Studies currently open:

For more information about these studies, please email our team.

Paediatric studies currently open: 

  • NIHR BioResource- Rare Diseases Cohort:- Sickle Cell Disorder and Thalassemia. Patients of all ages diagnosed with either disorder may be eligible. More information on the inclusion/exclusion criteria can be found at: web-summary-hbp-v1-2-18052022.pdf (nihr.ac.uk). 

  • GBT 440-038- An Open-Label Extension Study of Voxelotor administered orally to paediatric participants with Sickle Cell Disorder who have participated in Voxelotor Clinical Trials. 

  • Rare and Undiagnosed diseases Study (RUDY)- Using validated questionnaires and other information such as diagnostic history, clinical events, and treatments we hope to gather information on rare disorders. Patients of all ages with a rare disorder, are eligible to take part. View the list of rare disorders we are interested in. 

  • The Molecular Investigation of Unexplained Anaemias and Related Congenital Anaemias - Inclusion criteria- Patients of all ages diagnosed with persistent likely genetic anaemia (anaemia on more than one occasion) OR has a relative who is being investigated for persistent anaemia.  

  • CROSSWALK-a: A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disorder (SCD) ≥12 to ≤55 years – Patients experiencing Sickle crisis are eligible in certain circumstances. (Closed to screening) 

  • ROTHEM: An observational study to quantify and qualify micro vesicles and vasculopathy in patients with sickle cell disorder in the in- and out-patient setting. Patients with SCD of all ages may be eligible and patients with no health complications may be eligible to take part in the control arm of the study. 

  • SMILES: Study of Montelukast In ChiLdrEn with Sickle Cell Disease. A Double-Blind Randomised Controlled Trial (RCT) 

  

Studies in set-up:   

  • GBT021601-021: A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease 

  • GBT021601-022: An Open-label Extension Study to Evaluate the Long-term Safety of Osivelotor Administered to Participants with Sickle Cell Disease Who Have Participated in an Osivelotor Clinical Trial 

  • PRECISE PASS: Safety and efficacy registry (real world) study of liquid hydroxycarbamide (Xromi®) for children under the age of 2 years with Sickle Cell Disease.  

We also support these adult studies: 

Open: 

  • Sickle Eye Project: an epidemiological, cross-sectional, non-interventional study to determine the prevalence of visual impairment due to sickle cell retinopathy and/or maculopathy in the United Kingdom. 

Closed: 

  • CROSSWALK-c: A randomised double blinded-blind phase IIA study evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab as adjunct treatment in prevention of vaso-occlusive episode (VOE) in sickle cell disorder (SCD)- Patients with Sickle Cell Age ≥12 to ≤55 years may be eligible if meets inclusion/exclusion criteria. 

 

GBT021601-021: A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease.

A new clinical trial is underway to evaluate osivelotor, a potential disease-modifying treatment for sickle cell disease (SCD), focusing on reducing anemia, hemolysis, and organ damage. This study will assess the drug's safety, efficacy, and pharmacological effects, such as increasing red blood cells and hemoglobin levels, in adults aged 18–65 with specific SCD genotypes. Participants must meet strict health and treatment criteria and avoid certain medications or substances. Exclusion factors include recent blood transfusions, severe infections, or significant organ impairments. If successful, osivelotor could represent a significant advancement in SCD management. 

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. 

Key inclusion: 

  • Age ≥ 18 years  
  • Confirmed haploidentical donor 
  • Severe SCD phenotype 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  
    • History of ≥3 severe pain crises per year in a 2-year period preceding enrolment  
    • Administration of regular transfusion therapy  
    • 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  

   Key exclusion: 

  • Fully matched sibling donor 

The Sickle Eye Project: Prevalence of visual impairment due to sickle cell retinopathy and maculopathy in the United Kingdom - The Sickle Eye Project is looking for anyone with Sickle Cell Disease, to help us improve their eye care and vision-related quality of life. The study is open to all Sickle Cell patients regardless if they have been experiencing issues with vision or not. Participation involves completing two brief questionnaires and having eye tests and scans that are quick, painless and routine in the NHS. 

