Sunday, 25 February 2018

Current Research Trials, Scleroderma, Raynaud's, Autoimmune Rare Disease. Scleroderma Unit, The Royal Free Hospital, London

 Current Research Trials,

Scleroderma Unit, The Royal Free Hospital, London.
Scleroderma, Raynaud's, Autoimmune Rare Disease. 


A brief summary outlining the current situation regarding research trials for 
scleroderma patients at the Scleroderma Unit, The Royal Free Hospital, London. 



Drug Investigational trials:

At the Royal Free Hospital we participate in numerous clinical trials to improve the treatment options for patients with scleroderma.

ACT14604 – Efficacy and safety of SAR156597 in the treatment of dcSSc.
This trial is over a 24 week period, with the objective being to evaluate the efficacy of the drug SAR156597 in treating diffuse scleroderma when compared with placebo.

SENSCIS (Nintedanib) – An ongoing trial to investigate the efficacy of the trial drug nintedanib in reducing lung fibrosis and treating skin fibrosis. The trial team recruit patients with systemic sclerosis-associated interstitial lung disease. This is a randomised, placebo-controlled study.

PRedSS – This is a new study to identify the efficacy of Oral prednisolone in early diffuse cutaneous systemic sclerosis compared with placebo. The Royal Free is currently in the set-up phase of the study.

Observational Trials:
As well as drug clinical trials, the Royal Free recruits patients for research studies that gather information to further our understanding of this disease.

STRIKE – Stratification for RisK of progression in scleroderma
This is a research study run by the university of leeds involving patients with Raynaud’s and/or scleroderma. The study follows patients over a period of 5 years, at 6 monthly clinic visits, with questionnaires and blood serum analysis.  The aim is to create a tool that will inform clinicians of ways they can screen for patients at risk of developing scleroderma and ways to prevent or delay the onset of disease-related complications.

SPIN – The Scleroderma Patient-centred Intervention Network (SPIN) Cohort
This study is an ongoing, longitudinal scleroderma patient cohort to better understand problems faced by patients living with scleroderma. Once enrolled, patients complete online questionnaires relating to their health and wellbeing every 3 months. Information from this study is being used to develop further interventions for patients with scleroderma.  
Feb 2018. 

A Big Thank You to Rachel, Stephanie, and the clinical trials team for their ongoing dedication and commitment - sharing the same dream #SclerodermaFreeWorld. 
 
If you are interested in participating in any trial please contact the team on
02073177544 or email: rf.clinicaltrials@nhs.net
   

At last year’s annual Scleroderma Family Day, 2017 programme, Dr Anna Gill gave a presentation about current clinical trials taking place at the Scleroderma Unit. View here

A further update on clinical trial research can be seen in the Scleroderma Unit Christmas 2017 newsletter
 
This year’s Scleroderma Family Day is to be held on Saturday 19th May in the atrium at the Royal Free Hospital. To view the program, Click here

I am very much hoping to attend this year's family day, as I am celebrating my 20th year anniversary of being a patient at the Unit. This Scleroderma Olympian is already in training! 

I am eternally grateful to the superhumans Dame Prof Black, and Prof Denton, whose dedication and commitment to their patients is beyond outstanding. 

They helped me achieve my dream of becoming a barrister, albeit, my symptom management is my current full time job

These superhuman beings continue to be a huge source of inspiration to me. Seeing how happy Prof Denton becomes upon examining my symptom reversal, remains my elixir to keep going. 

For, the day to day reality of living with this diagnosis is no easy feat... Fact... However, best practice care, combined with a good attitude, are crucial in making this health challenge journey, less turbulent.  



In my view, clinical research is of huge importance and paramount to the rare disease patient. It provides immense hope to the rare disease patient. In that- improved medicines are being investigated, along with the cause and cure, being understood. 

Thereby, unlocking the rare disease enigma. #SclerodermaFreeWorld. 

