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

NCT ID: NCT04439071

Last Updated: 2023-06-26

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 189 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-07-09
• Study Completion Date: 2022-07-20

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: EXPERIMENTAL

PTC299

Intervention Type: DRUG

Oral tablets

SOC

Intervention Type: OTHER

As defined per local written policies or guidelines.

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.

Group Type: PLACEBO_COMPARATOR

SOC

Intervention Type: OTHER

As defined per local written policies or guidelines.

Placebo

Intervention Type: DRUG

Oral tablets

Interventions

PTC299

Oral tablets

Intervention Type: DRUG

SOC

As defined per local written policies or guidelines.

Intervention Type: OTHER

Placebo

Oral tablets

Intervention Type: DRUG

Other Intervention Names

Emvododstat

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

PTC Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Quintus Ngumah, OD, PhD

Role: STUDY_DIRECTOR

PTC Therapeutics

Locations

University of California, Irvine

Orange, California, United States

Augusta University

Augusta, Georgia, United States

Johns Hopkins Hospital

Baltimore, Maryland, United States

University of Massachusetts Memorial Health Care

Worcester, Massachusetts, United States

University Hospitals Cleveland

Cleveland, Ohio, United States

Ralph H. Johnson VA Medical Center

Charleston, South Carolina, United States

Westmead Hospital

Westmead, New South Wales, Australia

Sunshine Hospital

St Albans, VC, Australia

Monash Medical Centre

Clayton, Victoria, Australia

St. Pierre University Hospital

Brussels, , Belgium

Clinique Saint Pierre

Ottignies, , Belgium

Hospital Vera Cruz

Belo Horizonte, Minas Gerais, Brazil

Hospital Moinhos de Vento

Porto Alegre, Rio Grande do Sul, Brazil

Centro Hospitalar Unimed (CHU) - Joinville

Joinville, Santa Catarina, Brazil

Hospital Guilherme Alvaro

Santos, São Paulo, Brazil

Hospital Alemao Oswaldo Cruz

São Paulo, São Paulo, Brazil

Escola Paulista de Medicina (UNIFESP)

São Paulo, São Paulo, Brazil

Fundação Faculdade Regional de Medicina de São José do Rio Preto

São Paulo, São Paulo, Brazil

Hospital Santa Casa de Misecórdia de Sorocoba

Sorocaba, São Paulo, Brazil

Fundación Santa Fe de Bogotá

Bogotá, , Colombia

Centro Cardiovascular Somer Incare

Rionegro, , Colombia

Hôpital Pitié-Salpêtrière

Paris, , France

Centro Hospitalario MAC

Irapuato, Guanajuato, Mexico

Hospital Universitario Dr. José Eleuterio Gonzalez

Monterrey, Nuevo León, Mexico

Integra RGH Centro de Investigación/ Hospital MAC Puebla

Puebla City, , Mexico

SOMECO - Sociedad de Metabolismo y Corazón S.C.

Veracruz, , Mexico

Central Clinic Hospital of the MSWiA in Warsaw

Warsaw, , Poland

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

Lisbon, , Portugal

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

Santa Maria da Feira, , Portugal

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

Vila Nova de Gaia, , Portugal

Worthwhile Clinical Trials

Benoni, , South Africa

TREAD Research

Cape Town, , South Africa

Tiervlei Trial Centre

Cape Town, , South Africa

Ahmed Al-Kadi Private Hospital

Durban, , South Africa

Global Clinical Trials

Pretoria, , South Africa

Hospital Del Mar

Barcelona, , Spain

Hospital Universitario de Bellvitge

Barcelona, , Spain

Hospital Universitario Infanta Leonor

Madrid, , Spain

Hospital Universitario Ramón y Cajal

Madrid, , Spain

Hospital 12 de Octubre

Madrid, , Spain

Hospital Universitario Infanta Sofía

San Sebastián de los Reyes, , Spain

Countries

United States; Australia; Belgium; Brazil; Colombia; France; Mexico; Poland; Portugal; South Africa; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://filehosting-v2.pharmacm.com/api/Attachment/Download?tenantId=80217411&amp;parentIdentifier=PTC299-VIR-015-COV19&amp;attachmentIdentifier=642c87e2-eb4d-4a5c-85e5-1df1ccd361ef&amp;fileName=Global_PTC299-VIR-015-COV19_Clinical_Protocol_V7.0_-_02JUN2021_final_redacted.pdf&amp;versionIdentifier=

FINAL REDACTED PROTOCOL

Other Identifiers

2020-001872-13

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PTC299-VIR-015-COV19

Identifier Type: -

Identifier Source: org_study_id