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. 

 

Adult Thalassaemia studies currently open:

Transfusion dependant Thalassaemia: ENERGIZE-T (AGIOS 018) CCTU - Recruiting

Phase 3, Double-blind, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Mitapivat in Subjects with Alpha- or Beta-Thalassaemia

Key inclusion:

  • Aged 18 or over
  • β-thalassaemia with or without α-globin gene mutations, HbE/β-thalassaemia, or α-    thalassaemia/HbH disease
  • Hb ≤10.0 g/dL
  • 6 to 20 RBC units transfused and a ≤6-week transfusion-free period during the 24-week period before randomization
  • Hydroxycarbamide dose must be stable for ≥16 weeks

Key exclusion:

  • Homozygous or heterozygous HbS or HbC
  • Received luspatercept/ hematopoietic stimulating agents within 36 weeks of randomisation

Transfusion dependent Thalassaemia & Non-Transfusion dependent Thalassaemia: NIHR Bioresource CCTU - Recruiting

NIHR BioResource – Rare Diseases study project (Haemoglobinopathies)

Key inclusion:

  • Confirmed clinical diagnosis of thalassaemia

Key exclusion:

  • Thalassaemia trait

Non-Transfusion dependent Thalassaemia: ENERGIZE (AGIOS 017) CCTU – Recruiting

Phase 3, Double-blind, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Alpha- or Beta-Thalassaemia

Key inclusion:

  • Aged 18 or over
  • β-thalassaemia with or without α-globin gene mutations, HbE/β-thalassaemia, or α-thalassaemia/HbH disease
  • ≤5 RBC units during the 24-week period before randomization, and no RBC transfusions ≤8 weeks before informed consent
  • Hydroxycarbamide dose must be stable for ≥16 weeks

Key exclusion:

  • Homozygous or heterozygous HbS or HbC
  • Received luspatercept/ hematopoietic stimulating agents within 18 weeks of randomisation

Thalassaemia studies currently in set up:

Transfusion dependent Thalassaemia & Non-Transfusion dependent Thalassaemia: GLADIOLUS (FT-4202) CCTU - In set up

Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassaemia or Sickle Cell Disease

Key inclusion:

  • Age 12 to 65 years
  • Documented diagnosis of β-thalassaemia, Haemoglobin E/ β-thalassaemia or Haemoglobin H (α-thalassaemia)

Key exclusion:

  • For Transfusion dependent Thalassaemia patients cannot be taking Hydroxyurea, for Non-transfusion dependent Thalassaemia this is allowed.

Adult Sickle Cell Disease studies currently open:

Transfusion dependent Sickle Cell Disease & Non-Transfusion Sickle Cell Disease: NIHR Bioresource CCTU – Recruiting

NIHR BioResource – Rare Diseases study project (Haemoglobinopathies)

Key inclusion:

  • Confirmed clinical diagnosis of sickle cell disease

Key exclusion:

  • Sickle cell trait

Non-Transfusion Sickle Cell Disease: CSL889 haemopexin CRF – Recruiting

Safety and tolerability of CSL889 in Sickle Cell Disease:  A Phase 1, Multi-Center, Open Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CSL889 in Adult Patients with Stable Sickle Cell Disease

Key inclusion:

  • Diagnosis of SCD characterized by HbSS or SCD characterized by HbSβ0 confirmed based on clinical record of genetic, electrophoresis, or high-performance liquid chromatography testing
  • Aged 18 to 60 years, inclusive
  • Stable SCD for at least 30 days before Day 1
  • If taking hydroxyurea or L-glutamine, stable dose for 30 days

Non-Transfusion Sickle Cell Disease: CROSSWALK-a CCTU - Recruiting

A Phase IB Randomized, Placebo-Controlled Study Evaluating The Safety, Pharmacokinetics, Pharmacodynamics, And Efficacy Of Crovalimab For The Management Of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) In Patients With Sickle Cell Disease (SCD)

Key inclusion:

  • Age 12-55 inclusive
  • Confirmed diagnosis of HbSS or HbSβ0
  • Patients receiving therapeutic agents for sickle cell must be on a stable dose for ≥ 28 days

Key exclusion:

  • More than 10 VOEs within the last 12 months prior to presentation
  • Current or previous treatment with a complement inhibitor therapy
  • Investigational agent within 28 days of VOE presentation

Sickle Cell Disease studies currently in set up:

Transfusion dependent Sickle Cell Disease: GLADIOLUS (FT-4202) CCTU - In set up

Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassaemia or Sickle Cell Disease

Key inclusion:

  • Age 12 to 65 years
  • Documentation of SCD genotype (HbSS, HbSβ0-thalassaemia or other sickle cell syndrome variants)
  • Chronically RBC transfused

Key exclusion:

  • RBC exchange within the previous 3 months
  • Current use of other therapeutic agents for SCD

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

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 sequestrationNormal 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.