Therapeutic area

Congenital, familial and genetic disorders – page 57

796 clinical trials
308 recruiting
374 conditions
27 countries

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

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796 clinical trials

Cardiac amyloidosis+1

Not recruiting

Registered drugNo placebo

Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

Registered drugNo placebo

Belgium

Participants:18–64 years · 65+ years
Substances:Patisiran
Sponsor:Alnylam Pharmaceuticals Inc.

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Factor VIII deficiency

Not recruiting

InvestigationalNo placebo

Study on the Safety and Effectiveness of Giroctocogene Fitelparvovec for Adult Men with Moderate to Severe Hemophilia A

InvestigationalNo placebo

France

Participants:18–64 years
Substances:Giroctocogene Fitelparvovec
Sponsor:Pfizer Inc.

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Cystic fibrosis lung

Not recruiting

InvestigationalNo placebo

Long-Term Safety Study of BI 3720931 for Patients with Cystic Fibrosis from a Previous Trial

InvestigationalNo placebo

France

Participants:18–64 years · 65+ years
Substances:Bi 3720931
Sponsor:Boehringer Ingelheim Espana S.A.

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Cystic fibrosis lung

Not recruiting

Registered drug

Study on the Safety and Effectiveness of BI 3720931 for Adults with Cystic Fibrosis Who Cannot Use CFTR Modulators

Registered drug

France

Participants:18–64 years · 65+ years
Substances:Bi 3720931
Sponsor:Boehringer Ingelheim Espana S.A.

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Factor VIII deficiency

Not recruiting

Registered drugNo placebo

Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A

Registered drugNo placebo

Belgium

Participants:18–64 years · 65+ years
Substances:Valoctocogene Roxaparvovec
Sponsor:Biomarin Pharmaceutical Inc.

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Evaluating Higher Dose Nusinersen in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

Not recruiting

InvestigationalNo placebo

Evaluating Higher Dose Nusinersen in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

InvestigationalNo placebo

Germany

Participants:0–17 years · 18–64 years
Substances:Nusinersen
Sponsor:Biogen Idec Research Limited

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Fabry's disease

Not recruiting

Investigational

Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven't received treatment for 6 months

Investigational

Austria

Participants:0–17 years · 18–64 years · 65+ years
Substances:Venglustat
Sponsor:Sanofi-Aventis Recherche & Developpement

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Adrenoleukodystrophy

Not recruiting

InvestigationalNo placebo

Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant

InvestigationalNo placebo

France

Participants:0–17 years · 18–64 years
Substances:Leriglitazone
Sponsor:Minoryx Therapeutics S.L.

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Retinitis pigmentosa

Not recruiting

Registered drugNo placebo

Long-Term Safety and Efficacy Study of Botaretigene Sparoparvovec for Patients with X-Linked Retinitis Pigmentosa

Registered drugNo placebo

Belgium

Participants:0–17 years · 18–64 years
Substances:Cefazolin Sodium

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Cystic fibrosis

Not recruiting

Registered drugNo placebo

Study on Long-term Safety of VX-121, Tezacaftor, and Deutivacaftor in Patients with Cystic Fibrosis

Registered drugNo placebo

Austria

Participants:0–17 years · 18–64 years · 65+ years
Substances:Deutivacaftor
Sponsor:Vertex Pharmaceuticals Inc.

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Focal segmental glomerulosclerosis+1

Not recruiting

InvestigationalNo placebo

Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

InvestigationalNo placebo

Belgium

Participants:0–17 years · 18–64 years
Substances:R3R01
Sponsor:River 3 Renal Corp.

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Haemophilia A without inhibitors

Not recruiting

Registered drugNo placebo

Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab

Registered drugNo placebo

Germany

Participants:0–17 years · 18–64 years · 65+ years
Substances:Emicizumab
Sponsor:F. Hoffmann-La Roche AG

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Substances studied in this area

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

Sirolimus21 clinical trials Sodium Chloride21 clinical trials Prednisolone12 clinical trials Plerixafor11 clinical trials Busulfan10 clinical trials Ivacaftor9 clinical trials Mitapivat9 clinical trials Paracetamol8 clinical trials Tezacaftor8 clinical trials

Congenital, familial and genetic disorders – page 57

Conditions in this area

Clinical trials in Congenital, familial and genetic disorders

796 clinical trials

Cardiac amyloidosis+1

Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

Factor VIII deficiency

Study on the Safety and Effectiveness of Giroctocogene Fitelparvovec for Adult Men with Moderate to Severe Hemophilia A

Cystic fibrosis lung

Long-Term Safety Study of BI 3720931 for Patients with Cystic Fibrosis from a Previous Trial

Cystic fibrosis lung

Study on the Safety and Effectiveness of BI 3720931 for Adults with Cystic Fibrosis Who Cannot Use CFTR Modulators

Factor VIII deficiency

Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A

Evaluating Higher Dose Nusinersen in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

Evaluating Higher Dose Nusinersen in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

Fabry's disease

Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven't received treatment for 6 months

Adrenoleukodystrophy

Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant

Retinitis pigmentosa

Long-Term Safety and Efficacy Study of Botaretigene Sparoparvovec for Patients with X-Linked Retinitis Pigmentosa

Cystic fibrosis

Study on Long-term Safety of VX-121, Tezacaftor, and Deutivacaftor in Patients with Cystic Fibrosis

Focal segmental glomerulosclerosis+1

Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

Haemophilia A without inhibitors

Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab

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Substances studied in this area

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