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PWS Clinical Trials Panel [2020 CONFERENCE VIDEO]

In this video, Dr. Theresa Strong explains the drug development pathway for PWS, followed by trial representatives describing current PWS clinical trials.

PWS clinical trials are taking place around the world to help us understand Prader-Willi syndrome and investigate new treatments. In this 60-minute video, Dr. Strong summarizes the drug development pathway, followed by trial representatives describing their studies and explaining what is involved in participation and who is eligible to participate. Following presentations by Dean Carson (Saniona - Tesomet), Dr. Eric Hollander (CBDV), and Dr. Deepan Singh (Guanfacine), the panel answers questions from attendees at the 2020 FPWR Virtual Conference.

Click below to watch the video. If you're short on time, scroll down for time stamps to find the portions you're most interested in.


Presentation Summary With Timestamps

1:21 Steps of the Drug Development Process

  • Preclinical steps involves testing done on animal models for safety and efficacy testing
  • Clinical Trials have three phases, and in the third phase, there is a longer exposure to the drug in order to compare to the ‘current standard’ or standard of care. 
  • After FDA approval, the drugs are applied to the population 

3:53 What happens after the Phase 3 Clinical Trial?

  • And NDA or “New Drug Application” is formally proposed to the FDA for sales and marketing in the U.S.
  • FDA reviews data and does the benefit outweigh the drug negative effects.
  • #1 reason that PWS clinical trials are delayed or not completed is due to an insufficient number of participants. 

5:55 Risks and Benefits of Clinical Trials

  • There are many benefits to participating in trials, and there are protections for study participants.
  • Find out about the specific Clinical Trials, speak to your doctor or medical professional, ask questions and download useful resources.
  • Make the decision that is right for you and your loved one.

9:50 Drug Development Pipeline for PWS 

14:00 Q&A with Dr. Strong

15:27 Dr. Singh, presenting on the Guanfacine Clinical Trial 

  • Guanfacine is a drug that has been used for ADHD/impulsiveness in autism
  • Studies have shown that the part of the brain in charge or executive planning is the prefrontal cortex, and he has seen positive results in extended release of guanfacine in PWS patients ages 6 to 26.

22:55 Guanfacine Study Events

  • There are a total of four visits that the patients need to make to Dr. Singh’s office in order to be able to complete the three phases for this study.
  • All other scheduled visits can be done via videoconference.

26:12 Q&A with Dr. Singh

31:00 Dean Carson, PhD, from Saniona, presenting on Tesomet in PWS and the proposed Clinical Trial

33:07 Overview of Tesomet

  • A combination of two drugs that is meant to reduce hyperphagia by controlling craving for food and appetite in people with PWS.
  • It also increases metabolic activity.

34:41 Overview of the completed Phase 2a in PWS

  • This is the first time Tesomet is used in the PWS population.
  • In development, and took place with 18 participants (9 adults, 9 adolescentes) in Hungary and Czech Republic. 
  • Adult population took 0.5 milligram doses of Tesomet.

37:27 Data for Change in Body Weight in Adults

  • There was a general 5% decrease in body weight, while hyperphagia was also successfully reduced.
  • There were 2-3x higher than expected plasma concentrations.
  • Adverse events related to Tesomet were mostly physiatric, and understanding baseline physiatric history is important.
  • There were no clinically meaningful differences in heart rate of blood pressure between treatment groups.

41:04 Adolescent adjusted doses for Tesomet were based on the adult study results and data

  • The adjusted dose for adolescents was 0.125 milligrams of Tesomet during the first phase.
  • There was a 2x increase in the second, open-label phase of the study, making it a dose of 0.25 milligrams of Tesomet per dose.
  • There was a reduction in the body weight but not hyperphagia scores. Also, hyperphagia scores are highly variable in adolescents with PWS.
  • AEs related to Tesomet were mostly psychiatric.

43:35 Overview of proposed future clinical trials with PWS

  • 16-week double blind study. The initial dosed populations would be given a variant of concentrations of Tesamet ranging from 0.125 to 0.375 mg, and then it would be likely to have an open label extension with dose adjustments.

45:35 Summary of proposed study population for PWS Clinical Trial

  • Males and females
  • Confirmed PWS genetic diagnosis including determination of genetic subtype
  • Ages 12 to 65, and specific BMI ranges for both adults and adolescents.

48:16 Eric Hollander, presenting on the CBDV Clinical Trial

48:40 Explanation of the mechanisms of CBDV

  • The Clinical Trial will take place with children from 12 up to adults 30 years old. 
  • Phytocannabinoid
  • No THC, non-endocannabinoid 
  • Not medical marijuana

51:30 Therapeutics of CBDV

  • Anti-convulsant, anti-anxiety, anti-inflammatory, anti-psychotic, anti-oxidative/neuro-protective, anti-addictive

54:12 Primary focus and secondary focus

  • Primary focus is on irritability.
  • Secondary focus in repetitive behaviors, hyperphagia using the hyperphagia scale for Clinical Trials (HQ-CT), sleep quality, behavioral rigidity, global improvement and ABC subscales in lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech.

57:15 The study design overview

  • The patient is taken in, done a complete physical exam and then randomized into the treatment or placebo group. After the 12-week treatment trial ends, then the patient is taken off during two weeks off the treatment, and then a follow-up happens on week 14.
  • Side effects of CBDV: Gastrointestinal, fatigue, drowsiness and headaches. 

1:00:51 Q&A with Dr. Hollander

1:05:00 Q&A with all four panelistsNew call-to-action

Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.