Airtable - Red Cell Disorders

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AHP

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ELEVATE AHP Registry

Ascelus-H

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

Sickle Cell Lines

Safety and tolerability of CSL889 in Sickle Cell Disease Patients

SCD UK Natural History Study

REDRESS - Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease

HOPE - A study to Evaluate the Effect of GBT440 in Pediatrics with Sickle Cell

FORMA 4204-HEM-301 (PRAISE Study)

RESPECT

Improving Black Health Outcomes (IBHO)

Treatment stage

Multiple stages

Subtype

Haemochromatosis

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Treatment stage

Multiple stages

Subtype

SCD

Count8

Treatment stage

Multiple stages

Subtype

SCD

Thalassemia

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Treatment stage

Multiple stages

Summary

For adults with AHP

Research database: longitudinal prospective registry of patients with acute hepatic porphyria

Not specified

Rees, D

ELEVATE, a global observational longitudinal prospective registry of patients with acute hepatic porphyria (AHP)

Documented diagnosis of AHP, per physician’s determination

For adults with stable CLL, Haemochromatosis, irron-deficient anaemia, MDS, MGUS, Smouldering myeloma, SCD, or Waldenstrom’s macroglobulinemia

Digital follow-up pathway monitoring long-term stable blood conditions

None

Benjamin, R.

This study has 5 arms: Arm 1 - Patient and carer co-design,collecting qualitative data on patient satisfaction and empowerment on follow-up pathways,via focus groups and individual interviews Arm 2 – Healthcare professionals co-design and feedback,collecting qualitative data on Healthcare professionals views and feedback of the digital follow-up pathways,via focus groups and interviews Arm 3 – Key Opinion Leader (KOL) interviews,investigating issues of implementation,scalability,and commercialis

Inclusion for Arm 1:

For adults and adolescents with SCD

Phase 2/3 study: Etavopivat vs placebo to improve Hb count and reduce vaso-occlusive crises

No specified

Stuart-Smith, S

Gardner, K

Etavopivat is designed to activate PKR and thereby modulate RBC metabolism by impacting two critical pathways in RBCs. The etavopivat clinical development program will investigate whether decreasing 2,3-DPG may help oxygen bind to hemoglobin (i.e. increasing oxygen affinity), and thereby increase ATP and impact RBC function. This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients age 12 to 65 years (inclusive), with sickle cell disease. There are two

Provision of consent

For healthy patients between 10 and 40 years with SCD

Biobanking: stem cell collection

Not specified

Rees, D

Inherited hemoglobin disorders, such as sickle cell dieses (SCD) have been declared as a world health problem by the World Health Organisation in 2008. Specifically, 400,000 children are born annually with SCD. Hemoglobin disorders affect the life span and properties of red blood cells in the circulation of patients, thus leading to several complications ranging from severe anemia and painful vas-occlusive crisis to organ failure. To dat, there is no cure but only treatments to ameliorate the pa

Sickle Cell Disease including HbSS, HbSC, HbS/beta thalassaemia, HbSOman, HbSDpunjab genotypes.

For adults with stable SCD

Phase 1 single dose study: CSL889 for SCD

Not specified

Awogbade, M

Kesse-Adu, R

The study drug CSL889 is being developed for the treatment of vaso-occlusive crisis (also known as pain crisis) that occurs in patients with sickle cell disease. All humans have hemopexin. However, patients with sickle cell disease may have very little hemopexin. The main job of hemopexin is to combine with a substance in blood called haem and to clear haem from the blood. The haem comes from red blood cells when red blood cells die. Research suggests that haem may play a role in pain crisis. CS

Capable of providing written informed consent and willing and able to adhere to all protocol requirements.

For adults with SCD

Observational study: progression over a 5 year period

Not specified

Stuart-Smith, S

Gardner, K

This observational study will look at how patients progress over a five year period. Detailed medical history retrospective data will be collected on study enrollment and then at routine annual review appointments. Data from investigations will be collected from the electronic patient record (including laboratory results and imaging reports). Patients will also complete a health-related quality of life questionnaire every year.

None listed

For transplant-eligible adults with severe SCD

Randomised trial: related haplo-donor HSCT vs standard of care

Not specified

Potter, V

Kesse-Adu, R

A open label, non-randomized, first-in-patient, phase I/II, proof-of-concept study following subjects for two years after transplantation of either genome-edited autologous HSPC investigational drug product. The study consist of 3 parts - Part A include treatment of adults with OTQ923; Part B include treatment of adults with HIX763; Part C include treatment of kids 2-17 years old with either OTQ923 or HIX763

Adult patients age > = 18 years

For children with SCD who have experienced at least one vaso-occulisive crisis in the last year

Randomised study: GBT440 vs placebo

Not specified

Babiker, S

Sickle Cell Disease (SCD) is a serious life-long illness that causes low red blood cell counts (anaemia), fatigue, and damage to blood vessels leading to problems such as acute painful episodes, strokes and leg ulcers. It can also result in organ damage and lead to premature death. The main purpose of this research study is to determine the effect of the study drug GBT440, compared to placebo in increasing red blood cell counts and reducing the severity of symptoms of SCD. A placebo is a “dummy

Male or female study participants with sickle cell disease

For adults with SCD

Randomized study: oral FT-4202, a Pyruvate Kinase Activator for SCD

Not specified

Stuart-Smith, S

FT-4202 is a potent, selective, orally bioavailable, small-molecule activator of pyruvate kinase-red blood cell (PKR) being developed by Forma Therapeutics, Inc and is intended for use as a treatment for patients with sickle cell disease (SCD). The clinical hypothesis is that PKR activation will reduce the rate of sickle cell polymerization and improve red blood cell (RBC) membrane function, thereby reducing RBC sickling and RBC hemolysis that lead to vascular obstruction and anemia, two hallmar

None listed

For adults undergoing HSCT

Phase 3 randomised trial: Rezafungin vs standard treatment to prevent IFI

Not specified

Mehra, V

The ReSPECT trial is a global, randomized, double-blind, controlled, pivotal Phase 3 trial of rezafungin versus the standard antimicrobial regimen to prevent invasive fungal disease due to Candida, Aspergillus and Pneumocystis in subjects undergoing allogeneic BMT. Rezafungin, dosed once-weekly, will be compared to a daily regimen containing multiple drugs including fluconazole or posaconazole, and trimethoprim-sulfamethoxazole, also known as Bactrim, for 90 days, at which time fungal-free survi

≥18 years of age

For patients who identify as Black or having a Black ancestry, and / or who have SCD or Thalassemia

Observational study / BioResource

None specified

Brewin, J

The aim of the IBHO BioResource is to research the social and biological causes,and impacts,of a broad range of health conditions and illnesses in people from Black communities in the United Kingdom (UK). These include disorders that affect people from Black communities at increased rates,such as diabetes and pregnancy/childbirth complications,but also conditions that almost exclusively affect those from Black communities such as sickle cell disease. The IBHO Bioresource is also designed to exam

People who identify as Black or having a Black ancestry,including: Black African,Black Caribbean,Black African-Caribbean,Black British,Black Welsh,Black Irish,Black Scottish,Dual or Mixed Black heritage,and/or Black ‘other’.

Summary

11 records

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