Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 12:57 AM
Ignite Modification Date:
2025-12-25 @ 12:57 AM
NCT ID:
NCT02912793
Eligibility Criteria:
Inclusion Criteria:
1. Confirmed diagnosis of NPC-1 defined as one of the following
* Two NPC-1 mutations on genotyping
* One NPC-1 mutation and positive filipin staining (current or prior)
* Vertical supranuclear gaze palsy \[VSNGP\] plus either ≥ one NPC-1 mutation or positive filipin staining and no NPC-2 mutations
2. NIH NPC Severity Score \<30 and with no more than 4 individual domains with a score ≥ 3.
3. Age range: 2 years upwards
* Inclusion of the first three paediatric patients will be restricted to individuals aged ≥ 5 years. Once the first three paediatric patients have safely completed stage 1, study entry will be open to all ages ≥2 years as per the protocol
4. Negative pregnancy test for females of child bearing potential
5. Written, informed consent
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Exclusion Criteria:
1. The presence of NPC-2 mutations on genotyping
2. Previous receipt of cyclodextrin therapy
3. Lanksy score \< 50 if aged ≤16 or Karnofsky score \< 40 if aged \> 16.
4. Inability to comply with the proposed protocol assessments
5. Concurrent treatment with any type of cholesterol lowering agents such as statins, fibrates, ezetimibe
6. Concurrent medical conditions representing a contraindication to any of the study medications
7. Stage 3 renal impairment or worse as indicated by eGFR\< 60mL/min using the MDRD equation
8. Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or INR \>1. 8
9. Involvement in another interventional clinical trial within the previous 6 months
10. Weight \>100 kg
11. Females of childbearing potential who are not willing to use a method of highly effective contraception (hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, or true abstinence) during the study and the follow-up period. True abstinence can only be in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.
12. Females who are breastfeeding
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Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
2 Years
Study:
NCT02912793
Study Brief:
Protocol Section:
NCT02912793