Clinical trials listings

Red Cell Studies

Sickle Cell Disease - Thalassemia - Pyruvate Kinase Deficiency 

• Above 18 years old.
• Confirmed Haploidentical donor.

Severe SCD phenotype who are at high risk for morbidity and mortality. Severe SCD is defined by at least one of the following:

• Clinically significant neurologic event (stroke) or deficit lasting > 24 hours.
• History of ≥2 acute chest syndromes in a 2-year period preceding enrolment despite optimum treatment, e.g. with HU
• History of ≥3 severe pain crises per year in a 2-year period preceding enrolment despite the institution of supportive care measures (e.g. optimum treatment with HC).
• Administration of regular transfusion therapy (=8 packed red blood transfusions per year for 1 year to prevent VOCs).
• Patients assessed as requiring transfusion but with red cell allo-antibodies/very rare blood type, rendering it difficult to continue/commence chronic transfusion.
• Patients requiring HC/transfusion for treatment of SCD complications who cannot tolerate either therapy due to significant adverse reactions.
• Established end organ damage relating to SCD, including but not limited to progressive sickle vasculopathy and hepatopathy.

• Fully matched sibling donor.
• Previous bone marrow transplant. 
  Clinically significant donor specific HLA antibodies. 
  Active blood borne viruses. 
  Uncontrolled bacterial, fungal or viral infection.
  Pre-existing condition deemed to significantly increase the risk of haploidentical SCT by the local PI. 

Principal Investigator: Dr Steven Okoli
Study Coordinator: Charlotte Lyon
Charlotte.lyon6@nhs.net
Recruitment target: 2
Recruitment Closure Date: TBC

FERVENT-1:

IMP: one subcutaneous injection every 4 weeks. 

• 18-60 years old
• Clinical diagnosis of NTDT (history of serum ferritin >12 ng/mL, haemoglobin electrophoresis indicative of HbA2
• ≥3.5% and HbF >5%, and/or genetic evidence of a disease-conferring B-globin mutation.
• Iron overloading, defined as LIC ≥5 mg Fe/g DW as measured by R2* MRI at screening, or Serum ferritin ≥300 ng/mL

Primary exclusion criteria: 

• Haemoglobin ≤8 g/dL
• Any RBC transfusion within 12 weeks of visit 3.
• Greater than 3 RBC transfusions over approximately 6 months prior to screening visit 2
• For Part A only: Any ICT use in approximately 12 weeks prior to screening visit 2 or any intention to initiate or resume ICT
• For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening visit 2, or intention to change dose in following 6 months
• Previous Splenectomy

Hibiscus 2:

Primary inclusion criteria:

• Age 12 or above.
• Diagnosis of HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants.
• 2 -15 episodes of documented VOC in the past 12 months prior to screening.
• Haemoglobin ≥ 50 and ≤ 100 g/L.
• Dose of HC (mg/kg) must be stable for at least 90 days prior to start of study treatment .

• Receiving regularly scheduled RBC transfusion therapy, or 6 or more transfusion events in the past 6 months.
• Received RBC transfusion within any reason within 90 days prior to screening.
• DVT requiring systemic anti-coagulation therapy for ≥ 6 weeks, within 6 months prior to randomisation.
• History of overt clinical stroke within previous 2 years or any history of an intracranial haemorrhage
• Severe hepatic and/or renal dysfunction.
• Iron, folate or B12 deficiency. 

Sickle Eye Project: 

The prevalence of visual impairment due to SCR or maculopathy in a representative sample of the UK population with Sickle Cell Disease.

Inclusion criteria:

• Willingness to participate 
• Ability to provide informed consent 
• Age 16 years or older 
• Diagnosis of SCD of any genotype 

Exclusion criteria:

• Inability to consent 
• Poor image quality 
• Age <16 years 
• Sickle cell trait only 

Principal Investigator: Dr Steven Okoli
Study Coordinator: Sophie Newman sophie.newman20@nhs.net 
Recruitment Target: 35

Improving Black Health Outcomes (IBHO) NIHR BioResources

Main Inclusion Criteria: 

• Patients with a confirmed clinical diagnosis of sickle cell disease
• Principal Investigator: Dr Steven Okoli
• Study Coordinator: Spencer Mackie

RUDY: Rare and undiagnosed diseases study. 

Main Inclusion Criteria: 
* Age range 0 – 100 years
* Diagnosed with a rare disorder as defined by a prevalence of less than 5:10,000
* Resident within the United Kingdom

Main Exclusion Criteria:

• Children under 11 will not be invited to join any sub-studies

Principal Investigator: Dr Jeremy Anderson
Study Coordinator: Cynthia and Spencer

REDRESS:

REDRESS

A multi-centre open randomised controlled trial to assess the effect of related haplo-donor haematopoietic stem cell transplantation versus standard of care (no transplant) on treatment failure at 24 month in adults with severe sickle cell disease.

FERVENT-1:

FERVENT-1:
A phase 2, two-part, randomized, double blind, placebo controlled, multicenter study to evaluate the efficacy, safety, and tolerability of subcutaneously administrated REGN7999 (TMPRSS6 inhibitor) in Participants with iron overload due to non-transfusion dependent Beta Thalassemia.

Hibiscus2:

Hibiscus2:

A global phase 3, randomised, double blind and placebo-controlled study evaluating the efficacy and safety of Etavopivat (FT- 4202 – a Pyruvate Kinase Activator) in adults with sickle cell disease. The study has a 52-week double blind period, with a 2:1 randomisation (Etavopivat vs Placebo).

Improving Black Health Outcomes (IBHO) NIHR BioResource:

Improving Black Health Outcomes (IBHO) NIHR BioResource:

This new research initiative is dedicated to studying health conditions that disproportionately affect people from Black communities in the UK, such as sickle cell disease and thalassemia. This observational study invites individuals from Black ethnic backgrounds to participate in research aimed at improving our understanding of how these conditions might develop and specifically affect those from Black communities.