We have now been informed that preliminary findings from the ongoing worldwide clinical study called STAND (NCT038147460) indicates no statistically significant difference between Crizanlizumab 5mg/kg Crizanlizumab7.5mg/kg and placebo in making a difference to a sickle cell crisis. However, these findings are inconsistent with previous trial results from the SUSTAIN (NCT01895361) trial. As a result of this regulatory bodies such as the European Medicines Agency (EMA) will undertake a review of Crizanlizumab to evaluate the impact of these contrasting results on its currently authorised use.

We would like to reassure those who are taking Crizanlizumab , or who may be contemplating taking it, following discussions with their Health Care Professional, that it is safe. However, if you have any concerns, we recommend that you speak to your Consultant Haematologist in the first instance who will determine the best option for each patient depending on their individual situation.

We will provide more information when we have it.

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https://www.youtube.com/watch?v=6r72E2VMoH0

If you would like to take part, please go to the  project website and complete the participation form.

https://www.kcl.ac.uk/research/pericles

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CSL889 is a new potential treatment under development for the symptoms of SCD.

Flyer for the CSL889 Clinical Trial (Ethics Comity Approved)

Flyer for the CSL889 Clinical Trial (Ethics Comity Approved)

CSL889 is a plasma derived Hemopexin, an important protein produced naturally in the body whose levels are decreased in patients with SCD.

CSL889 will be developed for the treatment of acute vasoocclusive crisis, a severe and painful complication in SCD patients.
It is given via blood channels (veins) over a period of approx. 1 hour or less.

The Study

• This is a clinical research study involving a single administration of the study drug, CSL889
• This is the first time that the study drug is being given to humans.
• This study will run for approximately 54 days
• This involves 9 outpatient visits and a 2 night residential stay
• Eligible participants may receive compensation for their participation in the study, plus reasonable travel expenses.
• Approx. 24 participants will take part in the study

Currently Open Clinical Trial Locations:

• King’s College Hospital – Denmark Hill London SE5 9RS
• Guy’s & St Thomas Hospital – Great Maze Pond London SE1 9RT
• UCLH – 235 Euston Road London NW1 2BU
• Manchester Royal Infirmary – Oxford Road, Manchester M13 9WL. Contact Number: 0161 906 7510
• MAC’s Early Phase Unit – Nelson Street Manchester M13 9NQ
  • Free phone 0800 917 7637
  • Link to register for the trial: https://bit.ly/3wCNx8A

If you are interested in this trial, you can also speak to your treating physicians who can get in touch with the participating sites on your behalf.

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Pregnant women with Sickle Cell Disease (SCD) have an increased risk of both sickle and pregnancy complications, including raised blood pressure. Their babies may grow more slowly in the womb, are more likely to be born early, need special care and have a higher risk of dying. The only treatments currently available for women with SCD are Hydroxycarbamide (which cannot be used during pregnancy) and blood transfusion. Currently, blood transfusion is only used during pregnancy to treat emergency complications. It has been suggested that giving blood transfusions throughout pregnancy could improve outcomes for both mother and babies, but a trial is required to demonstrate this.

The TAPS2 study is a feasibility study which aims to recruit 50 women at six clinical trial centres (five in London and one in Manchester).   TAPS2 will look firstly at whether women choose to participate and then how acceptable women find taking part in the trial; additionally we will record the health statuses of women and babies who take part.  The results of this trial will be used to decide whether a larger study is indicated.

Women who choose to take part will be randomised to one of two groups; either serial prophylactic exchange blood transfusions (EBT) or to normal clinical care, whereby EBTs will be arranged when clinically indicated.  Some of the women will also be asked their about decision to participate in TAPS2 and what their experiences were from both groups.  TAPS2 will also be asking women who decline to participate about their thoughts and opinions when deciding to decline the trial.

To be eligible to take part in the trial, women need to meet the inclusion criteria and none of the exclusion criteria:

Inclusion criteria:

• Pregnant women with SCD (all types).  This must be confirmed by a laboratory report
• Singleton pregnancies
• 18 years or older
• Gestation less than 18 weeks
• Willing and able to give informed consent

Exclusion criteria:

•  Women on long term transfusion programme prior to pregnancy for the improvement of Sickle Cell Disease
• Prior Hyperhaemolysis
• Red cell phenotype or antibodies present prevent likely provision of adequate red cell units to support elective EBT programme
• Women unable to receive blood transfusion for social, clinical or religious reasons
• Pregnant women with sickle cell disease with current diagnosis of major medical or psychiatric complications which the clinicians working on the study believe is contraindicated for the TAPS2 trial.

You can find more information about the clinical trial, including how to contact the study coordinators, on clinicaltrials.gov under the study ID number NCT03975894 .

Clinical Trial Locations:

Guy’s and St Thomas Hospital Foundation Trust- London, United Kingdom

Contact: Professor Eugene Oteng-Ntim

Kings College Hospital NHS Trust – London, United Kingdom

Contact: Dr Jemma Johns/Dr Moji Awogbade

Royal London– London, United Kingdom

Contact: Dr. Paul Telfer/ Dr. Filipa Barroso

St George’s University Hospital NHS Foundation Trust– London, United Kingdom

Contact: Dr Ingrid Watt-Coote

St Mary’s Hospital- Manchester, United Kingdom

Contact: Dr Joseph Sharif

Whittington Hospital- London, United Kingdom

Contact: Dr Emma Drasar

For more information: e: Taps2@gstt.nhs.uk t: 020 7188 3634

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Imara is conducting a Phase 2a clinical trial, evaluating escalating doses of IMR-687 in adult patients with sickle cell disease (SCD). IMR-687 has the potential to reduce red blood cell sickling, red blood cell death and the blockage of blood vessels that are the result of having sickle cell disease.  This randomized, double-blind, multi-center Phase 2a study of IMR-687 will evaluate the safety of increasing doses of IMR-687 as compared to placebo in adult patients with sickle cell disease.  The study will also examine blood levels of IMR-687, as well as explore the effects that IMR-687 has on the various blood measurements.

Approximately 54 patients will be enrolled at several clinical trial centers in the United Kingdom and the United States. Patients will undergo 6 months of treatment with IMR-687 and Imara anticipates completion of the study in 2019.

Here are a few important details about the enrollment criteria:

• The study is open to adult patients age 18-50 years old.
• Both patients who are not or are taking hydroxyurea (HU; also known as hydroxycarbamide) can participate in this study. Patients who are taking hydroxyurea must have been on a stable dose for at least 60 days prior to entering the screening process.
• Patients must also have stable kidney and liver function.

You can find more information about the clinical trial, including how to contact the study coordinators, on clinicaltrials.gov under the study ID number NCT03401112.

Currently Open Clinical Trial Locations:

Birmingham and Sandwell
Birmingham, United Kingdom
Contact: Elizabeth Green

Croydon University Hospital
Croydon, United Kingdom, CR7 7YE
Contact: Linda Sawyer

Darent Valley Hospital
Dartford, United Kingdom, DA2 8DA

Whittington Hospital
London, United Kingdom, N19 5NF

University College Hospital (UCLH)
London, United Kingdom, NW1 2BU
Contact: Alyna Sheikh
Principal Investigator: Perla Elefjeriou

University Hospital Lewisham
London, United Kingdom, SE13 6LH

Kings College Hospital
London, United Kingdom, SE5 9RS

Guy’s and St Thomas Hospital
London, United Kingdom
Contact: Yemi Adelaja

Homerton Hospital
London, United Kingdom
Contact: Anna Price

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