Trial Outcomes & Findings for A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19) (NCT NCT04439071)

Last Updated: 2023-06-26

Results Overview

Respiratory improvement was defined as sustained peripheral oxygen saturation (SpO2) ≥94% on room air. Median time to respiratory improvement was estimated via the Kaplan-Meier product limit method.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

189 participants

Primary outcome timeframe

up to Day 28

Results posted on

2023-06-26

Participant Flow

Participant milestones

Participant milestones
Measure	PTC299 Participants received PTC299 at 200 milligrams (mg), administered orally, twice daily (BID) on Days 1 to 7, then at 50 mg administered orally, once daily (QD) on Days 8 to 14.	Placebo Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Overall Study STARTED	94	95
Overall Study Received at Least 1 Dose of Study Drug	92	95
Overall Study COMPLETED	80	77
Overall Study NOT COMPLETED	14	18

Reasons for withdrawal

Reasons for withdrawal
Measure	PTC299 Participants received PTC299 at 200 milligrams (mg), administered orally, twice daily (BID) on Days 1 to 7, then at 50 mg administered orally, once daily (QD) on Days 8 to 14.	Placebo Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Overall Study Lost to Follow-up	0	3
Overall Study Withdrawal by Subject	3	8
Overall Study Death	6	4
Overall Study Other than specified	3	3
Overall Study Randomized but not treated	2	0

Baseline Characteristics

A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	PTC299 n=92 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=95 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.	Total n=187 Participants Total of all reporting groups
Age, Continuous	52.0 years STANDARD_DEVIATION 12.44 • n=93 Participants	52.7 years STANDARD_DEVIATION 12.48 • n=4 Participants	52.3 years STANDARD_DEVIATION 12.44 • n=27 Participants
Sex: Female, Male Female	25 Participants n=93 Participants	27 Participants n=4 Participants	52 Participants n=27 Participants
Sex: Female, Male Male	67 Participants n=93 Participants	68 Participants n=4 Participants	135 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	53 Participants n=93 Participants	64 Participants n=4 Participants	117 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=93 Participants	26 Participants n=4 Participants	60 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	5 Participants n=93 Participants	5 Participants n=4 Participants	10 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	4 Participants n=93 Participants	6 Participants n=4 Participants	10 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) Black or African American	6 Participants n=93 Participants	1 Participants n=4 Participants	7 Participants n=27 Participants
Race (NIH/OMB) White	69 Participants n=93 Participants	69 Participants n=4 Participants	138 Participants n=27 Participants
Race (NIH/OMB) More than one race	12 Participants n=93 Participants	19 Participants n=4 Participants	31 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants

PRIMARY outcome

Timeframe: up to Day 28

Population: Intent-to-treat (ITT) population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	PTC299 n=73 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=76 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Time From Randomization to Respiratory Improvement	10.0 days Interval 6.0 to 15.0	10.0 days Interval 8.0 to 13.0

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants.

Number of participants requiring invasive ventilation at any time during the study were reported.

Outcome measures

Outcome measures
Measure	PTC299 n=94 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=95 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Number of Participants Requiring Invasive Ventilation	16 Participants	11 Participants

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Number of participants requiring supplemental oxygen or non-invasive ventilation at any point during the study in participants who did not require supplemental oxygen at baseline were reported.

Outcome measures

Outcome measures
Measure	PTC299 n=24 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=22 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Number of Participants Requiring Supplemental Oxygen or Non-Invasive Ventilation in Participants Who Did Not Require Supplemental Oxygen at Baseline	20 Participants	19 Participants

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Defervescence was defined as body temperature of \<37.6° C axilla, \<38.0° C oral, or \<38.6° C tympanic or rectal without taking any antipyretic treatment and sustained until discharge or Day 28. Median time to defervescence was estimated via the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	PTC299 n=31 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=22 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Time From Randomization to Defervescence in Participants Presenting With Fever at Enrollment (Temperature of ≥37.6℃ Axilla, ≥38.0℃ Oral, or ≥38.6°C Tympanic or Rectal)	7.0 days Interval 5.0 to 28.0	18.0 days Interval 4.0 to Due to smaller number of participants with an event, upper limit of 95% confidence interval (CI) could not be calculated.

