MitoCanada is committed to curating an active list of clinical trials important to patients and mitochondrial disease research.

University of Manitoba | Caregivers of Children with Rare Genetic Diseases Study

Final Posters (Public w Pre-screener).png

Eligibility to complete this survey has expanded to include a bigger range of caregivers! You are now eligible to complete the survey using the link below if you are the primary caregiver to a child with one of the following:
    1. A rare disease diagnosed in 2018 or after (2018 to present)
    2. A suspected rare disease who has gotten a genetic test in 2018 or after (2018 to present)
    3. You have interacted with a Canadian clinical geneticist or genetic counsellor during your child’s diagnostic journey
    4. You can complete a written online survey in English, French, or Simplified Chinese
If you were previously screened out of the survey but meet this new criteria and would still like to participate you can!

We anticipate that the survey will require approximately 15-20 minutes of your time and is available to complete in English, French, and Simplified Chinese. If you would like to complete the survey in more than one sitting, you will have the option of using a ‘return code’ to save your responses and return to the same survey later.

Take the Survey Now

We kindly ask that you do not post this link on public forums including social media.

If you would like more information about this study, please contact Vivien at truongv2@myumanitoba.ca

REN001 Study | nDNA Primary Mitochondrial Myopathy

M.A.G.I.C Clinic is conducting a study using an investigational medication called REN001, in people with
nuclear DNA (nDNA) PMM. The purpose of this study will be to assess the safety and efficacy of REN001.

Enrolment: Recruiting now

Study Site: M.A.G.I.C Clinic 215-971 64 Ave NE, Calgary AB, Canada T2E 7Z4.

Details:

  • Be willing to provide a blood sample at the Screening visit to check your kidney and liver function and glucose and general health (using a blood test)
  • How you were diagnosed with nuclear DNA primary mitochondrial (PMM) and how treatment was started. (a copy of the genetic results or biopsy report)
  • The age of the person interested in being part of the study (must be 18 years of age or older)
  • Persons below 25 years must provide a wrist radiograph to determine bone growth plate closure.

Sponsor: Reneo Pharmaceuticals

LEARN MORE

Tisento Study | MELAS Lived-Experience Interview Study

This interview study is aimed at learning more about the experiences of individuals with MELAS

Enrolment: Recruiting now

Study Details:

  • Individuals with genetic and clinical confirmation of MELAS, 16 years of age and older and fluent in English.
  • Interviews via phone or web-based teleconference
  • Study will consist of two 45- to 60-minute (1.5 hours total) interviews. 

Compensation: $75 (or Canadian equivalent) for completing each interview for a total of $150 (or Canadian equivalent).

Sponsor: Tisento Therapeutics

LEARN MORE about this study

LEARN MORE about Tisento

NuPower Study | Elampretide in PMD with nDNA Mutations, USA

Phase 3 randomized, double-blind trial evaluating the efficacy and safety of daily subcutaneous injections of Elamipretide in PMD w/nDNA mutations

Enrolment: Recruiting now

Study Site: Multi-USA site; Canadian recruits welcome

Details: 18-70 yrs, genetic confirmation of nDNA mutation

Sponsor: Stealth BioTherapeutics Inc

LC-FAOD In-Clinic Disease Monitoring Study | Global

Global observational long-term study collecting information from adult and pediatric patients with LC-FAOD

Enrolment: Recruiting now

Study Site: Multi-USA site; Canada: SickKids Hospital, Toronto

Details: 300 patients, child, adult and older adults with a confirmed diagnosis of any LC-FAOD sub-type.

Sponsor: Ultragenyx