Thalassaemia studies currently IN SET-UP: 

R7999-BTHAL-2350 (FERVENT-1) The FERVENT-1 study is a phase 2 clinical trial evaluating the safety, efficacy, and tolerability of REGN7999, a monoclonal antibody targeting iron overload in adults with non-transfusion-dependent β-thalassemia (NTDT). REGN7999 works by increasing hepcidin levels, reducing excess iron in the bloodstream, and offering a potential treatment for this condition. Participants must have confirmed NTDT with iron overload and meet specific health criteria, while exclusions include recent blood transfusions, severe liver conditions, or recent use of other iron-reducing therapies. If effective, REGN7999 could become an important option for managing iron-related complications in NTDT. 

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. Open for Sickle Cell and Thalassaemia patients. 

For more information about these studies, please email our team.

Studies currently open at Hammersmith Hospital

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:  

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.  

Hibiscus: 

An Adaptive, Randomized, Placebo-controlled, Double blind, Multi-centre study of Etavopivat (FT- 4202 – a Pyruvate Kinase Activator) in Patients with all sickle cell genotypes. The study has a 52-week double blind period, with a 2:1 randomisation (Etavopivat vs Placebo).  

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. 

Sickle Eye Project: 

The Sickle Eye Project is looking for anyone with Sickle Cell Disease, to help us improve his or her eye care and vision-related quality of life. The study is open to all Sickle Cell patients regardless if they have been experiencing issues with vision or not. Participation involves completing two brief questionnaires and having eye tests and scans that are quick, painless and routine in the NHS. 

For more information about any of these studies, please email our team. 

Paediatric Studies currently open: 

Hibiscus Etavopivat PK Activator Study key inclusion criteria 

  • Sickle cell disease: any genotype 
  • 12 to 18 years of age. 
  • At least 2 episodes of VOC in the past 12 months but no more than 10: 
    1. ACS 
    2. acute painful crisis with documentation of opiate use (A+E attendance or hospitalization). 
  • Hb 55 g/L to 105 g/L 
  • Hydroxycarbamide permitted but on stable dose 90 days (no more than 20% change in dose). 

Gene Editing Vertex 141 Study key inclusion criteria 

  • Sickle cell disease: HbSS or HbSb0 or HbSb+ 
  • 5 to 11 years of age. 
  • No HLA matched sibling donor. 
  • At least 2 episodes of VOC per year for 2 years: 
    1. ACS 
    2. VOC requiring visit to medical facility for opiates or red cell transfusion 
    3. Priapism >2 hours 
    4. Splenic sequestration Normal TCD. 
  • Normal TCD. 
  • Hydroxycarbamide failure or intolerance. 

PNH Pegcetacoplan Study key inclusion criteria 

  • 12 to 17 years of age 
  • PNH: granulocyte or monocyte clone >10%. 
  • Evidence of haemolysis: 
    1. naïve patient: Hb 1.5 × ULN. 
    2. switch patient: Hb ULN despite eculizumab. 
  • Platelet count >75 x 109/L. 
  • Neutrophils >1 x 109/L. 
  • Weigh >20 kg. 
  • BMI <95th percentile for their age. 

For more information about these studies or to make referrals, please email Kelly Hennessy.

The Sickle Eye Project: Prevalence of visual impairment due to sickle cell retinopathy and maculopathy in the United Kingdom - The Sickle Eye Project is looking for anyone with Sickle Cell Disease, to help us improve their eye care and vision-related quality of life. The study is open to all Sickle Cell patients regardless if they have been experiencing issues with vision or not. Participation involves completing two brief questionnaires and having eye tests and scans that are quick, painless and routine in the NHS.

RISE UP (AG348-C-020) – Recruiting

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disorder

Key Inclusion:

  • Aged 16 or over
  • Documented diagnosis of sickle cell (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants)
  • At least 2 but no more than 10 sickle cell pain crises in the 12 months prior to consent
  • Haemoglobin ≥5.5 and ≤10.5 g/dL
  • Hydroxyurea permitted but must be on stable dose for 90 days prior to randomisation

Key Exclusion:

  • Pregnant or breastfeeding
  • Regular RBC exchange or episodic RBC exchange within 60 days of consent
  • Current use of Voxelotor, Crizanlizumab or L-glutamine (eligible if last dose was more than 90 days prior to randomisation)

For more information, please email our team.