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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04439071




Registration number
NCT04439071
Ethics application status
Date submitted
17/06/2020
Date registered
19/06/2020
Date last updated
26/06/2023

Titles & IDs
Public title
A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
Scientific title
Evaluation of the Efficacy and Safety of PTC299 in Hospitalized Subjects With COVID-19 (FITE19)
Secondary ID [1] 0 0
2020-001872-13
Secondary ID [2] 0 0
PTC299-VIR-015-COV19
Universal Trial Number (UTN)
Trial acronym
FITE19
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pneumonia 0 0
COVID-19 0 0
Coronavirus 0 0
Condition category
Condition code
Infection 0 0 0 0
Other infectious diseases
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PTC299
Other interventions - SOC
Treatment: Drugs - Placebo

Experimental: PTC299 + Standard of Care (SOC) - Participants will receive 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.
SOC will also be administered according to local, written policies or guidelines.

Placebo Comparator: Placebo + SOC - Participants will receive PTC299-matching placebo administered orally, BID on Days 1 to 7, then administered orally, QD on Days 8 to 14.
SOC will also be administered according to local, written policies or guidelines.


Treatment: Drugs: PTC299
Oral tablets

Other interventions: SOC
As defined per local written policies or guidelines.

Treatment: Drugs: Placebo
Oral tablets

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time From Randomization to Respiratory Improvement
Timepoint [1] 0 0
up to Day 28
Secondary outcome [1] 0 0
Number of Participants Requiring Invasive Ventilation
Timepoint [1] 0 0
up to Day 28
Secondary outcome [2] 0 0
Number of Participants Requiring Supplemental Oxygen or Non-Invasive Ventilation in Participants Who Did Not Require Supplemental Oxygen at Baseline
Timepoint [2] 0 0
up to Day 28
Secondary outcome [3] 0 0
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)
Timepoint [3] 0 0
up to Day 28
Secondary outcome [4] 0 0
Time From Randomization to Respiratory Rate = 24 Breaths Per Minute on Room Air
Timepoint [4] 0 0
up to Day 28
Secondary outcome [5] 0 0
Time From Randomization to Cough Reported as Mild or Absent
Timepoint [5] 0 0
up to Day 28
Secondary outcome [6] 0 0
Time From Randomization to Dyspnea Reported as Mild or Absent
Timepoint [6] 0 0
up to Day 28
Secondary outcome [7] 0 0
Change From Baseline in Cytokine Levels at Day 28
Timepoint [7] 0 0
Baseline, Day 28
Secondary outcome [8] 0 0
Change From Baseline in Level of Acute Phase Protein (C Reactive Protein) at Day 28
Timepoint [8] 0 0
Baseline, Day 28
Secondary outcome [9] 0 0
Change From Baseline in Level of Acute Phase Protein (D-Dimer) at Day 28
Timepoint [9] 0 0
Baseline, Day 28
Secondary outcome [10] 0 0
Change From Baseline in Level of Acute Phase Protein (Ferritin) at Day 28
Timepoint [10] 0 0
Baseline, Day 28
Secondary outcome [11] 0 0
Change From Baseline in Level of Acute Phase Proteins (Troponin I and Troponin T) at Day 28
Timepoint [11] 0 0
Baseline, Day 28
Secondary outcome [12] 0 0
Number of Participants With Normalization of Complete Blood Count (CBC) Who Had CBC Out of Range at Baseline
Timepoint [12] 0 0
up to Day 28
Secondary outcome [13] 0 0
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
Timepoint [13] 0 0
Baseline, Day 28
Secondary outcome [14] 0 0
Change From Baseline in Viral Load at Day 28: SARS-CoV-2 IgM Antibody Absorbance
Timepoint [14] 0 0
Baseline, Day 28
Secondary outcome [15] 0 0
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
Timepoint [15] 0 0
Baseline, Day 28
Secondary outcome [16] 0 0
Duration of Hospitalization
Timepoint [16] 0 0
up to Day 28
Secondary outcome [17] 0 0
Number of Mortalities at Day 28
Timepoint [17] 0 0
Day 28
Secondary outcome [18] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Timepoint [18] 0 0
up to Day 60

Eligibility
Key inclusion criteria
- Signed and dated informed consent document(s).

- Agrees to the collection of nasopharyngeal swabs and venous blood and all other
protocol-specified procedures.

- Male or non-pregnant female adult =18 years of age at time of enrollment.

- Hospitalized and has laboratory-confirmed infection with severe acute respiratory
syndrome coronavirus 2 (SARS-CoV-2).

