Rare disease

Thalassaemia

11 clinical trials
6 recruiting
11 countries

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Clinical trials for Thalassaemia

11 trials across Europe

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11 Clinical trials

Thalassaemia beta+1

Recruiting

Registered drugNo placebo

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

Registered drugNo placebo

Italy

Participants:0–17 years · 18–64 years
Substances:Busulfan
Sponsor:Fondazione Telethon Ets

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Thalassaemia

Recruiting

Registered drugNo placebo

Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Registered drugNo placebo

Germany

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

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Sickle cell disease+1

Recruiting

InvestigationalNo placebo

Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment

InvestigationalNo placebo

France

Participants:0–17 years · 18–64 years · 65+ years
Substances:Etavopivat
Sponsor:Novo Nordisk A/S

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Anaemia+4

Recruiting

Registered drugNo placebo

Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

Registered drugNo placebo

Bulgaria

Participants:18–64 years · 65+ years
Substances:Luspatercept
Sponsor:Celgene Corp.

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Sickle cell anaemia+1

Recruiting

Registered drugNo placebo

Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia

Registered drugNo placebo

Belgium

Participants:0–17 years · 18–64 years
Substances:Exagamglogene Autotemcel
Sponsor:Vertex Pharmaceuticals Inc.

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Thalassaemia beta+1

Recruiting

Registered drugNo placebo

Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia

Registered drugNo placebo

The Netherlands

Participants:18–64 years · 65+ years
Substances:Luspatercept
Sponsor:Amsterdam UMC Stichting

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Thalassaemia

Clinical trials for Thalassaemia

Thalassaemia beta+1

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

Thalassaemia

Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Sickle cell disease+1

Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment

Anaemia+4

Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

Sickle cell anaemia+1

Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia

Thalassaemia beta+1

Study on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia

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