Mito Study Roundup: Studies Available for Patients, Caregivers

Press Room, Research News

Mito Study Roundup: Studies Available for Patients, Caregivers

Updated 8/22/24

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!

Patient Perspective Interview for Adolescent Patients with a MELAS diagnosis

  • Goal: Conduct interviews with adolescent 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 ages 12 – 17
  • Participants will receive $150 for completing two 45-minute interviews ($75 per interview) from the comfort of your home and at your convenience.
  • Contact MELAS@adelphivalues.com or call (617) 720-0001 if interested. 
  • This study is being conducted by Tisento Therapeutics.

Core Outcome Set for MNGIE Patients (COLT-MNGIE Project):

Goal: This project aims to develop a Core Outcome Set (COS) for MNGIE, in order to provide a consistent framework to evaluate the efficacy and safety of liver transplantation and other enzyme-replacement treatment standards and patient-centered outcomes.

  • You are eligible to participate in this survey if:
    • You are a caregiver of a patient with MNGIE AND/OR
    • You are a patient with MNGIE 18 years of age or older.
  • Responding to this survey is entirely voluntary and takes approximately 15 minutes. Responses are anonymous and no individual identifying information will be requested.
  • Please visit: https://eu.jotform.com/form/240093045599360 to participate in this important survey. (This survey is available in 5 different languages)
  • This survey will be available until September 27, 2024.
  • If you should have questions or concerns about the study, please contact the COLT-MNGIE study team at scientific.office@mitocon.it.

 NIH Metabolism, Infection and Immunity (MINI) Study:

 Goal: Conduct interviews with caregivers of children with mitochondrial disease to participate in a survey about the use of immune therapies in children with mitochondrial disease. The purpose of this study is to understand how widespread these therapies are in the community, why some people are receiving them and how they may or may not benefit their disease. We ask you to complete this survey regardless of whether or not your child with mitochondrial disease is receiving immunotherapy.

  • You are eligible to participate in this survey if:
    • You are a caregiver of a child (0-17) with mitochondrial disease AND
    • You are 18 years of age or older.
  • Responding to this survey is entirely voluntary and takes approximately 15 minutes.Responses are anonymous and no individual identifying information will be requested. Aggregate survey data will be shared within the NIH and published. No individual data will be shared. Only the study team will have access to individual answers, without any identifiers. 
  • Please visit: https://www.surveymonkey.com/r/UMDF-eblast to participate in this important survey. 
  • If you should have questions or concerns about the study, please contact the NIH study team at ministudy@nih.govor 301.451.9145.