Mitochondrial DiseasesPhase 1/2NCT06051448
Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).
A Phase 1/2 study testing Promoting Resilience in Stress Management (PRISM) and Clinical-focused narrative (CFN) in people with Mitochondrial Diseases and Mitochondrial Myopathies, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
The goal of this study is to find the best way to help people with primary mitochondrial disease deal with the stress of their condition, and to help these people be better able to "bounce back," or be resilient. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies
- What is being tested?
- Promoting Resilience in Stress Management (PRISM) and Clinical-focused narrative (CFN)
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Sep 2023to 28 Feb 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females at least 18 years old
- Capable of giving consent, or there is a legally authorized representative capable of giving consent on the subject behalf and if appropriate, assent.
- Able to speak and read English
- Able to participate in Web- and App-based interventions
- Confirmed molecular diagnosis of primary mitochondrial disease
Who cannot join
- 1. Suspected but no molecular diagnosis primary mitochondrial disease
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Harnessing Resilience in Adults With Primary Mitochondrial Disease: A Pilot Study Investigating the Feasibility of the Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Interventions
The goal of this study is to find the best way to help people with primary mitochondrial disease deal with the stress of their condition, and to help these people be better able to "bounce back," or be resilient. In order to do this, the investigators are going to test two interventions (an intervention means that it aims to change something): Promoting Resilience in Stress Management (PRISM) and clinical-focused narrative (CFN) intervention.
Read the detailed description
The investigators are going to test two interventions in this pilot study. The first is called Promoting Resilience in Stress Management (PRISM). The second is called clinical-focused narrative (CFN) intervention. These interventions involve talking with the researcher about several specific topics.
Participants will join six virtual study visits. They will be assigned to the PRISM group or the CFN group. During the first study visit, participants will learn about the study and will fill out consent forms if they want to participate. In the next four visits, participants will receive one of the study interventions (PRISM or CFN). Some interventions will be supported with a digital app for participants to track progress and review what was talked about in the intervention. The sixth virtual study visit will be a discussion group that happens a couple months after participants finish the intervention.
Participants will also be sent several surveys to complete. One set of surveys is sent just once after the participant enrolls in the study. Another set of surveys is sent after the participant enrolls and again after the participant finishes the interventions.
The study team will review participants' medical records to confirm they have primary mitochondrial disease and to review the genetic diagnosis. If participants do not have copies of their own medical records or if the study team does not already have access to them, the study team will ask participants to sign a release form to obtain a copy of the medical records and/or genetic testing report.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
- Being tested
- Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
- Medical subject headings
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS Syndrome, MERRF Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Promoting Resilience in Stress Management (PRISM)Behavioural
This program teaches the four pillars of resilience (stress management, goal setting, cognitive restructuring, and benefit-finding) in a one-on-one training program consisting of four 20-30 minute sessions occurring once weekly.
Clinical-focused narrative (CFN)Behavioural
This program consists of questions posed to adults with primary mitochondrial disease at regular mitochondrial medicine center visits. Specific topics will be discussed at each session. This consists four 20-30 minute sessions occurring once weekly.
| Study group | Role | What they receive |
|---|---|---|
| Promoting Resilience in Stress Management (PRISM) | Active comparator | Not described Receives: Behavioral: Promoting Resilience in Stress Management (PRISM) |
| Clinical-focused narrative (CFN) | Active comparator | Not described Receives: Behavioral: Clinical-focused narrative (CFN) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine mean score on validated scale assessing resilience in those who receive PRISM and those who receive CFN.
The Connor-Davidson Resilience Scale (CD-RISC) is a validated measure to assess resilience. Possible scores range from 0-100 (higher scores reflecting greater resilience). Mean scores will be calculated on scales administered to subjects before the intervention and scales administered after intervention completion.
Measured: Up to 3 months after the first intervention
Determine change in mean in scores on validated scale assessing resilience between those who receive PRISM and those who receive CFN.
The Connor-Davidson Resilience Scale (CD-RISC) is a validated measure to assess resilience. Possible scores range from 0-100 (higher scores reflecting greater resilience). Mean scores calculated on scales administered before the intervention will be compared to mean scores on scales administered after completion of the intervention.
Measured: Up to 3 months after the first intervention
Determine variability in scores on validated scale assessing resilience between those who receive PRISM and those who receive CFN.
The Connor-Davidson Resilience Scale (CD-RISC) is a validated measure to assess resilience. Possible scores range from 0-100 (higher scores reflecting greater resilience). Variability in scores of scales collected before starting and after completion of the interventions will be calculated.
Measured: Up to 3 months after the first intervention
Determine mean scores on validated scale assessing coping in those who receive PRISM and those who receive CFN.
The Family Crisis Oriented Personal Scale (F-COPES) is a validated measure to assess coping. Possible scores range from 30 to 150 (higher scores reflect higher levels of coping and problem-solving ability). Mean scores will be calculated on scales administered to subjects before the intervention and scales administered after intervention completion.
