MELAS SyndromePhase 4NCT01831934
Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)
A Phase 4 study testing Fluzone® in people with MELAS Syndrome, run by Stanford University at 1 site in United States.
Trial summary
This pilot clinical study evaluated the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV) in mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome (MELAS) volunteers and controls. Read more about this study
- Who is it for?
- Ages 13 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- MELAS Syndrome
- What is being tested?
- Fluzone®
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2010to Mar 2012
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 13 to 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Age between 13-60 years for MELAS volunteers OR age between 18-65 years for adult control volunteers. Control volunteers will be age-matched +/-5 years to enrolled MELAS volunteers. If the MELAS volunteer has diabetes and uses insulin daily, their control will be an adult with diabetes who uses insulin daily.
- General good health at time of enrollment
- Willing and able to sign Informed Consent
- Available for follow-up for the planned duration of the study
- Acceptable medical history by screening evaluation and brief clinical assessment
- All female participants of childbearing potential must use an acceptable method of contraception and not become pregnant for the duration of the clinical phase of the study (approximately 1 month to completion of Visit 4). (Acceptable contraception may include but is not limited to implants, injectables, combined oral contraceptives, effective intrauterine devices (IUDs), sexual abstinence, or a vasectomized partner).
Who cannot join
- Allergy to egg or egg products or allergy to vaccine components, including thimerosal
- Active systemic or serious concurrent illness, including febrile illness, within the 3 days prior to study vaccination
- History of immunodeficiency, known or suspected impairment of immunologic function, including, but not limited to, clinically significant liver disease, moderate to severe renal disease, or any other chronic disorder which, in the opinion of the investigator, might jeopardize volunteer safety or compliance with the protocol.
- Blood pressure >150 systolic or >95 diastolic at Visit 1.
- Hospitalization in the past year for congestive heart failure or emphysema.
- History of chronic Hepatitis B or C.
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- Recent or current use of immunosuppressive medication, including glucocorticoids (corticosteroid nasal sprays are permissible).
- Malignancy, other than squamous cell or basal cell skin cancer (includes solid tumors such as breast cancer or prostate cancer with recurrence in the past year, and any hematologic cancer such as leukemia).
- Autoimmune disease (including rheumatoid arthritis treated with immunosuppressive medication such as Plaquenil, methotrexate, prednisone, Enbrel) which, in the opinion of the investigator, might jeopardize volunteer safety or compliance with the protocol.
- History of blood dyscrasias, renal disease, or hemoglobinopathies requiring regular medical follow up or hospitalization during the preceding year
- Receipt of blood or blood products within the past 6 months
- Medical or psychiatric condition or occupational responsibilities that preclude subject compliance with the protocol
- Inactivated vaccine 14 days prior to vaccination or live, attenuated vaccine within 60 days of vaccination
- History of Guillain-Barré Syndrome
- Pregnant or lactating woman
- Use of investigational agents within 30 days prior to enrollment
- Donation of the equivalent of a unit of blood within 6 weeks prior to enrollment
- Any condition which, in the opinion of the investigator, might interfere with volunteer safety, study objectives or the ability of the participant to understand or comply with the study protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: Metabolic and Immune Responses to TIV in Patients With Mitochondrial Disease
This pilot clinical study evaluated the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV) in mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome (MELAS) volunteers and controls.
Read the detailed description
This pilot clinical study evaluated the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV). This study will consist of two cohorts: MELAS syndrome volunteers (a specific identified disorder of mitochondrial dysfunction: mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) between 13-60 years OR adult control volunteers between 18-65 years of age. Both cohorts will receive the same treatment: a single vaccination with an FDA-licensed intramuscular seasonal trivalent inactivated influenza vaccine (TIV).
All participants will receive a single administration of a licensed influenza vaccine. Prior to vaccination, participants will provide information regarding health history and responses to health questionnaires. A blood sample and urine specimen were collected prior to vaccination, and at 6 hours, 5-7 days and 26-30 days post-immunization.