- Symptom onset was =10 days prior to screening.

- Has oxygen saturation SpO2 <94% on room air.

- Has at least one of a respiratory rate >24 breaths/minute or cough.

- Lung involvement as confirmed by radiographic infiltrates observed on imaging (chest
X-ray, computed tomography (CT) scan, or an equivalent test).

- Women of childbearing potential (as defined in [CTFG 2014]) must have a negative
pregnancy test at screening and agree to abstinence or the use at least one of the
following highly effective forms of contraception (with a failure rate of <1% per year
when used consistently and correctly). Contraception or abstinence must be continued
for the duration of the study following discharge from the hospital, and for up to 50
days after the last dose of study drug:

i) combined (estrogen and progestogen containing) hormonal contraception associated
with inhibition of ovulation: oral, intravaginal, and transdermal ii) progestogen-only
hormonal contraception associated with inhibition of ovulation: oral, injectable, and
implantable iii) intrauterine device iv) intrauterine hormone-releasing system v)
vasectomized partner with confirmed azoospermia All females will be considered of
childbearing potential unless they are postmenopausal (at least 12 months consecutive
amenorrhea in the appropriate age group without other known or suspected cause) or
have been sterilized surgically (for example, bilateral tubal ligation, hysterectomy,
bilateral oophorectomy).

- Men sexually active with women of childbearing potential who have not had a vasectomy
must agree to use a barrier method of birth control during the study following
discharge from the hospital and for up to 50 days after the last dose of study drug.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Requires mechanical ventilation.

- Current participation in any other interventional study.

- Alanine transaminase/aspartate transaminase levels =3 times the upper limit of normal
(×ULN) or total bilirubin (Tbili) =2×ULN.

- Lymphocyte count <500 lymphocytes/microliter (µL) or hemoglobin <11 grams/deciliter
(g/dL).

- Stage 4 severe chronic kidney disease or requiring dialysis (that is, estimated
glomerular filtration rate <30).

- Any other condition, that in the opinion of the Investigator, may be cause to exclude
the participant from the study.

- Use of steroids (except dexamethasone), sensitive CYP2D6 substrates, CYP2C inducers,
IL-6 neutralizing antibodies, IL-6 receptor inhibitors, or any investigational
therapy.

- Pregnancy or breast feeding.

- Anticipated transfer to another hospital which is not a study site within 72 hours.

- Known allergy to PTC299 or excipients.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Westmead Hospital - Westmead
Recruitment hospital [2] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [3] 0 0
Sunshine Hospital - St. Albans
Recruitment postcode(s) [1] 0 0
02145 - Westmead
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
03021 - St. Albans
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
South Carolina
Country [7] 0 0
Belgium
State/province [7] 0 0
Brussels
Country [8] 0 0
Belgium
State/province [8] 0 0
Ottignies
Country [9] 0 0
Brazil
State/province [9] 0 0
MG
Country [10] 0 0
Brazil
State/province [10] 0 0
RS
Country [11] 0 0
Brazil
State/province [11] 0 0
SC
Country [12] 0 0
Brazil
State/province [12] 0 0
SP
Country [13] 0 0
Colombia
State/province [13] 0 0
Bogotá
Country [14] 0 0
Colombia
State/province [14] 0 0
Rionegro
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
Mexico
State/province [16] 0 0
Guanajuato
Country [17] 0 0
Mexico
State/province [17] 0 0
Nuevo León
Country [18] 0 0
Mexico
State/province [18] 0 0
Puebla
Country [19] 0 0
Mexico
State/province [19] 0 0
Veracruz
Country [20] 0 0
Poland
State/province [20] 0 0
Warsaw
Country [21] 0 0
Portugal
State/province [21] 0 0
Lisboa
Country [22] 0 0
Portugal
State/province [22] 0 0
Santa Maria da Feira
Country [23] 0 0
Portugal
State/province [23] 0 0
Vila Nova de Gaia
Country [24] 0 0
South Africa
State/province [24] 0 0
Benoni
Country [25] 0 0
South Africa
State/province [25] 0 0
Cape Town
Country [26] 0 0
South Africa
State/province [26] 0 0
Durban
Country [27] 0 0
South Africa
State/province [27] 0 0
Pretoria
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Spain
State/province [30] 0 0
San Sebastián de los Reyes

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
PTC Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a randomized, double-blind, placebo-controlled, multicenter, 28-day study of adult
participants hospitalized with COVID-19, with a safety follow-up telephone call at Day 60.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04439071
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Quintus Ngumah, OD, PhD
Address 0 0
PTC Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04439071