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Fondazione Telethon Ets
Sponsor

Fondazione Telethon Ets

Italy Via Varese 16 B, 00185 Rome, Italy

About the sponsor

Research activity in pediatric hereditary bone disease includes autologous hematopoietic stem and progenitor cell approaches for osteopetrosis linked to TCIRG1 mutations, with attention to safety and clinical efficacy.

  • TCIRG1-associated autosomal recessive osteopetrosis
  • Ex vivo hematopoietic stem cell gene therapy
  • Pediatric bone marrow and skeletal disease

The sponsor’s work in this area centers on genetic correction strategies for severe inherited skeletal disease affecting children.

About Specializations

Fondazione Telethon Ets runs clinical trials in conditions such as Thalassaemia beta, Thalassaemia, Osteopetrosis, Wiskott-Aldrich syndrome, among others.

Recruiting now

Ongoing trials

Clinical trials run by this sponsor – those enrolling participants first.

Condition
Country
City
Type of trial
Recruitment status
Quick filters

5 clinical trials

Thalassaemia beta+1

Recruiting

A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Fondazione Telethon Ets

Osteopetrosis

Recruiting

Study of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Plerixafor
  • Sponsor:Fondazione Telethon Ets

Wiskott-Aldrich syndrome

Recruiting

Long-term Safety and Efficacy Study of Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Etuvetidigene Autotemcel
  • Sponsor:Fondazione Telethon Ets

Immunodeficiency

Recruiting

Study on the Use of OTL-103 Gene Therapy and Drug Combination for Patients with Wiskott-Aldrich Syndrome

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Fondazione Telethon Ets

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

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