Mito Study Roundup – November 2023; Seven Studies Available for Patients, Caregivers

Mito Study Roundup – November 2023; Seven Studies Available for Patients, Caregivers

We all want more and better therapies for mitochondrial disease, and to achieve this goal we need patients who are willing to help by participating in clinical research even when no drug is being tested. Please consider engaging in one or more of the non-drug clinical research opportunities below. If you have questions about any of these studies or are unsure of your diagnosis, feel free to contact UMDF’s science team or speak with your healthcare provider. We are here to help!

Video Assessment Study for PolG and Primary Mitochondrial Myopathy (PMM) Patients

  • Goal: Develop better, more meaningful measures of disease by analyzing short self-made videos of normal daily activities using an app on your phone.
  • Why participate? Better, less burdensome measures leads to better designed clinical trials, which gives a better chance of success and ultimately the availability of meaningful therapeutics for mitochondrial disease patients.
  • Enrolling US/Canada patients, ages 16 and older, genetically diagnosed with PolG or PMM.
  • No travel or clinic visits – typically 20-30 minutes of your time in total over 2 weeks
  • $50 compensation for each set of videos uploaded to the app  (up to $150).
  • Click HERE to contact the study organizers!
  • This study is being conducted by Emmes.

Patient Perspective Interview for Primary Mitochondrial Myopathy (PMM) patients

  • Goal: The aim of this research study is to interview people with PMM to better understand the experience of living with PMM, and to discuss your feedback on a questionnaire that is being used to capture information on fatigue.
  • Why participate? As drug trials continue to advance for PMM, it is imperative that drug sponsors have a clear sense of the patient’s perspective on living with the disease.
  • To be eligible you must be at least 18 years or older and have been diagnosed with a Primary Mitochondrial Myopathy.
  • This is an interview style study that will take approximately 60 minutes, and you will be compensated for your time.
  • Click HERE to learn more or to enroll! 
  • This study is being conducted by Clinical Outcome Solutions.

Patient Perspective Survey for Patients with an m.3243A>G Mutation or MELAS diagnosis

  • Goal: Conduct a brief online survey with patients who have a genetically-confirmed 3243 mutation or a MELAS diagnosis to validate key aspects of a clinical development protocol to support a successful and impactful product development
  • Why participate? The 3243 mutation and a diagnosis of MELAS are common forms of adult mitochondrial disease for which significant interest exists in developing therapeutics. Lend your voice in developing better clinical trials!
  • Open to patients between the ages of 18-65, both male and female
  • Confirmed diagnosis of m.3243A>G or MELAS
  • Click HERE to start the survey!
  • This study is being conducted by Trinity Life Sciences.

Patient Perspective Interview for Patients with a MELAS diagnosis

  • Goal: Conduct interviews with patients who are clinically and genetically diagnosed with MELAS to collect perspectives that will help shape how the effects of a drug in development will be assessed in an upcoming clinical trial
  • Open to English-speaking patients at least 16 years of age
  • Participants will receive $150 for completing two 45-minute interviews ($75 per interview) from the comfort of your home and at your convenience.
  • Contact or call (617) 720-0001 if interested.
  • This study is being conducted by Tisento Therapeutics.

Caregiver Perspective Study- Are you a father to a MELAS patient? 

  • Goal: Conduct a brief online survey with fathers of MELAS patients to better understand their perspective as a caregiver of a MELAS patient
  • Why participate? Understanding caregiver burden is an important topic when developing therapeutics, and the father perspective is under-researched. Please encourage any caregivers in your life to consider joining mitoSHARE and participate in this study.
  • Participants will receive a $25 gift card for their time upon completion of the study.
  • Click here to conduct the survey!
  • This study is being conducted by the Wisconsin School of Medicine.

Patient Perspective Interview for Patients with a mitochondrial disease diagnosis.

    • Goal: The study aims to identify the symptoms that have the greatest impact on quality-of-life for those with mitochondrial disease.
    • Why participate? This will help inform future research involving individuals with mitochondrial disease.
    • Open to patients in the United States who are at least 18 years of age
    • Contact if interested.
    • This study is being conducted by the University of Rochester.

Patient/Caregiver Perspective Interview to share your experience with mitochondrial disease.
Update: 11/16/23 — This study is now fully recruited. We’ll adjust this link should it reopen. 

  • Goal: To understand the journey and experiences of patients and caregivers of patients living with mitochondrial disease.
  • Why participate? Your stories and opinions will help industry partners gain a better understanding of the experiences of living with mitochondrial disease.
  • Participants should have two or more of the following symptoms, muscle weakness, difficulty eating, chewing or swallowing, difficulty breathing, loss of functional independence or motor milestones, facial weakness, including ptosis.
  • Participants will receive compensation for their time upon completion of the study.