Phase IIa biomarker study for evaluating the effect, tolerability and safety of study drug, N-acetýlcýsteine in patients with hereditary stroke due to L68Q mutations in the cystatin C gene (HCCAA).
- Conditions
- Hereditary Cystatin C Amyloid Angiopathy (HCCAA)Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
- Registration Number
- EUCTR2017-004776-56-IS
- Lead Sponsor
- Arctic Therapeutics ehf
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 50
Inclusion Criteria
1Patient is male or female, ages 18 or older, of Icelandic ancestry known to carry the HCCAA mutation L68Q.
2Patient is judged to be in sufficient medical health to be able to participate in the study.
3Patient has HCCAA confirmed by mutation status detection of L68Q
4Patient has been genotyped/sequenced and confirmed to carry the L68Q mutation in the cystatin C gene
5Patient is willing to have a baseline and follow up skin biopsy every 3 months for up to 9 months.
6Patient has provided informed consent for participation in trial
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria
1.Patient does not have L68Q mutation
2.Patient has clinically significant illness, mental or physical, that, in the opinion of the investigator, might confound the results of the study, pose additional risk to the patient by their participation, or prevent/impede the patient from completing the study.
3.Patient is pregnant or attempting to become pregnant
4.Patient tests positive for illicit drugs (including marijuana) or has history of drug abuse within the last 2 years.
5.Patient consumes excessive amounts of alcoholic beverages.
6.There is any concern by the investigator regarding the patient’s safety, compliance, or suitability with respect to his/her participation in the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method