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Median time to respiratory rate in participants who had abnormal respiratory rate at baseline was estimated via the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	PTC299 n=57 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=62 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Time From Randomization to Respiratory Rate ≤ 24 Breaths Per Minute on Room Air	7.0 days Interval 5.0 to 11.0	8.0 days Interval 6.0 to 11.0

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Cough was rated on a scale of severe, moderate, mild, absent, in those with cough at enrollment rated severe or moderate. Median time to cough reported as mild or absent was estimated via the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	PTC299 n=34 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=34 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Time From Randomization to Cough Reported as Mild or Absent	3.0 days Interval 3.0 to 4.0	5.0 days Interval 3.0 to 7.0

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Dyspnea was rated on a scale of severe, moderate, mild, absent, in those with dyspnea at enrollment rated as severe or moderate. Median time to dyspnea reported as mild or absent was estimated via the Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	PTC299 n=31 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=35 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Time From Randomization to Dyspnea Reported as Mild or Absent	6.0 days Interval 3.0 to 9.0	5.0 days Interval 4.0 to 7.0

SECONDARY outcome

Timeframe: Baseline, Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Cytokines included Granulocyte Colony Stimulating factor; Interleukin 10, 17, 2, 6, 7; Macrophage Inflammatory Protein 1 Alpha; Monocyte Chemotactic Protein 1; and Tumor Necrosis Factor.

Outcome measures

Outcome measures
Measure	PTC299 n=66 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=64 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Cytokine Levels at Day 28 Granulocyte Colony Stimulating Factor	-0.423 nanograms (ng)/liter (L) Standard Deviation 0.4431	-0.467 nanograms (ng)/liter (L) Standard Deviation 0.4750
Change From Baseline in Cytokine Levels at Day 28 Interleukin 10	-0.367 nanograms (ng)/liter (L) Standard Deviation 0.4655	-0.491 nanograms (ng)/liter (L) Standard Deviation 0.4003
Change From Baseline in Cytokine Levels at Day 28 Interleukin 17	0.018 nanograms (ng)/liter (L) Standard Deviation 0.1447	0.002 nanograms (ng)/liter (L) Standard Deviation 0.1425
Change From Baseline in Cytokine Levels at Day 28 Interleukin 2	0.021 nanograms (ng)/liter (L) Standard Deviation 0.0868	0.022 nanograms (ng)/liter (L) Standard Deviation 0.1448
Change From Baseline in Cytokine Levels at Day 28 Interleukin 6	-0.375 nanograms (ng)/liter (L) Standard Deviation 0.5274	-0.442 nanograms (ng)/liter (L) Standard Deviation 0.5062
Change From Baseline in Cytokine Levels at Day 28 Interleukin 7	-0.025 nanograms (ng)/liter (L) Standard Deviation 0.1233	-0.035 nanograms (ng)/liter (L) Standard Deviation 0.1556
Change From Baseline in Cytokine Levels at Day 28 Macrophage Inflammatory Protein 1 Alpha	0.072 nanograms (ng)/liter (L) Standard Deviation 0.2459	0.044 nanograms (ng)/liter (L) Standard Deviation 0.1888
Change From Baseline in Cytokine Levels at Day 28 Monocyte Chemotactic Protein 1	-0.011 nanograms (ng)/liter (L) Standard Deviation 0.4077	0.018 nanograms (ng)/liter (L) Standard Deviation 0.3804
Change From Baseline in Cytokine Levels at Day 28 Tumor Necrosis Factor	-0.015 nanograms (ng)/liter (L) Standard Deviation 0.1509	-0.027 nanograms (ng)/liter (L) Standard Deviation 0.1666

SECONDARY outcome

Timeframe: Baseline, Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	PTC299 n=51 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=46 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Level of Acute Phase Protein (C Reactive Protein) at Day 28	-1.027 mg/L Standard Deviation 0.5966	-1.111 mg/L Standard Deviation 0.5633

SECONDARY outcome

Timeframe: Baseline, Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	PTC299 n=48 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=45 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Level of Acute Phase Protein (D-Dimer) at Day 28	0.029 micrograms (µg)/L D-dimer units (DDU) Standard Deviation 0.9278	-0.121 micrograms (µg)/L D-dimer units (DDU) Standard Deviation 0.9392

SECONDARY outcome

Timeframe: Baseline, Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	PTC299 n=47 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=44 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Level of Acute Phase Protein (Ferritin) at Day 28	-0.410 picomoles (pmol)/L Standard Deviation 0.3096	-0.497 picomoles (pmol)/L Standard Deviation 0.3162

SECONDARY outcome

Timeframe: Baseline, Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable for specified category.