Measured: Up to 3 months after the first intervention
Determine change in mean scores on validated scale assessing coping between those who receive PRISM and those who receive CFN.
The Family Crisis Oriented Personal Scale (F-COPES) is a validated measure to assess coping. Possible scores range from 30 to 150 (higher scores reflect higher levels of coping and problem-solving ability). Mean scores calculated on scales administered before the intervention will be compared to mean scores on scales administered after completion of the intervention.
Measured: Up to 3 months after the first intervention
Determine variability in scores on a validated scale assessing coping between those who receive PRISM and those who receive CFN.
The Family Crisis Oriented Personal Scale (F-COPES) is a validated measure to assess coping. Possible scores range from 30 to 150 (higher scores reflect higher levels of coping and problem-solving ability). Variability in scores of scales collected before starting and after completion of the interventions will be calculated.
Measured: Up to 3 months after the first intervention
Determine mean scores on validated scale assessing quality of life in those who receive PRISM and those who receive CFN.
The 36-Item Short Form Survey Instrument (SF-36) is a validated measure to assess quality of life. Possible scores range from 0-100 (higher scores reflect a more favorable health state). Mean scores calculated on scales administered before the intervention will be compared to mean scores on scales administered after completion of the intervention.
Measured: Up to 3 months after the first intervention
Determine change in mean in scores on validated scale assessing quality of life between those who receive PRISM and those who receive CFN.
The 36-Item Short Form Survey Instrument (SF-36) is a validated measure to assess quality of life. Possible scores range from 0-100 (higher scores reflect a more favorable health state). Mean scores will be calculated on scales administered to subjects before the intervention and scales administered after intervention completion.
Measured: Up to 3 months after the first intervention
Determine variability in scores on a validated scale assessing quality of life between those who receive PRISM and those who receive CFN.
The 36-Item Short Form Survey Instrument (SF-36) is a validated measure to assess quality of life. Possible scores range from 0-100 (higher scores reflect a more favorable health state). Variability in scores of scales collected before starting and after completion of the interventions will be calculated.
Measured: Up to 3 months after the first intervention
Secondary outcomes (1)
Explore participant experience with PRISM and CFN and assess their acceptability among adults with primary mitochondrial disease.
Semi-structured interviews will be conducted at discussion groups.
Measured: Three months after completing the intervention, discussion groups will occur.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2023
- Study start22 Sep 2023
- Primary completion28 Feb 2024
- Study completion28 Feb 2024
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.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- North American Mitochondrial Disease Consortium (NAMDC), National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Sponsor
- Investigators
- Elizabeth M McCormick, MS, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06051448
- Sponsor's protocol ID
- 22-020222
- Other IDs
- U54NS078059-12
- Status last verified
- Nov 2024
- First submitted
- 12 Sep 2023
- Last updated
- 29 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Data from this study, without direct patient identifiers will be released to an NIH data repository for the purpose of facilitating data sharing according to NIH policy
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Harnessing Resilience in Adults With Primary Mitochondrial Disease: A Pilot Study Investigating the Feasibility of the Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Interventions
- ClinicalTrials.gov ID
- NCT06051448
- Acronym / sponsor ID
- 22-020222
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- North American Mitochondrial Disease Consortium (NAMDC), National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 22 Sep 2023 (actual)
- Primary completion
- 28 Feb 2024 (actual)
- Study completion
- 28 Feb 2024 (actual)
- First posted
- 25 Sep 2023 (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 goal of this study is to find the best way to help people with primary mitochondrial disease deal with the stress of their condition, and to help these people be better able to "bounce back," or be resilient. In order to do this, the investigators are going to test two interventions (an intervention means that it aims to change something): Promoting Resilience in Stress Management (PRISM) and clinical-focused narrative (CFN) intervention.
Detailed description
The investigators are going to test two interventions in this pilot study. The first is called Promoting Resilience in Stress Management (PRISM). The second is called clinical-focused narrative (CFN) intervention. These interventions involve talking with the researcher about several specific topics.
Participants will join six virtual study visits. They will be assigned to the PRISM group or the CFN group. During the first study visit, participants will learn about the study and will fill out consent forms if they want to participate. In the next four visits, participants will receive one of the study interventions (PRISM or CFN). Some interventions will be supported with a digital app for participants to track progress and review what was talked about in the intervention. The sixth virtual study visit will be a discussion group that happens a couple months after participants finish the intervention.
Participants will also be sent several surveys to complete. One set of surveys is sent just once after the participant enrolls in the study. Another set of surveys is sent after the participant enrolls and again after the participant finishes the interventions.
The study team will review participants' medical records to confirm they have primary mitochondrial disease and to review the genetic diagnosis. If participants do not have copies of their own medical records or if the study team does not already have access to them, the study team will ask participants to sign a release form to obtain a copy of the medical records and/or genetic testing report.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Promoting Resilience in Stress Management (PRISM) | Active comparator | Behavioral: Promoting Resilience in Stress Management (PRISM) |
| Clinical-focused narrative (CFN) | Active comparator | Behavioral: Clinical-focused narrative (CFN) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Males or females at least 18 years old
- Capable of giving consent, or there is a legally authorized representative capable of giving consent on the subject behalf and if appropriate, assent.
