Safety and Pharmacokinetics of Oral PTC844 in Healthy Participants for Autoimmune Disorder Treatment

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What is this study about?

The study looks at a medicine called PTC844 that is being tested for possible use in people with autoimmune disorders. Autoimmune disorders are conditions where the body’s immune system mistakenly attacks its own tissues. The purpose of the trial is to see whether the medicine is safe and how the body handles it.

Healthy volunteers will first receive a single dose of the medicine or a matching placebo, which looks the same but contains no active drug. After a short observation period, some participants will receive several doses over a few weeks. Researchers will watch for any unwanted effects (adverse events) such as feeling unwell, changes in laboratory tests, heart rhythm checks (ECG), blood pressure, and physical examinations. Blood samples will also be taken to measure how much medicine is in the bloodstream and how quickly it is cleared.

1 initial study visit

you arrive at the study site after you have been enrolled in the trial.

the study staff will confirm that you meet the eligibility criteria and will explain the procedures that will be performed during the trial.

2 baseline assessments

a series of medical checks are performed, including blood tests, electrocardiogram (ecg), vital‑sign measurements, and a physical examination.

the results create a reference point for later comparison.

3 single‑dose phase (part 1)

you receive a single oral dose of ptc844 45 mg as an oral suspension, or a matching placebo that looks the same but contains no active ingredient.

the dose is taken once under supervision of the study staff.

after dosing, you remain at the site for a period of observation during which blood samples are taken at several time points to measure the amount of drug in your blood.

your health is monitored for any side effects, changes in laboratory values, ecg results, vital signs, or physical findings.

4 washout period

a short interval follows the single‑dose phase to allow the drug to leave your body before the next part of the study begins.

no study medication is taken during this time.

5 multiple‑dose phase (part 2)

you receive the same oral suspension of ptc844 45 mg (or matching placebo) on a repeated schedule as defined by the study protocol.

each dose is taken by mouth under supervision, and the dosing continues for the number of days specified in the protocol.

blood samples are collected after several doses to evaluate how the drug accumulates in your body.

continuous monitoring for side effects and regular safety assessments are performed throughout this period.

6 final assessments and study completion

at the end of the multiple‑dose phase, a final set of medical tests is performed, including blood work, ecg, vital‑sign checks, and a physical examination.

the collected information is used to determine the safety and how the drug behaved in your body.

after the final visit, no further study medication is taken.

Who Can Join the Study?

Signed informed consent: You must sign a document that shows you understand the study and agree to take part.
All prescription medicines must be stopped at least 14 days (or five half‑lives, whichever is longer) before the first study visit; the same rule applies to over‑the‑counter drugs, vitamins, supplements and herbal products. You may continue to take up to 2 g of paracetamol per day until the first visit.
You must not drink any alcohol from 48 hours before the first visit until you leave the study.
You must avoid grapefruit juice from 7 days before the first visit until you leave the study.
You must have a negative test for alcohol, illegal drugs and cotinine (a substance that shows recent nicotine use) at both the screening and the first visit.
For the second part of the study, you must be fully vaccinated against SARS‑CoV‑2 (the virus that causes COVID‑19) at least two weeks before the first visit (either one dose of the Janssen vaccine or two doses of an mRNA vaccine).
You must be willing and able to attend all scheduled study visits, follow the treatment plan, have laboratory tests done, and follow any study restrictions.
Your age must be between 18 and 55 years at the time of screening.
Your body mass index (BMI) must be greater than 18.5 and no more than 30.0 kg/m², and you must weigh at least 50 kg at screening.
You must be considered healthy based on your medical history, physical exam, lab results, an ECG (a heart test) showing a QTcF (a corrected heart‑beat interval) of 450 milliseconds or less, and normal vital signs.
If you are a male participant who is sexually active with women who could become pregnant and have not had a vasectomy, you must use a barrier method of birth control (such as condoms) during the study and for 90 days after the last dose, and you must not donate sperm during the study and for 90 days after the last dose.
If you are a male participant, you must not donate sperm during the study and for 90 days after the last dose.
If you are a female participant, you must be of non‑childbearing potential. This means you are either postmenopausal (no periods for at least 12 months and hormone levels in the postmenopausal range) or have been surgically sterilized (such as tubal ligation, hysterectomy, or removal of both ovaries).
Female participants must have a negative blood (serum) pregnancy test at screening and a negative urine pregnancy test at the first visit.
You must not use any nicotine‑containing products (like cigarettes, vaping, or nicotine patches) within one month before the first visit.

Who Cannot Join the Study?

You have taken part in another drug or device study within the last 60 days, or you plan to join another study while this one is running.
You tested positive for COVID-19 (the virus called SARS‑CoV‑2) by a PCR test, or you have been in close contact with someone who tested positive for COVID-19 in the two weeks before the study start.
You received a COVID‑19 vaccine or any other vaccine within two weeks before the study start, or you plan to receive a vaccine during the study. Live vaccines (vaccines that contain a weakened form of a germ) are not allowed from four weeks before the study start until the study ends.
You have a current or past medical condition, illness, or mental health problem that the doctor thinks could make the study unsafe for you or could affect the study results.
You have any important abnormal test result, such as abnormal liver function tests (blood tests that check how well your liver is working).
You have ever had tuberculosis (an infection that usually affects the lungs).
You have psychological or emotional problems, or are receiving therapy for such problems, that could prevent you from understanding the study information or following the study rules.
You gave plasma (the liquid part of blood) within 7 days before receiving the first dose of the study drug, or you gave more than 50 mL of blood (excluding routine testing or menstrual blood) within 30 days, or more than 499 mL of blood within 56 days before the first dose.
You regularly drink a lot of alcohol – more than 21 units per week if you are male or more than 14 units per week if you are female – or you drank any alcohol within 24 hours before the screening visit. One unit equals about half a pint (280 mL) of beer, one measure (25 mL) of spirits, or a small glass (125 mL) of wine.
You have a history of alcohol abuse (problematic drinking) within the past two years.
Blood tests show you are positive for any of the following: Hepatitis B surface antigen (a marker of Hepatitis B infection), Hepatitis C antibody (a marker of Hepatitis C infection), or HIV antibody (a marker of HIV infection).

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Pwiuormiodkxnx Rhuvsoxf Acbunrffom Gkbzc Bhia	Groningen	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not recruiting	20.12.2022

Trial locations

PTC844 is an experimental medicine being studied for the first time in healthy volunteers. It is taken by mouth as a liquid suspension. In this trial, researchers are giving single and multiple doses to see how safe it is and how the body absorbs, distributes, and clears the drug. The goal is to learn whether participants can tolerate it without serious side effects and to gather information that will guide future studies.

Investigated Diseases:

Autoimmune disorder

Autoimmune disorders – Autoimmune disorders are conditions where the immune system mistakenly attacks the body’s own tissues. They can affect many organs and may start with mild symptoms that gradually worsen as more tissue is damaged. The disease course often involves periods of flare‑ups with increased activity and remission phases with fewer symptoms. Over time, repeated attacks can lead to chronic inflammation and functional loss in the affected organ. The progression varies with the specific organ involved and the individual’s immune response.

Trial ID:

2022-501986-38-00

Protocol code:

PTC844-SMAD-101-HV

Trial Phase:

Human Pharmacology (Phase I) – First administration to humans

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Safety and Pharmacokinetics of Oral PTC844 in Healthy Participants for Autoimmune Disorder Treatment

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?