Outcome measures

Outcome measures
Measure	PTC299 n=32 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=18 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Level of Acute Phase Proteins (Troponin I and Troponin T) at Day 28 Troponin I	0.080 µg/L Standard Deviation 1.5879	-0.036 µg/L Standard Deviation 0.2128
Change From Baseline in Level of Acute Phase Proteins (Troponin I and Troponin T) at Day 28 Troponin T	0.001 µg/L Standard Deviation 0.0015	0.061 µg/L Standard Deviation 0.1638

SECONDARY outcome

Timeframe: up to Day 28

Number of participants who returned to normal range CBC were reported. CBC included red blood cell (RBC), hemoglobin (HGB), white blood cell (WBC), and Platelets.

Outcome measures

Outcome measures
Measure	PTC299 n=27 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=26 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Number of Participants With Normalization of Complete Blood Count (CBC) Who Had CBC Out of Range at Baseline RBC	7 Participants	13 Participants
Number of Participants With Normalization of Complete Blood Count (CBC) Who Had CBC Out of Range at Baseline HGB	9 Participants	13 Participants
Number of Participants With Normalization of Complete Blood Count (CBC) Who Had CBC Out of Range at Baseline WBC	22 Participants	17 Participants
Number of Participants With Normalization of Complete Blood Count (CBC) Who Had CBC Out of Range at Baseline Platelets	21 Participants	19 Participants

SECONDARY outcome

Timeframe: Baseline, Day 28

Outcome measures

Outcome measures
Measure	PTC299 n=50 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=49 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Viral Load at Day 28: SARS-CoV-2 Immunoglobulin A (IgA) Antibody Ratio and SARS-CoV-2 Immunoglobulin G (IgG) Antibody Ratio SARS-CoV-2 IgA Antibody Ratio	0.003 ratio Standard Deviation 0.6387	0.165 ratio Standard Deviation 0.5122
Change From Baseline in Viral Load at Day 28: SARS-CoV-2 Immunoglobulin A (IgA) Antibody Ratio and SARS-CoV-2 Immunoglobulin G (IgG) Antibody Ratio SARS-CoV-2 IgG Antibody Ratio	0.890 ratio Standard Deviation 0.6846	0.874 ratio Standard Deviation 0.7604

SECONDARY outcome

Timeframe: Baseline, Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	PTC299 n=45 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=47 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Viral Load at Day 28: SARS-CoV-2 IgM Antibody Absorbance	0.000 absorbance (Abs) Standard Deviation 0.4691	0.127 absorbance (Abs) Standard Deviation 0.4218

SECONDARY outcome

Timeframe: Baseline, Day 28

Outcome measures

Outcome measures
Measure	PTC299 n=4 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=2 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Change From Baseline in Viral Load at Day 28: SARS-CoV2 v2, SARS-CoV2 v2 Nasopharyngeal Swab (NPsw), and Severe Acute Resp Syndrome Coronavirus 2 SARS-CoV2 v2	—	-0.345 copies/mL Standard Deviation 0.2235
Change From Baseline in Viral Load at Day 28: SARS-CoV2 v2, SARS-CoV2 v2 Nasopharyngeal Swab (NPsw), and Severe Acute Resp Syndrome Coronavirus 2 SARS-CoV2 v2 NPsw	-1.201 copies/mL Standard Deviation 0.7503	1.532 copies/mL
Change From Baseline in Viral Load at Day 28: SARS-CoV2 v2, SARS-CoV2 v2 Nasopharyngeal Swab (NPsw), and Severe Acute Resp Syndrome Coronavirus 2 Severe Acute Resp Syndrome Coronavirus 2	-0.432 copies/mL	—

SECONDARY outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	PTC299 n=77 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=83 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Duration of Hospitalization	8.1 days Standard Deviation 4.87	9.3 days Standard Deviation 5.49

SECONDARY outcome

Timeframe: Day 28

Population: Safety population included all randomized participants who received at least 1 dose of study drug.

Mortality was defined as a death event occurring at anytime before the specific date, after the first dose has been received.

Outcome measures

Outcome measures
Measure	PTC299 n=92 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=95 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Number of Mortalities at Day 28	6 Participants	4 Participants

SECONDARY outcome

Timeframe: up to Day 60

Population: Safety population included all randomized participants who received at least 1 dose of study drug.