- Conditions
- MELAS Syndrome
- Being tested
- Fluzone®
- Keywords
- Mitochondrial disorder, Trivalent inactivated influenza vaccine, Adolescents and adults, Immune response, Metabolic response
- Medical subject headings
- MELAS Syndrome, Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fluzone®Biological
Also called trivalent inactivated influenza vaccine (TIV), FDA-licensed seasonal influenza vaccine
This vaccine is given intramuscularly, either the 2010-2011 or 2011-2012 vaccination was given as appropriate
| Study group | Role | What they receive |
|---|---|---|
| MELAS group:13-60 years of age. | Experimental | Fluzone® 2010-2011 Formula or 2011-2012 depending on year Receives: Biological: Fluzone® |
| Control Group: 18-65 years of age | Experimental | Fluzone® 2010-2011 Formula or 2011-2012 depending on year Receives: Biological: Fluzone® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Safety of TIV Vaccine
We will measure solicited local and systemic adverse events and SAEs for 1 month following immunization
Measured: Day 0 to Day28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2013 (estimated)
- Study startSep 2010
- Primary completionDec 2011
- Study completionMar 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for MELAS SyndromeStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe 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 4
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Principal investigator, Cornelia L. Dekker, Stanford University
- Investigators
- Cornelia L Dekker, MD, Principal investigator, Stanford UniversityGregory M Enns, MBChB, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01831934
- Sponsor's protocol ID
- SU-18615
- Other IDs
- R21HD061709-01A1
- Status last verified
- Apr 2017
- First submitted
- 19 Mar 2013
- Last updated
- 30 May 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Metabolic and Immune Responses to TIV in Patients With Mitochondrial Disease
- ClinicalTrials.gov ID
- NCT01831934
- Acronym / sponsor ID
- SU-18615
- Conditions
- MELAS Syndrome
- Keywords
- Mitochondrial disorder, Trivalent inactivated influenza vaccine, Adolescents and adults, Immune response, Metabolic response
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- Sep 2010
- Primary completion
- Dec 2011 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 15 Apr 2013 (estimated)
- Results
- Posted 21 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This pilot clinical study evaluated the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV) in mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome (MELAS) volunteers and controls.
Detailed description
This pilot clinical study evaluated the safety and metabolic responses to a licensed inactivated seasonal influenza vaccine (TIV). This study will consist of two cohorts: MELAS syndrome volunteers (a specific identified disorder of mitochondrial dysfunction: mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) between 13-60 years OR adult control volunteers between 18-65 years of age. Both cohorts will receive the same treatment: a single vaccination with an FDA-licensed intramuscular seasonal trivalent inactivated influenza vaccine (TIV).
All participants will receive a single administration of a licensed influenza vaccine. Prior to vaccination, participants will provide information regarding health history and responses to health questionnaires. A blood sample and urine specimen were collected prior to vaccination, and at 6 hours, 5-7 days and 26-30 days post-immunization.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MELAS group:13-60 years of age. | Experimental | Biological: Fluzone®Fluzone® 2010-2011 Formula or 2011-2012 depending on year |
| Control Group: 18-65 years of age | Experimental | Biological: Fluzone®Fluzone® 2010-2011 Formula or 2011-2012 depending on year |
Who may qualify
- Ages
- 13 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Age between 13-60 years for MELAS volunteers OR age between 18-65 years for adult control volunteers. Control volunteers will be age-matched +/-5 years to enrolled MELAS volunteers. If the MELAS volunteer has diabetes and uses insulin daily, their control will be an adult with diabetes who uses insulin daily.
- General good health at time of enrollment
- Willing and able to sign Informed Consent
- Available for follow-up for the planned duration of the study
- Acceptable medical history by screening evaluation and brief clinical assessment
Key exclusion criteria (18 listed)
- Allergy to egg or egg products or allergy to vaccine components, including thimerosal
- Active systemic or serious concurrent illness, including febrile illness, within the 3 days prior to study vaccination
- History of immunodeficiency, known or suspected impairment of immunologic function, including, but not limited to, clinically significant liver disease, moderate to severe renal disease, or any other chronic disorder which, in the opinion of the investigator, might jeopardize volunteer safety or compliance with the protocol.
- Blood pressure >150 systolic or >95 diastolic at Visit 1.
- Hospitalization in the past year for congestive heart failure or emphysema.
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 |
|---|---|---|
| Fluzone®Also: trivalent inactivated influenza vaccine (TIV), FDA-licensed seasonal influenza vaccineThis vaccine is given intramuscularly, either the 2010-2011 or 2011-2012 vaccination was given as appropriate | Biological | Control Group: 18-65 years of age, MELAS group:13-60 years of age. |
Primary outcome
Clinical Safety of TIV Vaccine
Time frame: Day 0 to Day28
We will measure solicited local and systemic adverse events and SAEs for 1 month following immunization
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for MELAS Syndrome
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Not yet recruitingPhase 1/2
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
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North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
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MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
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.
Cornelia L Dekker, MD principal investigator
Stanford University
Gregory M Enns, MBChB principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Fluzone® in people with MELAS Syndrome, run by Stanford University 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 6 inclusion and 18 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 30 May 2017. 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 phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Apr 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Clinical Safety of TIV Vaccine (time frame: Day 0 to Day28). 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 30 May 2017.