Trial · NCT05099640
A Study of PTC923 in Participants With Phenylketonuria
NCT05099640, Phase 3, Completed, PTC Therapeutics; results first posted 2024-01-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of PTC923 in Participants With Phenylketonuria. Trials, record NCT05099640. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05099640
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of PTC923 in Participants With Phenylketonuria
- NCT number
- NCT05099640
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 157 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Phenylketonuria
- Interventions
- PTC923, Placebo
- First posted
- 2021-10-29
- Last updated
- 2024-01-10
- Other registries' IDs
- eudract:2021-000474-29
- Results first posted
- 2024-01-10
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 5 and 6 (average of the 2-week period) | Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. Least square (LS) mean and standard error (SE) were calculated using mixed model repeated measures (MMRM) method. |
| Primary | Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 5 and 6 (average of the 2-week period) | Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. LS mean and SE were calculated using MMRM method. |
| Secondary | Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥600 μmol/L Who Achieved Phe Levels <600 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Weeks 5 and 6 (average of the 2-week period) | Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. |
| Secondary | Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥360 μmol/L Who Achieved Phe Levels <360 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Weeks 5 and 6 (average of the 2-week period) | Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. |
| Secondary | Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 (average of each 2-week period) | Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean levels at Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 were calculated as the average of blood Phe levels collected during the Week 1-2, Week 3-4, and Week 5-6 analysis visit windows, respectively. |
| Secondary | Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 (average of each 2-week period) | Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean levels at Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 were calculated as the average of blood Phe levels collected during the Week 1-2, Week 3-4, and Week 5-6 analysis visit windows, respectively. |
| Secondary | Part 1 Open-label Run-in Phase: Plasma Concentration of Tetrahydrobiopterin (BH4) and Sepiapterin | Predose, 0.5, 1, 2, 4, 6, 8, and 24 hours postdose at Day 1; 2 and 6 hours postdose at Day 14 | |
| Secondary | Part 2 Double-blind Phase: Plasma Concentration of BH4 and Sepiapterin | Predose and 4 hours postdose at Days 1, 14, 28, and 42 | |
| Secondary | Part 1 Open-label Run-in Phase: Area Under the Concentration-time Curve From Time 0 to 24 Hours Postdose (AUC0-24h) of Sepiapterin and BH4 Following the First Dose of Sepiapterin at 60 mg/kg | 0 to 24 hours postdose at Day 1 | |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Day 42 | An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs were considered: * Part 1 TEAEs, which included all AEs occurring after first dose in Part 1 but before first dose in Part 2; * Part 2 TEAEs, which included all AEs after first randomized dose in Part 2. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 5 and 6 (average of the 2-week period) | Full Analysis Set (FAS) included all participants who were randomized and received at least 1 dose of double-blind study drug in Part 2. Non-responders in Part 1 did not continue to Part 2 and were not included in the analysis. Here, 'Overall number of participants analyzed' = participants of FAS with Phe reduction from Baseline ≥30% during Part 1, who continued in Part 2. This outcome measure was pre-specified to collect data only for Part 2. | μmol/L | LEAST_SQUARES_MEAN |
| PRIMARY | Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 5 and 6 (average of the 2-week period) | FAS included all participants who were randomized and received at least 1 dose of double-blind study drug in Part 2. Non-responders in Part 1 did not continue to Part 2 and were not included in the analysis. Here, 'Overall number of participants analyzed' = participants of FAS with Phe reduction from Baseline ≥30% during Part 1, who continued in Part 2. This outcome measure was pre-specified to collect data only for Part 2. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥600 μmol/L Who Achieved Phe Levels <600 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Weeks 5 and 6 (average of the 2-week period) | FAS included all participants who were randomized and received at least 1 dose of double-blind study drug in Part 2. Non-responders in Part 1 did not continue to Part 2 and were not included in the analysis. Here, 'Overall number of participants analyzed' = participants of FAS with Phe reduction from baseline ≥30% during Part 1 and had Part 2 baseline Phe levels ≥600 μmol/L. This outcome measure was pre-specified to collect data only for Part 2. | percentage of participants | NUMBER |
| SECONDARY | Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥360 μmol/L Who Achieved Phe Levels <360 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Weeks 5 and 6 (average of the 2-week period) | FAS included all participants who were randomized and received at least 1 dose of double-blind study drug in Part 2. Non-responders in Part 1 did not continue to Part 2 and were not included in the analysis. Here, 'Overall number of participants analyzed' = participants of FAS with Phe reduction from baseline ≥30% during Part 1 and Part 2 baseline Phe levels ≥360 μmol/L. This outcome measure was pre-specified to collect data only for Part 2. | percentage of participants | NUMBER |
| SECONDARY | Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 (average of each 2-week period) | FAS included all participants who were randomized and received at least 1 dose of double-blind study drug in Part 2. Non-responders in Part 1 did not continue to Part 2 and were not included in the analysis. Here, 'Overall number of participants analyzed' = participants of FAS with Phe reduction from Baseline ≥30% during Part 1 and continued in Part 2. 'Number analyzed' = participants evaluable at specified timepoint. This outcome measure was pre-specified to collect data only for Part 2. | μmol/L | MEAN |
