PRA-179041 Cystic Fibrosis tot € 1.769,-

Full

Description

Study of an investigational compound that is being developed for the treatment of Cystic Fibrosis

Requested participants

  • M/F, healthy, 18 - 55 years old
  • Non-smokers only

Duration and visits

  • 6 till 15 days stay

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Research details

Particulars

  • You do not smoke.
  • Your Body Mass Index (BMI) is between 18.0 and 32.0 kg/m2 and your body weight is at least 50 kg. The BMI shows the relation between body weight in kilograms and height in meters.
  • Females are only allowed to participate in this study if they are sterilized (removal of both ovaries or/and uterus) or postmenopausal for at least 12 months (without menstrual periods).
  • You can only participate in Group A9 when you do not have a known or a suspected lactose intolerance or milk allergy. 
  • To determine if you are suitable to participate in this study, you will undergo a medical screening visit.
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Compensation

You will receive a gross compensation of € 750,- for complete participation in Group A6.‚Äč

You will receive a gross compensation of €1.769,- for complete participation in Group B5.

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 Group A6 the study consists of 1 period in which you will stay in our research facility in Groningen (for 6 days (5 nights).

For Group B5, the study consists of 1 period in which you will stay in our research facility in Groningen for 15 days (14 nights).

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.

Information sheet PRA-179041

Type of study

CF is a genetic disorder that causes the body to produce unusually thick mucus. The thick mucus results in malfunction of organs like the lungs, pancreas and liver.

The study will consist of 3 parts (Parts A, B, and C).  Part A will be performed in 56 subjects, Part B will be performed in 48 subjects, and Part C will be performed in 16 subjects. 

The purpose of this study is to investigate how safe the investigational compound is and how it may affect the body when it is administered to healthy subjects.

Part A will investigate different doses of the investigational compound or placebo administered as a single dose.

Part B will investigate different doses of the investigational compound or placebo which will be administered once or twice daily for 10 days.

Part C will investigate different doses of the investigational compound combined with 2 other compounds (tezacaftor and ivacaftor) which will be administered once and/or twice daily for 14 days to investigate safety and tolerability of multiple ascending doses.

The study will only take place after it has been approved by the Independent Ethics Committee.

The purpose of Part A is to investigate the safety and tolerability of the investigational compound administered as a single dose. The study will also investigate how quickly and to what extent the investigational compound is absorbed into and eliminated from the body (this is called pharmacokinetics). The effect of milk and 2 different formulations (tablet and oral suspension) on the pharmacokinetics will be investigated as well.

The purpose of Part B is to investigate how safe the investigational compound is and how well the investigational compound is tolerated when administered once or twice daily for 10 days. The study will also investigate how quickly and to what extent the investigational compound is absorbed into and eliminated from the body (this is called pharmacokinetics).

The purpose of Part C is to investigate how safe the investigational compound is and how well the investigational compound is tolerated when administered in combination with tezacaftor and ivacaftor for 14 days. The study will also investigate how quickly and to what extent these compounds are absorbed into and eliminated from the body (this is called pharmacokinetics).

Setup and duration of the study

To determine if you are suitable to participate in this study, you will undergo a medical screening visit).

Part A: Following the screening visit, if you are eligible for participation, the study consists of 1 period

(with the exception of Group A3 and A9), in which you will stay in our research facility in Groningen for 6 days (5 nights).

For Group A3, the study consists of 1 period in which you will stay in our research facility in Groningen for 17 days (16 nights).

For Group A9, the study consists of 1 period in which you will stay in our research facility in Groningen for 25 days (24 nights).

On certain time-points you will be asked to remain in a supine position for 15 minutes without moving, talking or sleeping. During these moments you may not listen to music, watch television or use a lap top, media player and phone in the room.

Part B: Following the screening visit, if you are eligible for participation, the study consists of 1 period in which you will stay in our research facility in Groningen (location Martini) for 15 days (14 nights).

Blood samples will be taken regularly during the study. In addition, your heart rhythm will be monitored continuously from approximately 1 hour before (each) administration of the study compound until 24 hours thereafter. As long as you are connected to the telemetry you cannot take a shower, you have to behave as relaxed as possible and have to prevent that you will sweat. As soon as you are connected, you cannot use electric devices (razor device, electric toothbrush, hairdryer) anymore and you may not wear jewelry.

Part C: Following the screening visit, if you are eligible for participation, the study consists of 1 period in which you will stay in our research facility in Groningen (location Martini) for 19 days (18 nights).

Blood samples will be taken regularly during the study.

Risks and medical supervision- Part A

Groups A1, A2, A4, A5 and A6

During the study, you will receive a single dose of the investigational compound or placebo as an oral suspension (drink). A placebo is an oral suspension without the active ingredient (a fake medicine).

