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Congenital, familial and genetic disorders – page 49

  • 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
Quick filters

803 clinical trials

Factor VIII deficiency

Not recruiting

Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Efanesoctocog Alfa
  • Sponsor:Swedish Orphan Biovitrum AB (publ)

Pyogenic sterile arthritis pyoderma gangrenosum and acne syndrome

Not recruiting

Study on Canakinumab for Patients with PAPA Syndrome

Registered drug
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Canakinumab
  • Sponsor:IRCCS Istituto Giannina Gaslini

Retinitis pigmentosa+1

Not recruiting

Study of AAVB-081 and Prednisolone for Patients with Usher Syndrome Type 1B Retinitis Pigmentosa

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years
  • Substances:Prednisolone
  • Sponsor:Aavantgarde Bio S.r.l.

Adrenoleukodystrophy

Not recruiting

Study on the Safety and Effectiveness of SBT101 Gene Therapy for Adults with Adrenomyeloneuropathy

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years
  • Substances:Adeno-Associated Viral Vector Serotype 9 Containing The Human Habcd1 Gene
  • Sponsor:Swanbio Therapeutics Limited

Wolfram syndrome

Not recruiting

Study on the Effects of Sodium Valproate for Hearing Preservation in Patients with Wolfram Syndrome

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Sodium Valproate
  • Sponsor:Centre D'Etude Des Cellules Souches

Cystic fibrosis

Not recruiting

Study on the Safety and Tolerability of Elexacaftor, Tezacaftor, and Ivacaftor in Children with Cystic Fibrosis Aged 12 to Less Than 24 Months

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Substances:Elexacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Muscular dystrophy

Not recruiting

Study on the Effects of Ribitol (BBP-418) in Patients with Limb Girdle Muscular Dystrophy Type 2I

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Substances:Ribitol
  • Sponsor:ML Bio Solutions Inc.

Cystic fibrosis

Not recruiting

Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis

Registered drug
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Anakinra
  • Sponsor:Universitaetsklinikum Heidelberg AöR

Adrenoleukodystrophy

Not recruiting

Study on the Effects of Dimethyl Fumarate for Patients with Adrenomyeloneuropathy

Registered drug
Countries:SpainSpain
  • Participants:18–64 years
  • Substances:Dimethyl Fumarate
  • Sponsor:Fundacio Institut D'Investigacio Biomedica De Bellvitge IDIBELL

Bulbospinal muscular atrophy congenital

Not recruiting

Study of NIDO-361 for Patients with Spinal and Bulbar Muscular Atrophy (SBMA)

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:NIDO-361
  • Sponsor:Nido Biosciences Inc.

Haemophilia B with anti factor IX+1

Not recruiting

Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors

InvestigationalNo placebo
Countries:CroatiaCroatia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Concizumab
  • Sponsor:Novo Nordisk A/S

Phenylketonuria

Not recruiting

Study on the Long-Term Safety of PTC923 (Sepiapterin) for Patients with Phenylketonuria

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:(S)-2-Amino-6-(2-Hydroxypropanoyl)-7,8-Dihydropteridin-4(3H)-One
  • Sponsor:PTC Therapeutics Inc.

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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