Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

• Status: Completed
• Trial end date: 2021-03-03
• Target enrollment: 0
• Participant gender: All

Summary

This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who have this condition. NPC-1 is caused by a defect in a protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in which this experimental treatment works is not fully understood but laboratory experiments have shown that it can potentially remove cholesterol build up from the cells in people who have NPC-1. Approximately 12 patients will be asked to take part in this research study for up to 56 weeks in total. recruitment is expected to take 9 months.Patients who take part will receive treatment by an intravenous infusion every two weeks. The study will look at what the body does to the drug as well as what the drug does to the body by taking and examining blood and urine samples. Samples of Cerebrospinal fluid (CSF) are also taken by lumbar puncture during and following the first treatment dose. Patients will also have their hearing tested, be asked questions by their doctor as well completing questionnaires to help assess any changes in their condition during treatment. Optional assessments patients can choose to take part in include liver biopsies, additional lumbar punctures for CSF.examinations to see if the drug is affecting these. This study is being sponsored and funded by CTD holdings INC. It is planned to be run in the UK, Italy, and Sweden.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: CTD Holdings, Inc.; Cyclo Therapeutics, Inc.
• Treatments: Betadex

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

-

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

-