Learn more about this research?
- For this study we are looking for healthy Caucasian males between the age of 18 and 45 years.
- Your Body Mass Index (BMI) has to be between 18.5 and 30.0 kg/m2 and your body weight must be at least 50 kg. The BMI shows the relation between the bodyweight in kilograms and the body length in meters.
- Both light smokers and non-smokers are allowed to participate. However, smoking in the clinic is not allowed.
- To determine if you are suitable to participate in this study, you will undergo medical screening. Before the medical screening you will visit the medical screening center for a short visit to have a blood sample taken to determine what your metabolism is.
For part 2, also the following particulars are applicable:
- After approval at the screening center you will bring a separate visit to the Keizer Clinic in Assen to undergo a gastroscopy.
- The target of the study compound administered in this study is the muscle tissue. To investigate the amount of study compound that has reached the muscle tissue a muscle biopsy will be taken in the clinic once.
- Volunteers who have had a vasectomy are allowed to participate but have no semen collection. Collection of several semen samples at screening, 3 days after the screening, at follow-up, 3 days after the follow-up, at day 105 and day 109.
You will receive a gross compensation of € 4.759,- for participation in one of the groups below untill day 109 and you will receive € 25,- extra for the short visit. This compensation also includes a compensation for the days between the follow-up and the last visit.
Sterilized men will receive a gross compensation of € 3.307,- for participation in one of the groups mentioned above untill day 28 and you will receive € 25,- extra for the short visit.
Traveling expenses will be reimbursed based on kilometers (€ 0,19 net per kilometer) with a minimum of € 12,- and a maximum of € 160,- (840 kilometers) per return, regardless of the mode of transportation.
Period of stay and research
The study consists of one period in which you will stay in our research facility in Groningen for 20 days (19 nights), followed by four short visits: at follow-up, 3 days after the follow-up, at day 105 and at day 109.
Information sheet PRA-133871 (part 2)
Type of study
The purpose of the study is to investigate to what extent the study medication is safe and tolerated. It will also be investigated how quickly and to what extent the study medication is absorbed and eliminated from the body. The study drug is not registered as a drug and has not been given to humans before. The study is already approved by the Independent Ethics Committee.
This study consists of two parts; Part 1 and Part 2. The remainder of this document only refers to Part 2.
Setup and duration of the study
Part 2 of this study will consist of 1 period during which you will stay in our clinical research center in Groningen, location UMCG for 20 days (19 nights). During Part 2 of this study you will receive the study drug or placebo once a day in the form of an oral tablet.
The screening process is devided into several visits and will take place in the medical screening center in Groningen, location Martini.
- First screening: a blood sample will be taken to determin your metabolism
- Second screening: Actual screening and first semen sample
- Third screening: Second semen sample
- Forth screening: After approval from the first 3 screenings you will bring a separate visit to the Keizer Clinic in Assen to undergo a gastroscopy. A gastroscopy is a save examination.
Volunteers who have had a vasectomy are allowed to participate but have no semen collection. Collection of several semen samples at screening, at follow-up and at day 105 and day 109.
Blood samples will be taken and urine will be collected regularly during the study. Besides this also a gastroscopy will take place three times (on screening, during study and if necessary at follow-up). The target of the study compound administered in this study is the muscle tissue. To investigate the amount of study compound that has reached the muscle tissue a muscle biopsy will be taken in the clinic once.
Risks and medical supervision
Duchenne’s disease causes irreversible damage of muscle tissue and therefore muscle weakness. This muscle weakness is caused by a low calcium concentration in the muscles. The study drug causes the calcium levels to stay on a certain level, so the damage to muscle tissue and muscle weakness could be prevented.
Adverse effects observed during the first parts were: headache, nausea, abdominal pain, stomachache, bloating, flatulence, stomach discomfort, hunger, dry mouth, muscle pain, pain arms, chest muscle stiffness, muscle cramps and pain in the lower jaw, muscle twitching buttock, muscle cramp thigh, itching both hands, lower back pain, skin irritation at holter and ECG stickers, hematoma at venipuncture site, flu, common cold, nasal congestion, postural dizziness, dizziness, transpiration, sore throat, restlessness, tiredness, sleepiness, anxiety, loose stools, cannula site reaction, increased frequency urination, salivation increased, fast heartbeat, nosebleed, pink eye, sore eye, toothache, higher blood levels of liver enzymes, fungal infection in lower part of the esophagus, and erosion of the intestinal wall in the first part of the duodenum and the stomach wall. At this time it is not known if these effects are related to the study compound as the study is still blinded
You will be under strict medical supervision during the study. Before the study and at the end of the study you will undergo an extensive medical screening.
Conditions for participation
You are a healthy male between 18 and 45 years old. You can only participate in this study if you do nog smoke or smoke less than 5 cigarettes per day. Since the metabolism of the drug in the body can vary based on ethnicity, only caucasion volunteers can participate. This means, you belong to or originates from one of the original people of Europe, the Middle east or North Africa, or the Indian subcontinent.
The study will be executed under standardized conditions. Use of your own medication, alcohol, regular coffee and tea, cola, power drinks, chocolate (including products made of chocolate), grapefruit (juice) and tobacco products during the study is not allowed. Also before the study there will be restrictions for these products.
Use of decaffeinated coffee and (herbal) teas without caffeine is allowed. Before you will take part in the study, you will be medically screened. This screening will take place within four weeks before the start of the study.
Dates for stay in the clinic:
The follow up visit will be conducted 2 to 5 days after departure from the research facility, 3 or 4 days after follow-up and followed by two short visit at day 105 and day 109. A possibility is open for a follow-up after 8 weeks for a gastroscopy.
At entry in the clinic you must have fasted for 4 hours.
- Group 13a: 19 March – 07 April 2018
- Group 13b: 20 March – 08 April 2018
- Group 13c: 26 March – 14 April 2018
- Group 13d: 27 March – 15 April 2018
Schedule for day 105 and day 109:
- Group 13a: 03 en 06 July 2018
- Group 13b: 03 en 06 July 2018
- Group 13c: 10 en 13 July 2018
- Group 13d: 10 en 14 July 2018
- Period of stay
- 20 days
- 0 times
- € 4.759,-
Groep 13c€ 4.759,-
20 days stay
- 26 Mar 2018 up to and including 14 Apr 2018
Groep 13d€ 4.759,-
20 days stay
- 27 Mar 2018 up to and including 15 Apr 2018
Groep 13a€ 4.759,-
20 days stay
- 19 Mar 2018 up to and including 7 Apr 2018
Groep 13b€ 4.759,-
20 days stay
- 20 Mar 2018 up to and including 8 Apr 2018