Group A3

In addition to the investigational compound administration described above, Group 3 will receive an additional single dose of the investigational compound on Day 9. On Day 9, all subjects will receive the investigational compound, no placebo will be administered on Day 9. Administration of the investigational compound will be followed by a glass of skim milk instead of water (240 ml).

Group A9

Similar to the investigational compound administration described for Groups A1, A2, A4, A5 and A6, you will receive the investigational compound on Day 1 as an oral suspension (drink). An additional dose will be administered on Day 6 as a tablet (instead of an oral suspension) with 240 ml water, and on Day 11 the investigational compound will be administered as a tablet with 240 ml milk (instead of water).

All drugs have the potential to cause side effects; the extent to which this occurs differs. The investigational compound has not previously been tested in humans. The side effects associated with the investigational compound are unknown.

The investigational compound has been studied in rats and dogs. There were no adverse effects in these animal studies related to the investigational compound.

The investigational compound in the skin may produce damage upon exposure to light, similar to sunburn. People receiving Study Drug should minimize exposure to sun (such as mid-day summer sun and direct sunlight on bright days) or other UV radiation from the first dose of Study Drug through the Safety Follow-up Visit. You should use a broad-spectrum (UV-B combination) sunscreen with an SPF (sun protection factor) of at least 15, or whatever strength has been enough to prevent tanning in the past.

You should be aware that the adverse events mentioned above and possibly other, still unknown adverse events may occur during the study.

You will be under strict medical supervision during the study.

Risks and medical supervision- Part B

During the study, you will receive the investigational compound or placebo once or twice daily for 10 days. The investigational compound or placebo will be given as an oral suspension (drink). A placebo is an oral suspension without the active ingredient (a fake medicine).

All drugs have the potential to cause side effects; the extent to which this occurs differs. The investigational compound has not previously been tested in humans. The side effects associated with the investigational compound are unknown.

The investigational compound has been studied in rats and dogs. There were no adverse effects in these animal studies related to the investigational compound.

The investigational compound in the skin may produce damage upon exposure to light, similar to sunburn. People receiving Study Drug should minimize exposure to sun (such as mid-day summer sun and direct sunlight on bright days) or other UV radiation from the first dose of Study Drug through the Safety Follow-up Visit. You should use a broad-spectrum (UV-B combination) sunscreen with an SPF (sun protection factor) of at least 15, or whatever strength has been enough to prevent tanning in the past.

You should be aware that the adverse events mentioned above and possibly other, still unknown adverse events, may occur during the study.

You will be under strict medical supervision during the study.

Risks and medical supervision- Part C

During the study, you will receive the investigational compound or placebo once or twice daily for 10 days. The investigational compound or placebo will be given as an oral suspension (drink). In addition, you will receive a combination of tezacaftor (TEZ) and ivacaftor (IVA) (combined into 1 tablet) or placebo once daily in the morning and ivacaftor (IVA) alone or placebo in the evening.  A placebo is an oral suspension without the active ingredient (a fake medicine).

All drugs have the potential to cause side effects; the extent to which this occurs differs. The investigational compound has not previously been tested in humans. The side effects associated with investigational compound are unknown.

The investigational compound has been studied in rats and dogs. There were no adverse effects in these animal studies related to the investigational compound.

The investigational compound in the skin may produce damage upon exposure to light, similar to sunburn. People receiving Study Drug should minimize exposure to sun (such as mid-day summer sun and direct sunlight on bright days) or other UV radiation from the first dose of Study Drug through the Safety Follow-up Visit. You should use a broad-spectrum (UV-B combination) sunscreen with an SPF (sun protection factor) of at least 15, or whatever strength has been enough to prevent tanning in the past. 

You should be aware that the adverse events mentioned above and possibly other, still unknown adverse events, may occur during the study.

You will be under strict medical supervision during the study.

Conditions for participation

You are a healthy male or female between 18 and 55 years old and you do not smoke. Your Body Mass Index (BMI) is between 18.0 and 32.0 kg/m2 and your body weight is at least 50 kg. The BMI shows the relation between body weight in kilograms and height in meters.

Females are only allowed to participate in this study if they are sterilized (removal of both ovaries or/and uterus) or postmenopausal for at least 12 months (without menstrual periods).

You can only participate when you do not have a known or a suspected lactose intolerance or milk allergy.

You must take measures to avoid exposure to sun or to excessive UV-radiation during your stay at the research facility and until the follow-up visit.

The study will be done under standardized conditions. Use of your own medication, alcohol, regular coffee and tea, cola, power drinks, chocolate (including products made of chocolate), grapefruit (juice), some other juices and food, and tobacco products during the study are 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.

More info Less info

Group A6

€ 750,-

6 days stay

  • 1 Oct 2018 up to and including 6 Oct 2018

short stay

Inapplicable
Group full

Group B5

€ 1.769,-

15 days stay

  • 14 Oct 2018 up to and including 28 Oct 2018

short stay

Inapplicable
Group full

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