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Congenital, familial and genetic disorders – page 48

  • 796 clinical trials
  • 308 recruiting
  • 374 conditions
  • 27 countries

Conditions in this area

374 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Spinal muscular atrophy17Hypertrophic cardiomyopathy16Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Sickle cell anaemia13Congenital cystic kidney disease12Huntington's disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Fabry's disease10Hereditary angioedema with C1 esterase inhibitor deficiency10+350

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

Type of trial
Recruitment status
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796 clinical trials

Mucopolysaccharidosis II

Not recruiting

Study on the Effectiveness and Safety of DNL310 vs Idursulfase for Children and Young Adults with Mucopolysaccharidosis Type II

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Idursulfase
  • Sponsor:Denali Therapeutics Inc.

Friedreich's ataxia

Not recruiting

A Phase 2 Study to Evaluate Safety and Effectiveness of Omaveloxolone (RTA 408) in Patients with Friedreich's Ataxia

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years
  • Substances:Omaveloxolone
  • Sponsor:Reata Pharmaceuticals Inc.

Hereditary angioedema with C1 esterase inhibitor deficiency+1

Not recruiting

Study on the Long-Term Safety of STAR-0215 for Adults with Hereditary Angioedema

InvestigationalNo placebo
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:NAVENIBART
  • Sponsor:Astria Therapeutics Inc.

Neurofibromatosis

Not recruiting

Study on the Effect of Food on Selumetinib in Adolescents with Neurofibromatosis Type 1 Plexiform Neurofibromas

Registered drugNo placebo
Countries:PolandPoland
  • Participants:0–17 years · 18–64 years
  • Substances:Selumetinib
  • Sponsor:AstraZeneca AB

Tuberous sclerosis complex

Not recruiting

Long-Term Safety Study of Everolimus for Patients with Tuberous Sclerosis Complex and Refractory Seizures

Registered drugNo placebo
Countries:PolandPoland
  • Participants:0–17 years · 18–64 years
  • Substances:Everolimus
  • Sponsor:Novartis Pharma AG

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.

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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