The 'rare disease' label can often feel like a bleak and lonely existence. However, when combined together, the rare disease community has several commonalities: 


  • early diagnosis 
  • expert specialist centres 
  • access to innovative medicines  

Where  
RESEARCH is key 
to improving the entire rare disease landscape,
including, the rare disease patient's day to day reality. 



February 2018.

To read my articles: 


Rare Disease Day 2018, Research - Taking Part in Clinical Trials, Click here   

March Autoimmune Disease Awareness Month 2018, Click here   

Global patient video, Click here   

Becoming a Patient Research Ambassador for the NIHR, Click here  

If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here   


Scleroderma Awareness Month 2017 Patient Profiles Campaign, Click here     
 
Scleroderma Awareness Month 2017 Patient Profiles Campaign Patient Index, Click here        
The Pandora’s Box of the rare autoimmune disease Scleroderma, Raynaud's and Cancer, Click here


Key Event Dates 2018, Click here     

Importance of an early diagnosis, Click here     

Taking Part in Clinical Research Trials, Click here    


The Importance of a Multi Disciplinary Medical ‘Dream Team’, Click here 

Expert Specialist Centres, Click here      

My Skin is Cured from Scleroderma, Click here      

UK Guidelines for Managing and Treating Scleroderma, Click here      

Fatigue, Click here      

Mobility, Click here    

Diet and nutrition, Click here     


Raynaud's, Click here    



World Scleroderma Day 29th June 2016, Click here

World Scleroderma Day 29th June 2017, Click here   


Why Global Collaboration is important to the Rare Disease Patient, Click here      



RARE DISEASE DAY:
The theme to this years Rare Disease Day is Research

Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here     

2016 Rare Disease Day Patient Voice
2016 Rare Disease UK Parliamentary Reception 

2017 Rare Disease Day Flashback  

More unmet clinical needs  

Rare Disease Day 2016



Rare Disease Day is a fantastic opportunity for the entire rare disease community to shine a spotlight on their reality, combining as one unified voice. Where, at least one commonality presides –  
Medical Research provides the brightest light
for the illumination of the rare disease patients’ plight. 

I highlighted other areas of medical research interest within Week 3 of my Patient Profiles Campaign for Scleroderma Awareness Month 2017. 

VIDEO



Although rare disease patients are few in number, eg. 2.5 million scleroderma patients worldwide, (the World Scleroderma Foundation), the commonalities and golden hallmark for each rare disease patient are the same: 

Early Diagnosis
Expert Specialist Centres
Access to Innovative Medicines

Where MEDICAL RESEARCH investment is VITAL.

Research is the key. Abstracts from 2016 World Congress, Click here


Scleroderma Awareness Month Campaign 2016, Click here

Links

View video, here   

Preamble - here



The Family Day at the Scleroderma Unit, The Royal Free Hospital is taking place on the 19th May 2018. Browse the program, here

This year, I am celebrating 20 years of being a patient at this world leading expert specialist research centre. 

Sept 2017


I am eternally grateful to the global scleroderma trail blazers Dame Prof Black and Prof Chris Denton, whose commitment and dedication to unlocking the scleroderma enigma, is nothing other than, superhuman.
I am truly humbled and inspired by their work ethic. I am wholly appreciative for Prof Denton’s continued medical expertise and support, especially during my barrister qualifying years, 1997 - 2004. 

1st March 2004, I qualified as a self employed practising barrister. Further to having been told in 1997, by my diagnosing doctor, that I was looking at a 15month prognosis. 

I very much hope to utilise my professional skills and qualifications along with my patient experience, to help achieve the #SclerodermaFreeWorld dream, hoping to improve understanding and best practice, in the meantime.
  
For latest updates follow: 

Facebook Page:

Twitter: @SclerodermaRF  @RaynaudsRf  


#SclerodermaFreeWorld           #RaynaudsFreeWorld
#ADAM #Scleroderma
#Raynauds
#RareDisease  

Living the dream, scleroderma style.  


Please DONATE to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London.  



100% of your monies will be used for medical research purposes only. No wages or admin costs. Thank You.
#HOPE 


 

Last Update: April 2018





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