X-Linked Dystonia Parkinsonism
Conditions
Keywords
prospective randomized trial
Brief summary
To see whether MINGO, a food supplement, will be able to lessen the drastic weight loss seen among X-linked Dystonia Parkinsonism patients.
Detailed description
X-linked dystonia parkinsonism (XDP, formerly known as DYT3) occurs primarily in Filipino males and is characterized by neurodegenerative dystonia and parkinsonism. It is currently recognized to have wide phenotypic variability relating to age of onset, location of disease onset and rate of severity/disease progression. Most patients begin with focal dystonia that generalizes with the development of parkinsonism later in disease course. XDP patients suffer from severe nutritional loss due to symptoms such as dysphagia, loss of appetite, and consistently high metabolic requirements brought about by their movement disorder. Approximately 79% of patients with XDP have experienced rapid weight loss since the onset of their disease. It is the aim of the researchers to assess the clinical usefulness of adding a nutritional supplement to the daily dietary needs of these patients. MINGO is a supplement consisting of local ingredients such as moringa, rice and mung beans, which can be added to any type of edible paste, food, and liquid. Mingo has gained popularity as an emergency food in disaster relief operations due to the ease of its preparation, its high nutritional value and relatively low price. For the last two years, it has also been used as an agent for nutritional build up in malnourished populations of children. This study will provide valuable information on whether patients with XDP can increase their weight by consuming MINGO, which will lead to improvements in the patients' medical care and wellbeing.
Interventions
DIETARY_SUPPLEMENTMINGO
6 20oz sachets will be taken daily by intervention group
Sponsors
Jose R. Reyes Memorial Medical Center
Massachusetts General Hospital
Sunshine Care Foundation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* Subject is above the age of 18 * Positive Diagnosis of XDP by movement disorder specialist/fellow * Subject with a yes response to a supportive home environment in the patient's most recent doctors monthly report * Subject are easily accessible to passable roads and pubic transportation
Exclusion criteria
* Subjects who have extreme movements that prevent staff from performing BMI or MUAC measurements * Subjects who have abnormal metabolic labs prior to the start of the study * Subjects who are taking pharmacological or medicinal supplements that may effect weight * Subjects who have been hospitalized within the last 2 weeks from the start of the trial * Subjects who have a Nasogastric tube or G-tube * Subjects with a history of other diseases that my effect weight (eg: hypo/hyperthyroidism)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | after baseline Body Mass Index, we will measure Body Mass Index every 2 weeks for 3 months | The investigators will first take a baseline Body Mass Index in kg/m\^2 for both control and intervention group, then subsequent measures every two weeks for 3 month duration of he trial. The goal of the investigators is to compare the net change in Body Mass Index in a 3 month period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| All Cause Mortality | 3 months (study close out) | number of deaths in both arms at the end of the study |
| Number of Hospitalizations Secondary to Infectious Causes | 3 months (study close out) | number of hospitalizations secondary to infections in both arms at the end of the study |
| Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | After baseline measurements, the investigators will measure the MUAC evey month for 3 months | It has been reported that the Mid Upper Arm Circumference is a good predictor for BMI. It will serve as an indirect measurement to the subjects' BMI |
Countries
Philippines
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| MINGO MINGO is a supplement consisting of local ingredients such as moringa, rice and mung beans, which can be added to any type of edible paste, food, and liquid. The experimental group is required to take 6 sachets per day for 12 weeks in addition to their regular diet. They are required to keep a daily food diary
MINGO: 6 20oz sachets will be taken daily by intervention group | 24 |
| Control The control group is required to eat their normal diet. They are also required to keep a daily food diary. | 26 |
| Total | 50 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 0 | 1 |
Baseline characteristics
| Characteristic | MINGO | Total | Control |
|---|---|---|---|
| Age, Continuous | 54.2 years STANDARD_DEVIATION 1.6 | 55.4 years STANDARD_DEVIATION 1.2 | 56.4 years STANDARD_DEVIATION 1.6 |
| Body Mass Index (BMI) | 18.9 kg/m^2 STANDARD_DEVIATION 0.6 | 18.5 kg/m^2 STANDARD_DEVIATION 0.4 | 18.2 kg/m^2 STANDARD_DEVIATION 0.4 |
