A Step By Step Guide to Clinical Trials By Katrina Byrd

On November 18, 2019, Dora(my partner. 23 years) and I sat in the cold, stiff exam room listening to the doctor who couldn’t look us in the eye.

”ALS,” he said. “No cure. Life expectancy is two to five years.”

Dora sat motionless digesting the news of her death sentence. It was very clear that we were alone in this. Her only child was dead - shot by a seven year old over thirty years earlier. Her family wasn’t there. I was the only person there to care for her, the only reason she had for living.

We discussed future doctor’s appointments, changing her feeding tube formula and getting Affairs in order. We didn’t discuss clinical trials.  After Dora’s death, my advocacy work began. The main focus of my advocacy was to help people have the tools and resources needed to navigate this 100% fatal disease. 

While the goal of clinical trials is advancing medical research, many people with an  ALS diagnosis are in search of treatments to slow, halt, or reverse progression. Every clinical trial starts with a hypothesis or theory of an intervention that may affect the disease. All clinical trials have a sponsor- a drug company, a research institute or a single investigator. There are two main types of clinical trials: interventional and observational.

An interventional trail tests experimental treatments. individuals are placed into one of two groups - treatment group(receiving the intervention) or a placebo group(not receiving the intervention). In an observational trial, the investigators observe individuals and collect information. There is no placebo group in an observational trial. There are no phases and the observational trial is continually ongoing and does not need FDA approval. Datacollected helps investigators understand how fast or slow the disease progresses, loss of function and when that loss occurs. An example of an observational trial is the ALS TDI ARC study.  

All trials start in a preclinical phase which takes place in the laboratory. Potential targets are identified and animal studies are done. Phase 1, is the first time an intervention is given to trial participants. A Phase 1 trial, which is small, focuses on safety and efficacy. Phase 2 trials are larger and focuses on safety and efficacy. Phase 3 is a large trial focused on gathering data to demonstrate the efficacy of the treatment and to prepare for FDA approval. If and when all 3 trials are completed successfully, the investigator will apply for FDA review and hopefully gain FDA approval. If approved, the intervention becomes available to the public. Phase 4 trials are an ongoing gathering of data on an approved intervention. This process can last an average of 10 years from hypotheses to approval.

Every interventional clinical trial has eligibility criteria - key requirements or characteristics that people who want to participate must meet. Criteria can vary from trial to trial and are used to select participants who are likely to provide definitive results. For example, some drugs are geared to fast progression, some to specific

genes, and some to a certain type of ALS like bulbar onset. Criteria is divided into two

groups inclusion - criteria you must meet and exclusion - criteria you must not meet. Examples include time from diagnosis or symptom onset, typically under 2 years. Breathing score, usually and FVC must be greater than 60%, ability to

swallow and absence of tracheostomy are just a few.

Finding a clinical trial, a step by step guide.

1. Find an interventional trial that is of interest.

2. Discuss the trial with your physician.

3. If you decide to apply, the participant will need to review and sign an informed consent.

4. The participant will be screened for eligibility.

5. If accepted, the participant will be randomly assigned to the treatment or placebo group(neither the participant nor the principal investigator will know which group the participant is in). 

6. The participant starts treatments, data is collected. During trial participation, participants will continue to see their regular physician and they will continue going to their ALS clinic appointments. 

7. The study then concludes and the drug sponsor analyzes the data. It can take up to a year to analyze and release clinical trial findings. At this time, participants will find out whether they were receiving the intervention or if they were in the placebo group.

Acknowledgements - a soecial thank you to Jill Brattain and the IAM ALS Clinical Trials team for their efforts ensuring that everyone has the opportunity to learn about clinical trials.

Resources to search for clinical trials

1.   I AM ALS’s signal dashboard. This is a search tool for all ALS clinical trials, note that there are several lists where you can narrow your search. You can search by Country, State, Treatment,Remote trial, Trial Phase, Genetic Target and Recruitment status. At the top you see you can search genetic and observational trials. The next section shows both the EAPs available as well as the breakdown of drugs in the different phases. And a map where you can click on a state and see where clinical trial sites are located. The last section is a list of every clinical trial for ALS, most important here is a specific link to the trial information and an email address/phone number to inquire about the trial yourself. https://www.iamals.org/get-help/als-signal-clinical-research-dashboard/

ALS-TDI’s trial finder is an interactive tool that helps find a trial based on the information you provide. https://www.als.net/als-trial-navigator/

1. ALS Untangled reviews alternative and off label treatments. https://www.alsuntangled.com/

Katrina Byrd, writer and playwright living in Jackson, MS, received her MFA in Creative Writing from Mississippi University for Women. An emerging writer published in several magazines, Byrd is a six time Arts Commussion grantvrecipient. Diagnosed with amyotrophic lateral sclerosis(ALS) - 100% fatal- November 18, 2019, Dora - Katrina Byrd’s partner of 23 years - died on February 2, 2020, 76 days after diagnosis. A few weeks after Dora’s death, Byrd’s advocacy work began. For the past four years, Byrd has become fluent in telling hard and uncomfortable stories. With her business, Daily Flounce, Byrd offers virtual writing courses and coaching services for writers, creatives and advocates. With her Boa Flouncer alter ego, she inspires, motivates and spreads joy.