Mitochondrial DiseasesNCT04113447
Mitochondrial Donation: An 18 Month Outcome Study.
An observational study of Mitochondrial Diseases, run by Newcastle-upon-Tyne Hospitals NHS Trust at 1 site in United Kingdom.
Trial summary
The Investigator proposes to record the fetal and postnatal development of children conceived using Mitochondrial Donation (MD) and to perform expert assessment of development at 18 months (corrected for gestational age) using the internationally validated Bayley-III developmental assessment tool. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Mitochondrial Diseases
- What is being tested?
- observational
- How many people
- 25 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Nov 2017to 30 Nov 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Women with mitochondrial disease undergoing mitochondrial donation treatment
Who may be able to join
- Women will only be eligible if they meet all of the following criteria.
- Women with confirmed mtDNA mutation
- Suitable to undergo Mitochondrial Donation as a treatment (in line with HFEA license)
- Informed Consent for the study obtained before Mitochondrial Donation treatment commences
- Ability and willingness to adhere to the protocol including evaluation schedule
- Willingness to make available information collected during pregnancy, delivery and up to the child's age of 18 months (corrected for gestational age)
Who cannot join
- Women will not be eligible if they meet any of the following criteria
- Declined Mitochondrial Donation as a treatment (in line with HFEA license)
- Inability or unwillingness to adhere to the protocol including evaluation schedule
- Unwillingness to make available information collected during pregnancy, delivery and up to the child's age of 18 months (corrected for gestational age)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom1 site, 1 recruiting
- Recruiting
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne & Wear, NE1 4LP
Robert McFarland, 0191 2820340, robert.mcfarland@ncl.ac.uk
About this study
The Investigator proposes to record the fetal and postnatal development of children conceived using Mitochondrial Donation (MD) and to perform expert assessment of development at 18 months (corrected for gestational age) using the internationally validated Bayley-III developmental assessment tool.
Read the detailed description
To record the fetal and postnatal development of children conceived using Mitochondrial Donation and to perform internationally validated Bayley-III developmental assessment tool at 18 months (corrected for gestational age).
The null hypothesis for this research is that children born following the use of Mitochondrial Donation (IVF) techniques have normal neurodevelopment developmental outcomes at 18 months.
- Conditions
- Mitochondrial Diseases
- Being tested
- observational
- Medical subject headings
- Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
observationalOther
observational
What is being measured
The primary outcome is the main question the study is designed to answer.
A normal neurodevelopmental quotient scored using a Bayley-III ( Bayley Scales of Infant and Toddler Development, Third Edition) at 18 months (corrected
The Bayley Scale of Infant and Toddler Development, Third Edition (Bayley III) measures physical, motor, sensory, and cognitive development in babies and young children. The scale encompasses five developmental domains - cognitive, language, motor, social-emotional and adaptive behaviour. The structure of the Bayley-III Scales allows clinicians to administer each of the five scales (cognitive, language-receptive and expressive, motor-fine and gross, social-emotional, and adaptive behaviour) independent of others. Higher scores in each scale indicates more advanced development. Performance is summarised via scaled scores for each subtest, and composite scores for each scale, together with percentile ranks, developmental age equivalents, and growth scores. Discrepancy information is used to determine whether there are significant differences between a child's abilities in the domains measured, and how prevalent these differences are in the norming sample. A total score is not provided.
Measured: 18 months
Secondary outcomes (2)
1)Normal hearing as assessed by formal audiology,
Formal Audiology
Measured: 18 months
2)Normal vision as assessed by ophthalmologist
Ophthalmology Assessment
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2019
- Study start1 Nov 2017
- Primary completion30 Nov 2027 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Wellcome Trust
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04113447
- Sponsor's protocol ID
- 8075
- Status last verified
- May 2025
- First submitted
- 13 Aug 2018
- Last updated
- 20 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mitochondrial Donation: An 18 Month Outcome Study.
- ClinicalTrials.gov ID
- NCT04113447
- Acronym / sponsor ID
- 8075
- Conditions
- Mitochondrial Diseases
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Newcastle-upon-Tyne Hospitals NHS TrustUniversity, hospital or other
- Collaborators
- Wellcome Trust
- Study start
- 1 Nov 2017 (actual)
- Primary completion
- 30 Nov 2027 (estimated)
- Study completion
- 30 Nov 2027 (estimated)
- First posted
- 2 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Investigator proposes to record the fetal and postnatal development of children conceived using Mitochondrial Donation (MD) and to perform expert assessment of development at 18 months (corrected for gestational age) using the internationally validated Bayley-III developmental assessment tool.
Detailed description
To record the fetal and postnatal development of children conceived using Mitochondrial Donation and to perform internationally validated Bayley-III developmental assessment tool at 18 months (corrected for gestational age).
The null hypothesis for this research is that children born following the use of Mitochondrial Donation (IVF) techniques have normal neurodevelopment developmental outcomes at 18 months.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Women with mitochondrial disease undergoing mitochondrial donation treatment
Key inclusion criteria (6 listed)
- Women will only be eligible if they meet all of the following criteria.
- Women with confirmed mtDNA mutation
- Suitable to undergo Mitochondrial Donation as a treatment (in line with HFEA license)
- Informed Consent for the study obtained before Mitochondrial Donation treatment commences
- Ability and willingness to adhere to the protocol including evaluation schedule
Key exclusion criteria (4 listed)
- Women will not be eligible if they meet any of the following criteria
- Declined Mitochondrial Donation as a treatment (in line with HFEA license)
- Inability or unwillingness to adhere to the protocol including evaluation schedule
- Unwillingness to make available information collected during pregnancy, delivery and up to the child's age of 18 months (corrected for gestational age)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| observationalobservational | Other | – |
Primary outcome
A normal neurodevelopmental quotient scored using a Bayley-III ( Bayley Scales of Infant and Toddler Development, Third Edition) at 18 months (corrected
Time frame: 18 months
The Bayley Scale of Infant and Toddler Development, Third Edition (Bayley III) measures physical, motor, sensory, and cognitive development in babies and young children. The scale encompasses five developmental domains - cognitive, language, motor, social-emotional and adaptive behaviour. The structure of the Bayley-III Scales allows clinicians to administer each of the five scales (cognitive, language-receptive and expressive, motor-fine and gross, social-emotional, and adaptive behaviour) independent of others. Higher scores in each scale indicates more advanced development. Performance is summarised via scaled scores for each subtest, and composite scores for each scale, together with percentile ranks, developmental age equivalents, and growth scores. Discrepancy information is used to determine whether there are significant differences between a child's abilities in the domains measured, and how prevalent these differences are in the norming sample. A total score is not provided.
2 secondary outcomes
1)Normal hearing as assessed by formal audiology,
Time frame: 18 months
2)Normal vision as assessed by ophthalmologist
Time frame: 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mitochondrial Diseases, run by Newcastle-upon-Tyne Hospitals NHS Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
A normal neurodevelopmental quotient scored using a Bayley-III ( Bayley Scales of Infant and Toddler Development, Third Edition) at 18 months (corrected (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2025.