Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Intra-subject Change in Intact Protein Intake |
Intra-subject change in intact protein intake will be assessed by participant-reported 3-day dietary records. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Primary |
Intra-subject Change in Medical Food Protein Intake |
Intra-subject change in medical food protein intake will be assessed by participant-reported 3-day dietary records. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Primary |
Sub study: Intra-subject change in intact protein and medical food protein intake. |
Intra-subject change in medical food protein intake will be assessed by participant-reported 3-day dietary records. |
12 Months Post-Response through additional five years (6 years post-response) |
|
Secondary |
Change in Average Intact Protein Intake |
Average intact protein intake will be determined by participant-reported 3-day dietary records. |
Baseline, Response (up to 60 days) 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Days From Palynziq Initiation to Response |
The average length of time (in days) from Palynziq initiation until response, as defined by three consecutive plasma Phe levels lower than the upper limit of the recommended treatment range (<360 µmol/L) and a significant increase in intact protein intake (at least 60 percent of the dietary reference intake (DRI) or a two-fold increase from baseline diet prescription), will be determined. |
Baseline up to Response (up to 60 days) |
|
Secondary |
Days From Palynziq Initiation to Consumption of Dietary Reference Intake for Intact Protein |
The average length of time (in days) from Palynziq initiation it takes for participants to consume the dietary reference intake for intact protein (46g females, 56g males). |
Baseline up to 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in Neuro-QOL - Cognitive Function - Short Form Score |
The Neuro-QOL Cognitive Function Short Form includes 8 items asking participants about their cognition during the past 7 days. Responses to statements of if cognition problems are occurring are given on a scale from 1 to 5 where 1 = very often and 5 = never. The amount of difficulty experienced from daily life tasks are responded to on a scale from 1 to 5 as 1 = cannot do task and 5 = no difficulty. Scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent in experiencing more cognition problems than the average person. |
Baseline, Response (up to 60 days) 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in Neuro-QOL Sleep Disturbance - Short Form Score |
The Neuro-QOL Sleep Disturbance Short Form includes 8 items asking participants about their sleep during the past 7 days. Responses to statements of if sleep problems are occurring are given on a scale from 1 to 5 where 1 = never and 5 = always. Scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent in experiencing more sleep problems than the average person. |
Baseline, Response (up to 60 days) 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in The Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health Score |
The PROMIS Global Health instrument includes 10 items asking participants about their health and quality of life. Responses to items are given on a scale from 1 to 5 where 1 = poor and 5 = excellent. Scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent in experiencing greater quality of life than the average person. |
Baseline, Response (up to 60 days) 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in PROMIS-29 Anxiety and Depression Score |
The PROMIS-29 instrument includes 8 items asking participants about anxiety and depression. Responses to are given on a scale from 1 to 5 where 1 = never and 5 = always. The total summed score from these items ranges from 8 to 40 and higher scores indicate greater anxiety and depression. |
Baseline, Response (up to 60 days) 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in PROMIS Emotional Support Score |
The PROMIS Emotional Support instrument includes 16 items asking participants about forms of emotional support they have available. Responses to items are given on a scale from 1 to 5 where 1 = never and 5 = always. Raw scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent has greater emotional support than the average person. |
Baseline, Response (up to 60 days) 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in Plasma Neurotransmitters |
Intra-subject change in neurotransmitter analysis will be assessed using fasting blood samples. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in Plasma Amino Acids |
Intra-subject change in plasma amino acids will be assessed using fasting blood samples. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Change in Essential Fatty Acids |
Intra-subject change in essential acids will be assessed using fasting blood samples. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Intra-subject Change in Bone Mineral Density |
Bone mineral density will be assessed with dual-energy x-ray absorptiometry (DEXA) |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Intra-subject Change in Percent Fat Body Mass |
Percent fat body mass will be assessed with DEXA. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Intra-subject Change in Percent Lean Body Mass |
Percent lean body mass will be assessed with DEXA. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Intra-subject Change in Resting Energy Expenditure |
Resting energy expenditure (kilocalories/day) from Palynziq initiation through 12 months post-response will be assessed using indirect calorimetry and self-reported activity level. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Intra-subject Change in Grip Strength |
Muscle strength will be assessed using a dynamometer, which will measure grip strength on each hand. |
Baseline through 12 Months Post-Response (up to 14 months after baseline) |
|
Secondary |
Sub-Study: Change in Plasma Neurotransmitters |
Intra-subject change in neurotransmitter analysis will be assessed using fasting blood samples. |
12 Months Post-Response through additional five years (6 years post-response) |
|
Secondary |
Sub-Study: Change in Neuro-QOL - Cognitive Function - Short Form Score |
The Neuro-QOL Cognitive Function Short Form includes 8 items asking participants about their cognition during the past 7 days. Responses to statements of if cognition problems are occurring are given on a scale from 1 to 5 where 1 = very often and 5 = never. The amount of difficulty experienced from daily life tasks are responded to on a scale from 1 to 5 as 1 = cannot do task and 5 = no difficulty. Scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent in experiencing more cognition problems than the average person. |
12 Months Post-Response through additional five years (6 years post-response) |
|
Secondary |
Sub-Study: Change in Neuro-QOL Sleep Disturbance - Short Form Score |
The Neuro-QOL Sleep Disturbance Short Form includes 8 items asking participants about their sleep during the past 7 days. Responses to statements of if sleep problems are occurring are given on a scale from 1 to 5 where 1 = never and 5 = always. Scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent in experiencing more sleep problems than the average person. |
12 Months Post-Response through additional five years (6 years post-response) |
|
Secondary |
Sub-Study: Change in The Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health Score |
The PROMIS Global Health instrument includes 10 items asking participants about their health and quality of life. Responses to items are given on a scale from 1 to 5 where 1 = poor and 5 = excellent. Scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent in experiencing greater quality of life than the average person. |
12 Months Post-Response through additional five years (6 years post-response) |
|
Secondary |
Sub-Study: Change in PROMIS-29 Anxiety and Depression Score |
The PROMIS-29 instrument includes 8 items asking participants about anxiety and depression. Responses to are given on a scale from 1 to 5 where 1 = never and 5 = always. The total summed score from these items ranges from 8 to 40 and higher scores indicate greater anxiety and depression. |
12 Months Post-Response through additional five years (6 years post-response) |
|
Secondary |
Sub-Study: Change in PROMIS Emotional Support Score |
The PROMIS Emotional Support instrument includes 16 items asking participants about forms of emotional support they have available. Responses to items are given on a scale from 1 to 5 where 1 = never and 5 = always. Raw scores are scaled to a T-score with a mean of 50 and a standard deviation of 10. Scores higher than 50 indicate that the respondent has greater emotional support than the average person. |
12 Months Post-Response through additional five years (6 years post-response) |
|