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

NCT04439071 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: PTC299-VIR-015-COV192020-001872-13
• Study Type: interventional
• Target: 189
• Locations: 11 countries
• Sites: 41

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.

Conditions

Pneumonia; COVID-19; Coronavirus

Outcome Measures

Primary Outcomes (1)

• Time From Randomization to Respiratory Improvement

  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.

  up to Day 28

Secondary Outcomes (18)

• Number of Participants Requiring Invasive Ventilation

  up to Day 28

• Number of Participants Requiring Supplemental Oxygen or Non-Invasive Ventilation in Participants Who Did Not Require Supplemental Oxygen at Baseline

  up to Day 28

• 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)

  up to Day 28

• Time From Randomization to Respiratory Rate ≤ 24 Breaths Per Minute on Room Air

  up to Day 28

• Time From Randomization to Cough Reported as Mild or Absent

  up to Day 28

Other Outcomes (1)

• Time From Randomization to Respiratory Improvement Where Symptom Onset Occurred ≤5 Days

  up to Day 28

Study Arms (2)

PTC299 + Standard of Care (SOC)

EXPERIMENTAL

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.

Drug: PTC299; Other: SOC

Placebo + SOC

PLACEBO COMPARATOR

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.

Other: SOC; Drug: Placebo

Interventions

PTC299 DRUG

Oral tablets

Also known as: Emvododstat

PTC299 + Standard of Care (SOC)

SOC OTHER

As defined per local written policies or guidelines.

PTC299 + Standard of Care (SOC); Placebo + SOC

Placebo DRUG

Oral tablets

Placebo + SOC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (41)

University of California, Irvine

Orange, California, 92868, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

University of Massachusetts Memorial Health Care

Worcester, Massachusetts, 01605, United States

Location

University Hospitals Cleveland

Cleveland, Ohio, 44106, United States

Location

Ralph H. Johnson VA Medical Center

Charleston, South Carolina, 29401, United States

Location

Westmead Hospital

Westmead, New South Wales, 02145, Australia

Location

Sunshine Hospital

St Albans, VC, 03021, Australia

Location

Monash Medical Centre

Clayton, Victoria, 3168, Australia

Location

St. Pierre University Hospital

Brussels, 1000, Belgium

Location

Clinique Saint Pierre

Ottignies, B-1340, Belgium

Location

Hospital Vera Cruz

Belo Horizonte, Minas Gerais, 30190-130, Brazil

Location

Hospital Moinhos de Vento

Porto Alegre, Rio Grande do Sul, 90035-000, Brazil

Location

Centro Hospitalar Unimed (CHU) - Joinville

Joinville, Santa Catarina, 89204-061, Brazil

Location

Hospital Guilherme Alvaro

Santos, São Paulo, 11045-904, Brazil

Location

Hospital Alemao Oswaldo Cruz

São Paulo, São Paulo, 01508-000, Brazil

Location

Escola Paulista de Medicina (UNIFESP)

São Paulo, São Paulo, 04023-062, Brazil

Location

Fundação Faculdade Regional de Medicina de São José do Rio Preto

São Paulo, São Paulo, 15090-000, Brazil

Location

Hospital Santa Casa de Misecórdia de Sorocoba

Sorocaba, São Paulo, 18013-000, Brazil

Location

Fundación Santa Fe de Bogotá

Bogotá, 110311, Colombia

Location

Centro Cardiovascular Somer Incare

Rionegro, 054040, Colombia

Location

Hôpital Pitié-Salpêtrière

Paris, 75013, France

Location

Centro Hospitalario MAC

Irapuato, Guanajuato, 36520, Mexico

Location

Hospital Universitario Dr. José Eleuterio Gonzalez

Monterrey, Nuevo León, 64460, Mexico

Location

Integra RGH Centro de Investigación/ Hospital MAC Puebla

Puebla City, 72410, Mexico

Location

SOMECO - Sociedad de Metabolismo y Corazón S.C.

Veracruz, 91910, Mexico

Location

Central Clinic Hospital of the MSWiA in Warsaw

Warsaw, 02-507, Poland

Location

Centro Hospitalar Universitário de Lisboa Norte (CHULN), E.P.E - Hospital de Santa Maria

Lisbon, 1649-035, Portugal

Location

Centro Hospitalar de Entre o Douro e Vouga, EPE (CHEDV)

Santa Maria da Feira, 4520-211, Portugal

Location

Centro Hospitalar de Vila Nova de Gaia/Espinho, EPE (CHVNG/E)

Vila Nova de Gaia, 4434-502, Portugal

Location

Worthwhile Clinical Trials

Benoni, 1500, South Africa

Location

TREAD Research

Cape Town, 7500, South Africa

Location

Tiervlei Trial Centre

Cape Town, 7530, South Africa

Location

Ahmed Al-Kadi Private Hospital

Durban, 4058, South Africa

Location

Global Clinical Trials

Pretoria, 0001, South Africa

Location

Hospital Del Mar

Barcelona, 08003, Spain

Location

Hospital Universitario de Bellvitge

Barcelona, 08907, Spain

Location

Hospital Universitario Infanta Leonor

Madrid, 28031, Spain

Location

Hospital Universitario Ramón y Cajal

Madrid, 28034, Spain

Location

Hospital 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario Infanta Sofía

San Sebastián de los Reyes, 28702, Spain

Location

Related Links

• FINAL REDACTED PROTOCOL

MeSH Terms

Conditions

Pneumonia; COVID-19; Coronavirus Infections

Interventions

emvododstat

Condition Hierarchy (Ancestors)

Respiratory Tract Infections; Infections; Lung Diseases; Respiratory Tract Diseases; Pneumonia, Viral; Virus Diseases; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections

Results Point of Contact

• Title: Patient Advocacy
• Organization: PTC Therapeutics, Inc.

medinfo@ptcbio.com

1-866-562-4620

Study Officials

• Quintus Ngumah, OD, PhD

  PTC Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 17, 2020
• First Posted: June 19, 2020
• Study Start: July 9, 2020
• Primary Completion: July 20, 2022
• Study Completion: July 20, 2022
• Last Updated: June 26, 2023
• Results First Posted: June 26, 2023

Record last verified: 2023-05

Locations

FR (1); BR (8); ME (4); CO (2); AU (3); PL (1); ZA (5); US (6); BE (2); ES (6); PT (3)