone12 clinical trialsPlerixafor11 clinical trialsBusulfan10 clinical trialsIvacaftor9 clinical trialsMitapivat9 clinical trialsParacetamol8 clinical trialsTezacaftor8 clinical trials
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