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Congenital, familial and genetic disorders – page 53

  • 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

Hepato-lenticular degeneration

Not recruiting

Study on Gene Therapy with VTX-801 and Copper (64Cu) Chloride for Adults with Wilson's Disease

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Substances:Adeno-Associated Viral Vector Serotype 3B Encoding Shortened Human Atp7B
  • Sponsor:Vivet Therapeutics

Hereditary angioedema

Not recruiting

Study on Long-Term Safety of Donidalorsen for Patients with Hereditary Angioedema

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Donidalorsen
  • Sponsor:Ionis Pharmaceuticals Inc.

Myotonic dystrophy

Not recruiting

Safety and effectiveness study of DYNE-101 in patients with Myotonic Dystrophy Type 1 (DM1)

Investigational
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Humanised Igg1 Kappa Fragment Antibody Targeting Tfr1 Conjugated To P125 Oligonucleotide
  • Sponsor:Dyne Therapeutics Inc.

Osteochondrodysplasia

Not recruiting

Study on the Effects of Navepegritide and Lonapegsomatropin in Children with Achondroplasia

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Substances:Lonapegsomatropin
  • Sponsor:Ascendis Pharma Growth Disorders A/S

Mucopolysaccharidosis I

Not recruiting

Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Busulfan
  • Sponsor:Orchard Therapeutics (Europe) Limited

Alpha-1 antitrypsin deficiency+1

Not recruiting

Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Human ALPHA-1-PROTEINASE inhibitor immunoglobulin G fusion protein, recombinant
  • Sponsor:Sanofi AATD Inc.

Facioscapulohumeral muscular dystrophy

Not recruiting

Study on the Long-Term Safety and Effectiveness of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Losmapimod
  • Sponsor:Fulcrum Therapeutics Inc.

Hypertrophic cardiomyopathy

Not recruiting

Study of Mavacamten for Adolescents with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Investigational
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Mavacamten
  • Sponsor:Bristol-Myers Squibb Services Unlimited Company

K-ras gene mutation+1

Not recruiting

Study of Sotorasib Treatment in Patients with Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with KRAS G12C Mutation Who Have Previously Received Treatment

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Sotorasib
  • Sponsor:Institut Gustave Roussy

Fibrodysplasia ossificans progressiva

Not recruiting

Study on the Effects of Fidrisertib for Treating Fibrodysplasia Ossificans Progressiva in Children and Adults

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:(R)‑Tetrahydrofuran‑3‑Yl 4‑(6‑(5‑(4‑Ethoxy‑1‑Isopropylpiperidin‑4‑Yl)Pyridin-2-Yl)Pyrrolo[1,2-B]Pyridazin-4-Yl)Piperazine-1-Carboxylate Sesquisuccinate
  • Sponsor:Clementia Pharmaceuticals Inc. An Ipsen Company

Gaucher's disease type III+1

Not recruiting

Study of Eliglustat alone or with imiglucerase in children and adolescents aged 2-17 years with Gaucher disease type 1 and type 3

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Eliglustat Tartrate
  • Sponsor:Genzyme Corp.

Ataxia telangiectasia

Not recruiting

Study on the Effects of EryDex (Dexamethasone Sodium Phosphate) for Children Aged 6-9 with Ataxia Telangiectasia

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Dexamethasone Sodium Phosphate
  • Sponsor:Quince Therapeutics S.p.A.

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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