So you want to get into a clinical trial

The most commonly asked question by patients affected by Inherited Retinal Disorders [IRD] is- How can I get into a clinical trial.

The short answer is – via Retina South Africa and Myretinatracker.org.

This article will tell you why and how.  

Retina South Africa is in negotiation to bring promising but legitimate clinical trials to South Africa. We are doing this with the support of our Scientific and Medical Advisory Board.

What is a clinical trial

A clinical trial is undertaken to test a promising therapy in humans that has shown promise in cellular and animal models. They are approved by the relevant controlling body in a particular country. By the FDA in the USA, the EMA in Europe and the Clinical Trial Section in the Department of Health in South Africa etc.

They are done in 3 phases with strict control and predetermined inclusion/ exclusion criteria for patient selection such as:

• Condition
• Clinical and genetic diagnosis
• Age range
• State of vision
• Various factors set by the trial organiser

Phase 1 is normally conducted on a small number of patients with advanced vision loss and primarily tests the safety of the intervention. The patients need to live in close proximity to the clinic for regular assessment. If no adverse events occur the trial may sometimes be extended to a phase 1/ 2 where dosage and efficacy are also considered. If trial registration bodies are satisfied then the next phase is opened.

Phase 2 trials include a larger number of patients and may include younger patients with less severe vision loss. Again, safety, efficacy and dosage results are presented to trial controllers for permission to proceed to phase 3.

Phase 3 trials involve a larger number or patients and are often multi centered, and may be held in more than one country. It is here that Retina South Africa, with a large cohort of patients with good clinical and genetic diagnosis from all racial groups, is well placed for consideration for inclusion.

A clinical trial is NOT a cure. Some trials may be “blind’ – where some of the patients will not receive the trial medication or intervention but merely a placebo. Some trials are “Double blind” where even the Doctors do not know who is on the medication being tested and who is on placebo. Where an intervention is only given to one eye, as in gene replacement therapy or stem cell therapy, the second untreated eye acts as a control and no placebo control is required. This is known as open label. Where the treatment is given systemically by mouth or injection then a placebo group is usually necessary.

What can patients do

• Join Retina South Africa or ensure that your membership is active
• Get a recent clinical diagnosis- new imaging technologies such as OCT allows the Ophthalmologist a far better view of the retinal structure and he/ she  may be able to refine their diagnosis of your condition. This will save you time and money for the next step- genetic diagnosis.
• A genetic diagnosis is critical as clinical trials need to compare “like with like” and even for therapies that are not gene specific the inclusion of patients with different conditions will skew results.
• Sign-up to the confidential patient registry managed by the Foundation Fighting Blindness USA. Here researchers, looking for patients with specific conditions can, with your permission contact you for research projects
•  www.myretinatracker.org

 Genetic testing

Contact Retina South Africa for more details on genetic testing, but in brief:

• Consult with a Genetic Counsellor, making sure that you have a recent clinical diagnosis and any details of affected family members. Contact details from Retina SA.
• Make arrangements for the genetic test, with Retina South Africa. Some tests are done locally through the National Health Laboratory Services but mostly overseas in partnership with the University of Cape Town. PLEASE do not contact them directly, this has to be done via Retina South Africa.
• Some sponsorship is available for needy families with young children affected by IRD.

How can you help:

Support our fund raising projects:

• Buy or sell Fury Motor car raffle tickets
• Nominate Retina South Africa as a beneficiary on Myschool.co.za
• Join our Circle of Light competition to win a R500 monthly cash prize
• Introduce Retina South Africa to you companies CSR program- Section 18a Tax deduction certificate available on request
• Host a dinner in the dark for friends and family. Macular or RP simulators on request.
• INNOVATE- create a social media campaign to raise funds

CAUTION

Do not be lured to participate in untried interventions. Unscrupulous practitioners are peddling  interventions using Stem Cells harvested from your own body fat.  These un-differentiated cells can be very dangerous and can lead to complete loss of vision. Reputable researchers are  making great strides and the latest successful stem cell intervention for AMD is featured in our current ENews.

For more information:

Email: National@retinasa.org.za
Helpline: 0860595959

Retina South Africa is assisting in the enrolment of South African patients with a clinical diagnosis of Stargardt Disease. A phase 2 trial for a visual disrupting drug will begin shortly. Minimum age of participants is 16 years of age. For more information contact Retina South Africa email: national@retinasa.org.za

Tel: 0114501181