| Burkes-Fahn-Marsden (BFM) | 44.1 units on a scale STANDARD_DEVIATION 5.3 | 42.1 units on a scale STANDARD_DEVIATION 3.5 | 40.2 units on a scale STANDARD_DEVIATION 4.8 |
| Duration of Illness | 9.4 years STANDARD_DEVIATION 1.1 | 8.8 years STANDARD_DEVIATION 0.8 | 8.2 years STANDARD_DEVIATION 1.1 |
| Eating Assessment Tool (EAT-10) | 20.0 units on a scale STANDARD_DEVIATION 2.3 | 19.9 units on a scale STANDARD_DEVIATION 1.7 | 19.9 units on a scale STANDARD_DEVIATION 2.6 |
| Mean Upper Arm Circumference (MUAC) | 26.5 cm STANDARD_DEVIATION 0.5 | 26.5 cm STANDARD_DEVIATION 0.4 | 26.5 cm STANDARD_DEVIATION 0.6 |
| MGH-Swallowing Screening Tool (MGH-SST) | 3.8 units on a scale STANDARD_DEVIATION 0.3 | 3.8 units on a scale STANDARD_DEVIATION 0.2 | 3.8 units on a scale STANDARD_DEVIATION 0.3 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 24 Participants | 50 Participants | 26 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 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 | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment Philippines | 24 participants | 50 participants | 26 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 24 Participants | 50 Participants | 26 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 24 | 1 / 26 |
| other Total, other adverse events | 4 / 24 | 1 / 26 |
| serious Total, serious adverse events | 0 / 24 | 0 / 26 |
Outcome results
Primary
The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale.
The investigators will first take a baseline Body Mass Index in kg/m\^2 for both control and intervention group, then subsequent measures every two weeks for 3 month duration of he trial. The goal of the investigators is to compare the net change in Body Mass Index in a 3 month period.
Time frame: after baseline Body Mass Index, we will measure Body Mass Index every 2 weeks for 3 months
Population: We proposed an intention to treat analysis, so regardless of whether an XDP subject took the prescribed # of MINGO packets or not we analyzed everyone who entered the trial
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 4 | 19.14 Kg/m^2 |
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 8 | 19.15 Kg/m^2 |
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 2 | 19.00 Kg/m^2 |
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 10 | 19.03 Kg/m^2 |
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 6 | 19.11 Kg/m^2 |
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 12 | 19.07 Kg/m^2 |
| MINGO | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | baseline | 18.86 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 12 | 17.95 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | baseline | 18.17 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 2 | 18.31 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 4 | 18.27 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 6 | 18.06 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 8 | 18.24 Kg/m^2 |
| Control | The Body Mass Index (BMI in kg/m^2) Using a Bathroom Weighing Scale. | week 10 | 18.05 Kg/m^2 |
p-value: 0.084Mixed Models Analysis
Secondary
All Cause Mortality
number of deaths in both arms at the end of the study
Time frame: 3 months (study close out)
Population: Everyone who enrolled were analyzed
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| MINGO | All Cause Mortality | 0 Participants |
| Control | All Cause Mortality | 1 Participants |
Secondary
Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure
It has been reported that the Mid Upper Arm Circumference is a good predictor for BMI. It will serve as an indirect measurement to the subjects' BMI
Time frame: After baseline measurements, the investigators will measure the MUAC evey month for 3 months
Population: All participants in the trial
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| MINGO | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | baseline | 26.54 cm |
| MINGO | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | month 1 | 26.93 cm |
| MINGO | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | month 2 | 26.96 cm |
| MINGO | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | month 3 | 26.69 cm |
| Control | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | month 3 | 26.62 cm |
| Control | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | baseline | 26.54 cm |
| Control | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | month 2 | 26.69 cm |
| Control | Mid Upper Arm Circumference (MUAC) in cm Using a Tailor's Tape Measure | month 1 | 26.58 cm |
Secondary
Number of Hospitalizations Secondary to Infectious Causes
number of hospitalizations secondary to infections in both arms at the end of the study
Time frame: 3 months (study close out)
Population: all participants were analyzed
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| MINGO | Number of Hospitalizations Secondary to Infectious Causes | 0 Participants |
| Control | Number of Hospitalizations Secondary to Infectious Causes | 0 Participants |