Friedreich Ataxia
Conditions
Brief summary
The purpose of this study is to document the clinical effects of bupropion and citalopram in a single subject with Friedreich Ataxia.
Detailed description
This is a double blind study with a single subject to objectively document the clinical effects of bupropion and citalopram in a single patient with Friedreich Ataxia (FA), whose disorder symptoms have improved with the use of these medications.
Interventions
DRUGbupropion & Citalopram
DRUGBupropion & Placebo
DRUGPlacebo & Citalopram
DRUGPlacebo & Placebo
Sponsors
University of Colorado, Denver
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
SUPPORTIVE_CARE
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
Inclusion criteria
* Friedreich Ataxia
Exclusion criteria
* Unable to provide Informed Consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| International Cooperative Ataxia Rating Scale (ICARS) | Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 weeks (bupropion), and 20 weeks (citalopram + bupropion) | The ICARS is a 19 item rating scale of ataxia with the total score ranging from 0 to 100. A score of 0 means normal and higher scores represent worsened disease. |
| Friedreich Ataxia Rating Scale (FARS) | Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 (bupropion), and 20 weeks (citalopram + bupropion) | A rating scale developed for Friedreich ataxia in evaluation of ataxia. Score range from 0-159 with a score of 0 meaning normal and greater scores indicating worsened disease. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Comparison of FARS and ICARS | Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 (bupropion), and 20 weeks (citalopram + bupropion) | Differences between FARS - ICARS at each treatment interval |
Other
| Measure | Time frame | Description |
|---|---|---|
| Hamilton Depression Rating Scale | Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 (bupropion), and 20 weeks (citalopram + bupropion) | The Hamilton Depression Rating Scale is a 21 item questionnaire scored each item on a scale of 0 to 3 or 5. The max score is 66. Higher scores indicate worsened depression. All items are summed together to give a total score. A total score of 0-7 is considered normal, while total scores greater than 20 are indicative of moderate or greater depression. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Study Participant the participant received all intervention combinations in the following order: bupropion & Citalopram, then Bupropion & Placebo, then Placebo & Citalopram, then Placebo & Placebo | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Study Participant |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 65 Years STANDARD_DEVIATION 0 |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 0 / 1 | 0 / 1 | 0 / 1 | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 | 0 / 1 | 0 / 1 | 0 / 1 |
Outcome results
Primary
Friedreich Ataxia Rating Scale (FARS)
A rating scale developed for Friedreich ataxia in evaluation of ataxia. Score range from 0-159 with a score of 0 meaning normal and greater scores indicating worsened disease.
Time frame: Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 (bupropion), and 20 weeks (citalopram + bupropion)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Bupropion & Citalopram | Friedreich Ataxia Rating Scale (FARS) | 43 points |
| Bupropion & Placebo | Friedreich Ataxia Rating Scale (FARS) | 41 points |
| Placebo & Citalopram | Friedreich Ataxia Rating Scale (FARS) | 47 points |
| Placebo & Placebo | Friedreich Ataxia Rating Scale (FARS) | 42 points |
| Baseline | Friedreich Ataxia Rating Scale (FARS) | 45 points |
Primary
International Cooperative Ataxia Rating Scale (ICARS)
The ICARS is a 19 item rating scale of ataxia with the total score ranging from 0 to 100. A score of 0 means normal and higher scores represent worsened disease.
Time frame: Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 weeks (bupropion), and 20 weeks (citalopram + bupropion)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Bupropion & Citalopram | International Cooperative Ataxia Rating Scale (ICARS) | 39 units |
| Bupropion & Placebo | International Cooperative Ataxia Rating Scale (ICARS) | 30 units |
| Placebo & Citalopram | International Cooperative Ataxia Rating Scale (ICARS) | 47 units |
| Placebo & Placebo | International Cooperative Ataxia Rating Scale (ICARS) | 46 units |
| Baseline | International Cooperative Ataxia Rating Scale (ICARS) | 42 units |
Secondary
Comparison of FARS and ICARS
Differences between FARS - ICARS at each treatment interval
Time frame: Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 (bupropion), and 20 weeks (citalopram + bupropion)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Bupropion & Citalopram | Comparison of FARS and ICARS | 1 points |
| Bupropion & Placebo | Comparison of FARS and ICARS | -6 points |
| Placebo & Citalopram | Comparison of FARS and ICARS | 1 points |
| Placebo & Placebo | Comparison of FARS and ICARS | 12 points |
| Baseline | Comparison of FARS and ICARS | 6 points |
Other Pre-specified
Hamilton Depression Rating Scale
The Hamilton Depression Rating Scale is a 21 item questionnaire scored each item on a scale of 0 to 3 or 5. The max score is 66. Higher scores indicate worsened depression. All items are summed together to give a total score. A total score of 0-7 is considered normal, while total scores greater than 20 are indicative of moderate or greater depression.
Time frame: Assessements are performed in 5 different states in a single patient: baseline (Bupropion 100mg and Citalopram 20mg - unblinded), then blinded at 5 weeks (citalopram), 10 weeks (placebo), 15 (bupropion), and 20 weeks (citalopram + bupropion)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Bupropion & Citalopram | Hamilton Depression Rating Scale | 7 units on a scale |
| Bupropion & Placebo | Hamilton Depression Rating Scale | 10 units on a scale |
| Placebo & Citalopram | Hamilton Depression Rating Scale | 2 units on a scale |
| Placebo & Placebo | Hamilton Depression Rating Scale | 3 units on a scale |
| Baseline | Hamilton Depression Rating Scale | 4 units on a scale |