MitoCanada is committed to curating an active list of clinical trials important to patients and mitochondrial disease research.
Survey Study | Individuals living with Leigh Syndrome or caregivers of individuals living with Leigh Syndrome
This study aims to understand topics related to quality of life, physical, mental and social functioning and symptoms that are important to people affected by rare disorders such as Leigh syndrome.
Enrolment: Recruiting now
INTERESTED? Click on the link that is relevant to you:
I have been diagnosed with one of the listed disorders, such as Leigh syndrome
I am a parent or caregiver of someone diagnosed with one of the disorders listed
CONTACT: For questions, please contact: t.h.g.steenbergen@amsterdamumc.nl or a.r.muller@amsterdamumc.nl
Survey Study | Adults living with MCADD or caregivers of individuals living with MCADD
This study aims to better understand the experiences of adult patients living with MCADD and caregivers of pediatric patients living with MCADD
Enrolment: Recruiting now
Who may qualify:
- Patients located in the US and Canada
- Adult patients (i.e. over the age of majority where they live) with a confirmed diagnosis of MCADD or legal guardians/primary caregivers of a pediatric patient (i.e. younger than the age of majority where they live) living with MCADD
- Participants able to read, write, and comfortably communicate in English
- Participants willing and able to participate in a 30-minute online survey
- Participants able to describe age the formal MCADD diagnosis was made
- Participants able to describe what event(s) led to a formal MCADD diagnosis
- Participants able to describe the speciality of the physician who provided a formal MCADD diagnosis
- If needed, participants able to participate in a phone screening to answer additional questions regarding their diagnostic journey or the diagnostic journey of the person living with MCADD
Who may not qualify:
- Participants unable to understand the nature, scope, and consequences of the survey study and presents evidence of an uncooperative attitude
- Caregiver participant is paid to provide care for the person living with MCADD
Compensation: $50 for completion of survey.
INTERESTED? Complete the screening form to determine eligibility: Screening Form Link
CONTACT: For questions, call: 617-612-4054 or email: mcadd@lumanity.com
SPONSOR: Ultragenyx
Neurophth Study | LHON Gene Therapy Trial
this clinical study is to evaluate the safety, tolerability and preliminary efficacy of NFS-02 in the treatment of LHON caused by mitochondrial ND1 gene mutation.
Enrolment: Recruiting now
Study Details:
- Individuals must be 18 – 75 years of age
- Clinical manifested vision loss due to LHON, and any eye BCVA ≥ 0.5 LogMAR
- Genotype testing result shows the presence of G3460A mutation in the ND1 gene
- Willingness to comply with the clinical study protocol and 5 years of long-term follow-up
Trial Locations: Colorado, USA; Beijing, Guangzhou, Wenzhou China
Tisento Study | Teen MELAS Phone Interview Study
This interview study is aimed at learning more about the experiences of teens with MELAS
Enrolment: Recruiting now
Study Details:
- Share your MELAS experience to help shape the development of investigational medicines
- Individuals with genetic and clinical confirmation of MELAS, 12 – 17 years of age, are able to read, speak and comprehend English
- Interviews via phone or web-based teleconference
- Study will consist of two 45-minute interviews to be completed at your convenience.
Compensation: Receive up to $150 CAD for your time.
INTERESTED? Simply provide your first name and contact information (phone and/or email) to Adelphi Values, who is performing the interviews for Tisento and reference the MELAS study
CONTACT: MELAS@adelphivalues.com or call (617) 720-0001
SPONSOR: Tisento Therapeutics
Teens, learn more about this study here
Parents, learn more about this study here
