COVID-19 trials registries data warehouse

https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-001872-13/GR

PTC Therapeutics, Inc. - Patient Advocacy

medinfo@ptcbio.com

2022-03-11

Terminated

RCT

Randomized

Parallel

Blind label

multi-center

Treatment

1. Subject (or legally authorized representative) is willing and able to provide informed consent and comply with all protocol requirements. 2. Agrees to the collection of nasopharyngeal swabs and venous blood and all other protocol-specified procedures. 3. Male or non-pregnant female adult ≥18 years of age at time of enrollment. 4. Hospitalized and has laboratory-confirmed infection with SARS-CoV-2. 5. Symptom onset was ≤14 days prior to Screening 6. Has SpO2 <94% on room air 7. Has at least one of respiratory rate >24 breaths/minute or cough 8. Lung involvement as confirmed by radiographic infiltrates observed on imaging (chest X-ray, CT scan, or an equivalent test) 9. 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: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: - oral - intravaginal - transdermal progestogen-only hormonal contraception associated with inhibition of ovulation: - oral - injectable - implantable intrauterine device intrauterine hormone-releasing system 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 (eg, bilateral tubal ligation, hysterectomy, bilateral oophorectomy). 10. 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.

1. Requires mechanical ventilation 2. Current participation in any other interventional study. 3. Severe liver disease as defined by alanine transaminase/aspartate transaminase levels (ALT/AST) >4 times the upper limit of normal. 4. Lymphocyte count <500 lymphocytes/µL or hemoglobin <11.0 g/dL 5. Stage 4 severe chronic kidney disease or requiring dialysis (ie, estimated glomerular filtration rate <30) 6. Any other condition, that in the opinion of the investigator, may be cause to exclude the subject from the study. 7. Use of steroids (except dexamethasone), drugs metabolized by CYP2D6, CYP2C inducers, IL-6 neutralizing antibodies, IL-6 receptor inhibitors, or any investigational therapy. 8. Pregnancy or breast feeding. 9. Anticipated transfer to another hospital which is not a study site within 72 hours. 10. Known allergy to PTC299 or excipients

2

PTC Therapeutics, Inc.

18

100

Australia;Belgium;Brazil;France;Italy;Mexico;Poland;Portugal;Spain;United States

Severe disease at enrollment

6: Severe disease at enrollment

50

The primary endpoint is the time from randomization to respiratory improvement, defined as peripheral oxygen saturation (SpO2) ≥94% on room air sustained until discharge from the hospital or the end of the study (Day 28).

Declared number of arm (3.0) differs from found arms (2.0)

Phase 2/Phase 3

[{"arm_notes": "New Type treatment : Emvododstat", "treatment_id": 2187, "treatment_name": "Placebo", "treatment_type": "Placebo", "pharmacological_treatment": "Placebo"}, {"arm_notes": null, "treatment_id": 1038, "treatment_name": "Ptc2995 (emvododstat)", "treatment_type": "Others pharmacological treatment", "pharmacological_treatment": "Pharmacological treatment"}]