An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TEAEs were defined as any AEs that occurred on or after the first study treatment through 30 days after the last dose, or any AEs occurring before the first study treatment but worsening during the treatment through 30 days after the last dose. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Outcome measures

Outcome measures
Measure	PTC299 n=92 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=95 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	52 Participants	67 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: up to Day 28

Population: ITT population included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Respiratory improvement was defined as SpO2 ≥94% on room air. Median time to respiratory improvement was estimated via the Kaplan-Meier product limit method.

Outcome measures

Outcome measures
Measure	PTC299 n=13 Participants Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=10 Participants Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Time From Randomization to Respiratory Improvement Where Symptom Onset Occurred ≤5 Days	10.0 days Interval 4.0 to 28.0	28.0 days Interval 7.0 to Due to smaller number of participants with an event, upper limit of 95% CI could not be calculated.

Adverse Events

PTC299

Serious events: 21 serious events

Other events: 50 other events

Deaths: 9 deaths

Placebo

Serious events: 24 serious events

Other events: 63 other events

Deaths: 4 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	PTC299 n=92 participants at risk Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=95 participants at risk Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Investigations Alanine aminotransferase increased	15.2% 14/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	16.8% 16/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Aspartate aminotransferase increased	13.0% 12/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	12.6% 12/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Gamma-glutamyltransferase increased	4.3% 4/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	6.3% 6/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Gastrointestinal disorders Constipation	10.9% 10/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	12.6% 12/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Gastrointestinal disorders Diarrhoea	3.3% 3/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	5.3% 5/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Blood and lymphatic system disorders Anaemia	8.7% 8/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	3.2% 3/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Blood and lymphatic system disorders Lymphopenia	4.3% 4/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	6.3% 6/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Metabolism and nutrition disorders Hyperglycaemia	6.5% 6/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	2.1% 2/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Vascular disorders Hypertension	7.6% 7/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	2.1% 2/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Psychiatric disorders Anxiety	2.2% 2/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	5.3% 5/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Psychiatric disorders Insomnia	2.2% 2/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	6.3% 6/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
General disorders Condition aggravated	3.3% 3/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	9.5% 9/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Nervous system disorders Headache	2.2% 2/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	5.3% 5/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.

Additional Information

Patient Advocacy

PTC Therapeutics, Inc.

Phone: 1-866-562-4620

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
Publication restrictions are in place

Restriction type: OTHER

Measure	PTC299 n=92 participants at risk Participants received PTC299 at 200 mg, administered orally, BID on Days 1 to 7, then at 50 mg administered orally, QD on Days 8 to 14.	Placebo n=95 participants at risk Participants received PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
Respiratory, thoracic and mediastinal disorders Pneumothorax	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Pneumothorax spontaneous	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	3.2% 3/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Septic shock	4.3% 4/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Sepsis	2.2% 2/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Pneumonia acinetobacter	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Pneumonia bacterial	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Pulmonary sepsis	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Bacteraemia	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Dengue fever	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Pneumonia	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Infections and infestations Urinary tract infection bacterial	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Renal and urinary disorders Acute kidney injury	4.3% 4/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	2.1% 2/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Cardiac disorders Cardio-respiratory arrest	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Cardiac disorders Cardiogenic shock	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Cardiac disorders Right ventricular dysfunction	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Cardiac disorders Supraventricular tachycardia	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Cardiac disorders Acute myocardial infarction	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Cardiac disorders Cardiac arrest	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Alanine aminotransferase increased	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Aspartate aminotransferase increased	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	2.1% 2/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Blood alkaline phosphatase increased	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Haemoglobin decreased	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Investigations Blood bilirubin increased	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Vascular disorders Hypertension	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Vascular disorders Peripheral ischaemia	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Blood and lymphatic system disorders Lymphopenia	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	2.1% 2/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
General disorders Disease progression	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	2.1% 2/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Hepatobiliary disorders Hepatic failure	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Psychiatric disorders Psychotic disorder	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Metabolism and nutrition disorders Diabetic ketoacidosis	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Joint swelling	0.00% 0/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	1.1% 1/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Respiratory failure	10.9% 10/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	8.4% 8/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	3.3% 3/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	3.2% 3/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Organising pneumonia	1.1% 1/92 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.	0.00% 0/95 • up to Day 60 Safety population included all randomized participants who received at least 1 dose of study drug.