Study on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies

Registered drugNo placebo

Study on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies

Registered drugNo placebo

Spain

Participants:0–17 years
Substances:Imlifidase
Sponsor:Sarepta Therapeutics Inc.

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Cystic fibrosis

Not recruiting

Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor in Children and Adults with Cystic Fibrosis Aged 2 Years and Older

Registered drugNo placebo

Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor in Children and Adults with Cystic Fibrosis Aged 2 Years and Older

Registered drugNo placebo

Germany

Participants:0–17 years
Substances:Elexacaftor
Sponsor:Vertex Pharmaceuticals Inc.

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Osteogenesis imperfecta

Not recruiting

Study on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth

InvestigationalNo placebo

Study on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth

InvestigationalNo placebo

The Netherlands

Participants:0–17 years · 18–64 years
Substances:Allogeneic Fetal Mesenchymal Stem Cells
Sponsor:Karolinska Institutet

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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 43

Conditions in this area

Clinical trials in Congenital, familial and genetic disorders

803 clinical trials

DNA mismatch repair protein gene mutation+7

Study on Atezolizumab for Patients with High-Risk Stage II or Stage III Colorectal Cancer Not Eligible for Oxaliplatin Chemotherapy

K-ras gene mutation+2

Study Comparing Divarasib, Sotorasib, and Adagrasib for Patients with Advanced or Metastatic KRAS G12C-Positive Non-Small Cell Lung Cancer

Alport's syndrome

Study on the Safety of Vonafexor for Patients with Alport Syndrome at Risk of Progression

Pyruvate kinase deficiency anaemia

Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

Microvillous inclusion disease

Study on the Safety and Effectiveness of Crofelemer for Children with Microvillus Inclusion Disease (MVID)

Lymphatic malformation

Study on the Effects of Topical Sirolimus for Lingual Microcystic Lymphatic Malformations in Children and Adults

Hereditary angioedema with C1 esterase inhibitor deficiency

Study on the Safety and Effects of Sebetralstat for Children Aged 2-11 with Hereditary Angioedema Type I or II

Spinal muscular atrophy

Study on the Safety and Efficacy of RO7204239 and Risdiplam for Patients with Spinal Muscular Atrophy

Sickle cell anaemia

Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells

Duchenne muscular dystrophy gene carrier

Study on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies

Cystic fibrosis

Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor in Children and Adults with Cystic Fibrosis Aged 2 Years and Older

Osteogenesis imperfecta

Study on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth

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Substances studied in this area

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