MELAS Syndrome
Conditions
Keywords
Mitochondrial disorder, Trivalent inactivated influenza vaccine, Adolescents and adults, Immune response, Metabolic response
Brief 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.
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.
Interventions
BIOLOGICALFluzone®
This vaccine is given intramuscularly, either the 2010-2011 or 2011-2012 vaccination was given as appropriate
Sponsors
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Stanford University
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
13 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
Inclusion criteria include: * 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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Safety of TIV Vaccine | Day 0 to Day28 | We will measure solicited local and systemic adverse events and SAEs for 1 month following immunization |
Other
| Measure | Time frame | Description |
|---|---|---|
| Measurement of Intracellular Glutathione by Hi-D FACS (CD4 and CD8 T Cells) and Tandem Mass Spectrometry (Whole Blood) | Day 0-Day28 | This method relies on the ability of intracellular glutathione S-transferases to tag GSH to bimane to yield a bimane-GS conjugate that fluoresces at 440 nm. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| MELAS Group Fluzone®
Fluzone® | 12 |
| Control Group Fluzone®
Fluzone® | 10 |
| Total | 22 |
Baseline characteristics
| Characteristic | MELAS Group | Total | Control Group |
|---|---|---|---|
| Age, Continuous | 38.74 years STANDARD_DEVIATION 8.06 | 38.13 years STANDARD_DEVIATION 8.99 | 37.39 years STANDARD_DEVIATION 10.04 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 11 Participants | 20 Participants | 9 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 5 Participants | 4 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 2 Participants | 3 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) White | 9 Participants | 13 Participants | 4 Participants |
| Region of Enrollment United States | 12 participants | 22 participants | 10 participants |
| Sex: Female, Male Female | 9 Participants | 15 Participants | 6 Participants |
| Sex: Female, Male Male | 3 Participants | 7 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 12 | 0 / 10 |
| other Total, other adverse events | 4 / 12 | 0 / 10 |
| serious Total, serious adverse events | 0 / 12 | 0 / 10 |
Outcome results
Primary
Clinical Safety of TIV Vaccine
We will measure solicited local and systemic adverse events and SAEs for 1 month following immunization
Time frame: Day 0 to Day28
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| MELAS Group | Clinical Safety of TIV Vaccine | Number of Severe Adverse Events | 0 Participants |
| MELAS Group | Clinical Safety of TIV Vaccine | Number of Adverse Events | 4 Participants |
| MELAS Group | Clinical Safety of TIV Vaccine | Number with no Adverse Events | 8 Participants |
| Control Group | Clinical Safety of TIV Vaccine | Number of Adverse Events | 0 Participants |
| Control Group | Clinical Safety of TIV Vaccine | Number of Severe Adverse Events | 0 Participants |
| Control Group | Clinical Safety of TIV Vaccine | Number with no Adverse Events | 10 Participants |
Other Pre-specified
Measurement of Intracellular Glutathione by Hi-D FACS (CD4 and CD8 T Cells) and Tandem Mass Spectrometry (Whole Blood)
This method relies on the ability of intracellular glutathione S-transferases to tag GSH to bimane to yield a bimane-GS conjugate that fluoresces at 440 nm.
Time frame: Day 0-Day28