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Congenital, familial and genetic disorders – page 30

  • 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

Chronic granulomatous disease

Not yet recruiting

Safety and efficacy of autologous CD34+ cells transduced with a vector encoding human NCF1 in children and adults with p47‑deficient chronic granulomatous disease

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years

Cystic fibrosis

Not yet recruiting

Safety and Efficacy of VX-828, Deutivacaftor, and Tezacaftor in Adults with Cystic Fibrosis

Registered drug
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Sponsor:Vertex Pharmaceuticals Inc.

Sickle cell disease

Not yet recruiting

Methoxyflurane versus oral oxycodone hydrochloride and morphine sulfate for pain relief in sickle cell disease vaso‑occlusive crises

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Methoxyflurane 99.9%
  • Sponsor:Assistance Publique Hopitaux De Paris

Duchenne muscular dystrophy

Not yet recruiting

Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:SGT-003
  • Sponsor:Solid Biosciences Inc.

Type IIa hyperlipidaemia

Not yet recruiting

Atorvastatin, Placebo, and No Treatment in Adults with Familial Hypercholesterolaemia and Statin-Associated Muscle Symptoms

Registered drug
Countries:NorwayNorway
  • Participants:18–64 years · 65+ years
  • Substances:Atorvastatin
  • Sponsor:Oslo University Hospital HF

Primary immunodeficiency syndrome

Not yet recruiting

Autologous CD4+ T-Cell Gene Therapy for X-linked Hyper-IgM Syndrome Type 1 in Patients with X-linked Hyper-IgM Syndrome Type 1

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Diphtheria toxoid adsorbed on aluminium hydroxide, hydrated
  • Sponsor:Fondazione Telethon Ets

Congenital cystic kidney disease

Not yet recruiting

A study to evaluate the safety and tolerability of azathioprine in patients with autosomal dominant polycystic kidney and liver disease

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Azathioprine
  • Sponsor:UZ Leuven

Hereditary haemochromatosis

Not yet recruiting

A study to evaluate the effectiveness and safety of vamifeport in adults with hereditary hemochromatosis

Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Vamifeport trihydrochloride
  • Sponsor:CSL Behring LLC

Friedreich's ataxia

Not yet recruiting

A Phase 3 Study Evaluating the Efficacy and Safety of Subcutaneous Nomlabofusp in Adults and Children with Friedreich’s Ataxia

Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years

Sickle cell anaemia with crisis+1

Not yet recruiting

Study of the effectiveness of iloprost compared to a placebo for treating vaso-occlusive crises in adults with sickle cell disease

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Glucose Monohydrate
  • Sponsor:Centre Hospitalier Universitaire Rouen

Primary bilateral macronodular adrenal hyperplasia

Not yet recruiting

Study of the effect of metyrapone in patients with mild Cushing syndrome caused by primary bilateral macronodular adrenal hyperplasia

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Metyrapone
  • Sponsor:Assistance Publique Hopitaux De Paris

Hereditary optic atrophy

Not yet recruiting

A study to evaluate the effectiveness of nicotinamide in patients with Leber's hereditary optic neuropathy who have experienced vision loss within the last 18 months

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Nicotinamide
  • Sponsor:Centre Hospitalier Universitaire D'Angers

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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