- Able to speak and read English
- Able to participate in Web- and App-based interventions
- Confirmed molecular diagnosis of primary mitochondrial disease
Key exclusion criteria (1 listed)
- 1. Suspected but no molecular diagnosis primary mitochondrial disease
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 |
|---|---|---|
| Promoting Resilience in Stress Management (PRISM)This program teaches the four pillars of resilience (stress management, goal setting, cognitive restructuring, and benefit-finding) in a one-on-one training program consisting of four 20-30 minute sessions occurring once weekly. | Behavioural | Promoting Resilience in Stress Management (PRISM) |
| Clinical-focused narrative (CFN)This program consists of questions posed to adults with primary mitochondrial disease at regular mitochondrial medicine center visits. Specific topics will be discussed at each session. This consists four 20-30 minute sessions occurring once weekly. | Behavioural | Clinical-focused narrative (CFN) |
Primary outcome
Determine mean score on validated scale assessing resilience in those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The Connor-Davidson Resilience Scale (CD-RISC) is a validated measure to assess resilience. Possible scores range from 0-100 (higher scores reflecting greater resilience). Mean scores will be calculated on scales administered to subjects before the intervention and scales administered after intervention completion.
Determine change in mean in scores on validated scale assessing resilience between those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The Connor-Davidson Resilience Scale (CD-RISC) is a validated measure to assess resilience. Possible scores range from 0-100 (higher scores reflecting greater resilience). Mean scores calculated on scales administered before the intervention will be compared to mean scores on scales administered after completion of the intervention.
Determine variability in scores on validated scale assessing resilience between those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The Connor-Davidson Resilience Scale (CD-RISC) is a validated measure to assess resilience. Possible scores range from 0-100 (higher scores reflecting greater resilience). Variability in scores of scales collected before starting and after completion of the interventions will be calculated.
Determine mean scores on validated scale assessing coping in those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The Family Crisis Oriented Personal Scale (F-COPES) is a validated measure to assess coping. Possible scores range from 30 to 150 (higher scores reflect higher levels of coping and problem-solving ability). Mean scores will be calculated on scales administered to subjects before the intervention and scales administered after intervention completion.
Determine change in mean scores on validated scale assessing coping between those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The Family Crisis Oriented Personal Scale (F-COPES) is a validated measure to assess coping. Possible scores range from 30 to 150 (higher scores reflect higher levels of coping and problem-solving ability). Mean scores calculated on scales administered before the intervention will be compared to mean scores on scales administered after completion of the intervention.
Determine variability in scores on a validated scale assessing coping between those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The Family Crisis Oriented Personal Scale (F-COPES) is a validated measure to assess coping. Possible scores range from 30 to 150 (higher scores reflect higher levels of coping and problem-solving ability). Variability in scores of scales collected before starting and after completion of the interventions will be calculated.
Determine mean scores on validated scale assessing quality of life in those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The 36-Item Short Form Survey Instrument (SF-36) is a validated measure to assess quality of life. Possible scores range from 0-100 (higher scores reflect a more favorable health state). Mean scores calculated on scales administered before the intervention will be compared to mean scores on scales administered after completion of the intervention.
Determine change in mean in scores on validated scale assessing quality of life between those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The 36-Item Short Form Survey Instrument (SF-36) is a validated measure to assess quality of life. Possible scores range from 0-100 (higher scores reflect a more favorable health state). Mean scores will be calculated on scales administered to subjects before the intervention and scales administered after intervention completion.
Determine variability in scores on a validated scale assessing quality of life between those who receive PRISM and those who receive CFN.
Time frame: Up to 3 months after the first intervention
The 36-Item Short Form Survey Instrument (SF-36) is a validated measure to assess quality of life. Possible scores range from 0-100 (higher scores reflect a more favorable health state). Variability in scores of scales collected before starting and after completion of the interventions will be calculated.
1 secondary outcome
Explore participant experience with PRISM and CFN and assess their acceptability among adults with primary mitochondrial disease.
Time frame: Three months after completing the intervention, discussion groups will occur.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2024.
- Individual participant dataSharing planned: Yes. Data from this study, without direct patient identifiers will be released to an NIH data repository for the purpose of facilitating data sharing according to NIH policy
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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. This study is not recruiting at the moment.
Elizabeth M McCormick, MS principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Promoting Resilience in Stress Management (PRISM) and Clinical-focused narrative (CFN) in people with Mitochondrial Diseases and Mitochondrial Myopathies, run by Children's Hospital of Philadelphia at 1 site in United States.
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 5 inclusion and 1 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: finished, as of the sponsor's last update on 29 Nov 2024. 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 States. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine mean score on validated scale assessing resilience in those who receive PRISM and those who receive CFN. (time frame: Up to 3 months after the first intervention). The study lists 9 primary outcomes in all. 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 29 Nov 2024.