Clinical trials for Thalassaemia in the UK

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The list below provides information and links to a variety of clinical trials for Thalassaemia in the UK.

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Drug: AG-348
Age: Adult Participants with Non-transfusion-dependent Thalassemia
Primary Purpose: Treatment

Trial Phase: 2

Official Title:   A Phase 2, Study to determine the efficacy, safety, pharmacokinetics, and pharmacodynamics of AG-348 in adult subjects with non-transfusion-dependent thalassemia.

Description:

This study is to evaluate the efficacy, and safety of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT). Mitapivat (AG-348) is an oral, small-molecule, allosteric activator of the RBC-specific form of pyruvate kinase (PK-R). PK-R is a key enzyme for maintaining energy homeostasis in RBCs, as they rely almost exclusively on the process of glycolysis to generate ATP. In healthy adults, mitapivat activates wild-type PK-R and increases ATP levels in RBCs.

For more details click below:

https://clinicaltrials.gov/ct2/show/NCT03692052?cond=thalassaemia&map_cntry=GB&draw=2&rank=1

Drug: IMR-687
Age: 18 Years to 65 Years   (Adult, Older Adult)
Primary Purpose: Treatment

Trial Phase: 2

Official Title: A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia

Description:

This study is to evaluate the efficacy, safety and Tolerability of treatment with IMR-687 in adult participants with transfusion dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT).

For more details click below:

https://clinicaltrials.gov/ct2/show/NCT04411082?cond=thalassaemia&map_cntry=GB&draw=2&rank=3

Drug: SLN124
Age: 18 Years and older   (Adult, Older Adult)
Primary Purpose: Treatment

Trial Phase: 1

Official Title: A Phase 1 Study to Evaluate the Safety and Tolerability of SLN124 in Subjects with alpha or beta thalassemia

Description:

This study is to evaluate the efficacy, safety and Tolerability of treatment with SLN124 in adult participants with alpha or beta non-transfusion-dependent thalassemia (NTDT).

For more details click below:

https://clinicaltrials.gov/ct2/show/NCT04718844?cond=thalassaemia&map_cntry=GB&draw=2&rank=10

Drug: CTX001
Age: 12 Years to 35 Years   (Child, Adult)
Primary Purpose: Treatment

Trial Phase: 3

Official Title: A Phase 3 Study to Evaluate the Safety and Tolerability of CTX001 in Subjects with beta thalassemia

Description:

The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

For more details click below:

https://clinicaltrials.gov/ct2/show/NCT03655678?cond=thalassaemia&map_cntry=GB&draw=3&rank=12

Drug: LentiGlobin BB305
Age: up to 50 Years   (Child, Adult)
Primary Purpose: Treatment

Trial Phase: 3

Official Title: A Phase 3 Study to Evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 drug product in subjects with transfusion-dependent β-thalassemia (TDT)

Description:

It is delivered as a single dose straight into the blood following a course of chemotherapy. If LentiGlobin BB305 is licenced for use in the UK, it could be the first treatment option for beta-thalassaemia major that has the potential to add a working copy of the beta-globin gene to patients’ stem cells. It may improve survival and quality of life by reducing or eliminating the need for blood transfusions and iron-chelation therapy.

For more details click below:

https://clinicaltrials.gov/ct2/show/NCT03207009?cond=thalassaemia&map_cntry=GB&draw=3&rank=14
https://clinicaltrials.gov/ct2/show/NCT02633943?cond=thalassaemia&map_cntry=GB&draw=3&rank=15

Drug: iHSCs treatment group
Age: 2 Years to 60 Years   (Child, Adult)
Primary Purpose: Treatment

Trial Phase: 1

Official Title: A safety and efficacy study of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9 that Interevent subjects with β-thalassemia mutations.

Description:

This study is to evaluate the efficacy and safety of treatment with SLN124 in adult participants with alpha or beta non-transfusion-dependent thalassemia (NTDT).

For more details click below:

https://clinicaltrials.gov/ct2/show/study/NCT03728322

Drug: ST-400
Age: 18 years to 40 years
Primary Purpose: Treatment

Trial Phase: 1/2

Official Title: A Phase 1/2, study to assess the safety, tolerability, and efficacy of ST-400 autologous hematopoietic stem cell transplant for treatment of transfusion-dependent beta-thalassemia (TDT).

Description:

ST-400 is composed of the patient’s own blood stem cells which are genetically modified in the laboratory using Sangamo’s zinc finger nuclease (ZFN) technology to disrupt a precise and specific sequence of the enhancer of the BCL11A gene (which normally suppresses fetal hemoglobin production in erythrocytes). This process is intended to boost fetal hemoglobin (HbF), which can substitute for reduced or absent adult (defective) hemoglobin. ST-400 is then infused back into the patient after receiving conditioning chemotherapy to make room for the new cells in the bone marrow, with the aim of producing new erythrocytes with increased amounts of HbF. The primary objective is to understand safety and tolerability of ST-400, and secondary objectives are to assess the effects on HbF levels and transfusion requirements.

For more details click below:

https://clinicaltrials.ucsf.edu/trial/NCT03432364