| SECONDARY | Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6 (average of each 2-week period) | FAS included all participants who were randomized and received at least 1 dose of double-blind study drug in Part 2. Non-responders in Part 1 did not continue to Part 2 and were not included in the analysis. Here, 'Overall number of participants analyzed' = participants of FAS with Phe reduction from Baseline ≥30% during Part 1 and continued in Part 2. 'Number analyzed' = participants evaluable at specified timepoint. This outcome measure was pre-specified to collect data only for Part 2. | percent change | MEAN |
| SECONDARY | Part 1 Open-label Run-in Phase: Plasma Concentration of Tetrahydrobiopterin (BH4) and Sepiapterin | Predose, 0.5, 1, 2, 4, 6, 8, and 24 hours postdose at Day 1; 2 and 6 hours postdose at Day 14 | Pharmacokinetic (PK) Analysis Set included all participants who had at least 1 measurable plasma concentration of sepiapterin or BH4. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint. | nanograms (ng)/milliliter (mL) | MEAN |
| SECONDARY | Part 2 Double-blind Phase: Plasma Concentration of BH4 and Sepiapterin | Predose and 4 hours postdose at Days 1, 14, 28, and 42 | PK Analysis Set included all participants who had at least 1 measurable plasma concentration of sepiapterin or BH4. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint. | ng/mL | MEAN |
| SECONDARY | Part 1 Open-label Run-in Phase: Area Under the Concentration-time Curve From Time 0 to 24 Hours Postdose (AUC0-24h) of Sepiapterin and BH4 Following the First Dose of Sepiapterin at 60 mg/kg | 0 to 24 hours postdose at Day 1 | PK Analysis Set included all participants who had at least 1 measurable plasma concentration of sepiapterin or BH4. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | hours*ng/mL | MEAN |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Day 42 | Safety analysis set included all participants who received at least 1 dose of study drug, including during Part 1. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group83
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Sepiapterin | -415.75 | Standard Error 24.066 | μmol/L | |
Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Placebo | -19.88 | Standard Error 24.223 | μmol/L | |
| Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Sepiapterin | -63.40 | Standard Error 3.537 | percent change | |
| Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Placebo | 0.82 | Standard Error 3.561 | percent change | |
| Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥600 μmol/L Who Achieved Phe Levels <600 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Sepiapterin | 92.9 | 95% Confidence Interval 76.50 to 99.12 | percentage of participants | |
| Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥600 μmol/L Who Achieved Phe Levels <600 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Placebo | 30.0 | 95% Confidence Interval 14.73 to 49.40 | percentage of participants | |
| Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥360 μmol/L Who Achieved Phe Levels <360 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Sepiapterin | 84.1 | 95% Confidence Interval 69.93 to 93.36 | percentage of participants | |
| Part 2 Double-blind Phase: Percentage of Participants With Baseline Phe Levels ≥360 μmol/L Who Achieved Phe Levels <360 μmol/L in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Placebo | 9.3 | 95% Confidence Interval 2.59 to 22.14 | percentage of participants | |
| Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Sepiapterin | Weeks 1 and 2 | -341.18 | Standard Deviation 226.178 | μmol/L |
| Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1 | Part 2: Placebo | Weeks 1 and 2 | -53.27 | Standard Deviation 174.461 | μmol/L |
1 to 10 of 83Source ClinicalTrials.gov
See all 83 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1: Open-label (14 Days) | STARTED | Part 1: Sepiapterin | 157 | |
| Part 1: Open-label (14 Days) | STARTED | Part 2: Sepiapterin | 0 | |
| Part 1: Open-label (14 Days) | STARTED | Part 2: Placebo | 0 | |
| Part 1: Open-label (14 Days) | Received at Least 1 Dose of Study Drug | Part 1: Sepiapterin | 157 | |
| Part 1: Open-label (14 Days) | Received at Least 1 Dose of Study Drug | Part 2: Sepiapterin | 0 | |
| Part 1: Open-label (14 Days) | Received at Least 1 Dose of Study Drug | Part 2: Placebo | 0 | |
| Part 1: Open-label (14 Days) | COMPLETED | Part 1: Sepiapterin | 111 | |
| Part 1: Open-label (14 Days) | COMPLETED | Part 2: Sepiapterin | 0 | |
| Part 1: Open-label (14 Days) | COMPLETED | Part 2: Placebo | 0 | |
| Part 1: Open-label (14 Days) | NOT COMPLETED | Part 1: Sepiapterin | 46 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events21
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Part 1: Sepiapterin | 8 | 157 | |
| other | Diarrhoea | Gastrointestinal disorders | Part 2: Sepiapterin | 4 | 56 | |
| other | Diarrhoea | Gastrointestinal disorders | Part 2: Placebo | 1 | 54 | |
| other | Upper respiratory tract infection | Infections and infestations | Part 1: Sepiapterin | 7 | 157 | |
| other | Upper respiratory tract infection | Infections and infestations | Part 2: Sepiapterin | 3 | 56 | |
| other | Upper respiratory tract infection | Infections and infestations | Part 2: Placebo | 1 | 54 | |
| other | Gastroenteritis | Infections and infestations | Part 1: Sepiapterin | 0 | 157 | |
| other | Gastroenteritis | Infections and infestations | Part 2: Sepiapterin | 3 | 56 | |
| other | Gastroenteritis | Infections and infestations | Part 2: Placebo | 3 | 54 | |
| other | Nasopharyngitis | Infections and infestations | Part 1: Sepiapterin | 0 | 157 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1: Sepiapterin | 0 | 157 |
| Total, all-cause mortality | Part 2: Sepiapterin | 0 | 56 |
| Total, all-cause mortality | Part 2: Placebo | 0 | 54 |
| Total, other adverse events | Part 1: Sepiapterin | 15 | 157 |
| Total, other adverse events | Part 2: Sepiapterin | 14 | 56 |
| Total, other adverse events | Part 2: Placebo | 11 | 54 |
| Total, serious adverse events | Part 1: Sepiapterin | 0 | 157 |
| Total, serious adverse events | Part 2: Sepiapterin | 0 | 56 |
| Total, serious adverse events | Part 2: Placebo | 0 | 54 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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GET https://conductscience.org/api/trials/v1/NCT05099640
GET https://conductscience.org/api/trials/v1/NCT05099640/results?page=2