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- Your Body Mass Index (BMI) is between 18 and 28 kg/m2. The BMI shows the relation between body weight in kilograms and height in meters.
- Both non-smokers and light smokers are allowed to participate in this study; however, during your stay in the research facility, smoking is not allowed.
- Women are only allowed to participate when surgically sterile or post-menopausal or use acceptable methods of contraception (birth control) to participate in this study.
- To determine if you are suitable to participate in this study, you will undergo a medical screening.
For Part B (Group B3 and later groups) you will receive gross compensation of € 2.406,- for full participation.
Travelling expenses will be reimbursed based on the distance traveled (€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
For Part B, (Group B3 and later groups) the study consists of 4 periods during which you will stay in our research facility in Groningen. For periods 1 and 4 you will stay for 5 days (4 nights), for periods 2 and 3 you will stay for 2 days (1 night). There will be 1 short visit after the first three periods and there will be 6 short visits after period 4. Finally, there will be a follow up consisting of 2 separate visits.
Please note: You have to be available for all dates for the group of choice to be able to participate in this study. These are the currently planned dates; however, these may be subject to change.
The medical screening for this study will take place in the screening center in Groningen or Utrecht.
Information sheet PRA-172921
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. In addition, the tolerability of the new drug will be compared to a placebo. A placebo is a medicine without any active ingredient.
The study drug is not registered as a drug and has not been given to humans before. The study will only take place after it has been approved by the Independent Ethics Committee.
Setup and duration of the study
The study consists of three parts and each volunteer will participate in only one part.
For Part A, the actual study will consist of 1 period during which you will stay in the clinical research center in Groningen, location Martini for 5 days (4 nights) followed by 2 short visits. For Part A, Group 4 and Group 5, the actual study will consist of 1 period during which you will stay in the clinical research center in Groningen, location Martini for 5 days (4 nights) followed by 5 short visits.
For Part B, Group B1, the actual study will consist of 7 periods during which you will stay in the clinical research center in Groningen, location Martini for period 1 and 7 for 6 days (5 nights). For the other periods you will stay in the clinical research Center for 2 days (1 night). Period 7 will be followed by 1 short visit. For Part B, Group B2, the actual study will consist of 7 periods during which you will stay in the clinical research center in Groningen, location Martini for period 1 and 7 for 6 days (5 nights). For the other periods you will stay in the clinical research Center for 2 days (1 night). Period 7 will be followed by 4 short visits. For Part B, Group B3 and B4 the actual study consists of 4 periods during which you will stay in our research facility in Groningen, location Martini. For Period 1 and 4, you will stay for 5 days (4 nights) and for Periods 2 and 3, you will stay for 2 days (1 night). A short visit will follow after the first 3 Periods and Period 4 will be followed by 5 short visits.
For Part C, the actual study will consist of 2 periods during which you will stay in the clinical research center in Groningen, location Martini for 5 days (4 nights) followed by 2 short visits for period 1 and followed by 2 short visits for period 2. For Part A, you will receive the study drug or placebo once in the form of subcutaneous injection or for Group A5 and A6 in the form of a subcutaneous infusion. For Part B and C, you will receive the study drug or placebo recurrence in the form of subcutaneous injection or for Group B2, B3, B4 and C1 in the form of a subcutaneous infusion.
Blood samples will be taken, and urine will be collected regularly during the study
Risks and medical supervision
The new compound may eventually be used for the treatment of achondroplasia. People with achondroplasia have a short stature and achondroplasia is a common cause of dwarfism. Achondroplasia is caused by an alteration in the gene for a protein called fibroblast growth factor receptor 3 (FGFR3). FGFR3 plays an important role in bone growth and over-activation of FGFR3 causes shorter bones. In achondroplasia the alternated form of FGFR3 is overly active and this leads to shorter bones. FGFR3 is being activated by binding specific proteins called fibroblast growth factor.
The new compound is developed as a FGFR3 decoy protein. When the new compound is administered to humans a large part of the fibroblast growth factor will bind to the new compound instead of the endogenous FGFR3 whereby FGFR3 will not be activated or less activated. In this way, the new compound can be used as a treatment of achondroplasia. The new compound will be administered to humans for the first time in this study, side effects of the new compound in humans have not been reported to date. However, it has been studied in animals.
The compound is specifically developed for children with abnormal growth as result of an alteration in cartilage cells accountable for the growth of long bones. These cells have disappeared in adulthood hence we expect few side effects no effect on growth. It has been studied in both young and adult animal’s mice and monkeys and a proportion of the animals developed antibodies. It is possible you may develop antibodies against the compound. Based on experience with it is not expected that the presence of these antibodies will have consequences for your health. Because of the important role of FGFR3 in wound healing of the skin, in phosphate levels and in vitamin D levels, you will be monitored for changes in these as well.
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 or female between 21 and 55 years old. You can only take part in the study if you do not smoke or are a light smoker. Women must be surgically sterile or postmenopausal or use acceptable methods of contraception (birth control) to participate in this study.
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 are allowed to take part in the study, you will be medically screened; this screening will take place within four weeks before the start of the study
- Period of stay
- 2 times 2 days and 2 times 5 days days
- 9 times
- € 2.406,-
Group B4€ 2.406,-
2 times 2 days and 2 times 5 days days stay
- 25 Nov 2018 up to and including 29 Nov 2018
- 2 Dec 2018 up to and including 3 Dec 2018
- 9 Dec 2018 up to and including 10 Dec 2018
- 16 Dec 2018 up to and including 20 Dec 2018
- 1 Dec 2018
- 6 Dec 2018
- 13 Dec 2018
- 22 Dec 2018
- 24 Dec 2018
- 27 Dec 2018
- 31 Dec 2018
- 3 Jan 2019
- 7 Jan 2019