Open‐Label Study of Long‐Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann‐Pick Disease Type C
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology, Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 3/30/2019 |
| Start Date: | March 2019 |
| End Date: | March 2021 |
| Contact: | Sharon H Hrynkow, PhD |
| Email: | sharon.hrynkow@cyclodex.com |
| Phone: | 3869657273 |
An Open‐Label Extension Study of the Long‐Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP‐β‐CD) in Patients With Niemann‐Pick Disease Type C (NPC‐1)
The purpose of this study is to provide continued access to treatment for NPC-1 after
participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses
of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two
weeks.
participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses
of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two
weeks.
This is an open‐label extension study of intravenous HP‐β‐CD (administered as Trappsol®
Cyclo(TM)) in patients with NPC‐1 to evaluate long‐term safety and efficacy by providing
continued access to treatment following completion of study CTD‐TCNPC‐101. Patients will
receive medication at their home or local site under the supervision of a home nurse
professional or at the parent site under the supervision of the site principal investigator.
Patients will visit the parent site for safety and efficacy evaluations after receiving
treatment for 3 months in the extension protocol.
Thereafter, visits to the parent site will be every 6 months until cessation. The study will
continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient
discontinuation. All patients who complete study CTD‐TCNCP‐101 and pass the screening
criteria will be eligible for this study. The study will be directed from parent site in the
US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home
nurse professional or deputy at the local site and reported immediately to the parent site
for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.
For logistical reasons home infusions will be permitted as long as AEs, infusion details and
concomitants medications are reported directly to the parent site by an authorised deputy
according to local guidelines. A Safety Review Committee (SRC) consisting of an independent
advisor, the medical monitor, and the principal investigator will be established to review
AEs and laboratory data throughout the study.
Cyclo(TM)) in patients with NPC‐1 to evaluate long‐term safety and efficacy by providing
continued access to treatment following completion of study CTD‐TCNPC‐101. Patients will
receive medication at their home or local site under the supervision of a home nurse
professional or at the parent site under the supervision of the site principal investigator.
Patients will visit the parent site for safety and efficacy evaluations after receiving
treatment for 3 months in the extension protocol.
Thereafter, visits to the parent site will be every 6 months until cessation. The study will
continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient
discontinuation. All patients who complete study CTD‐TCNCP‐101 and pass the screening
criteria will be eligible for this study. The study will be directed from parent site in the
US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home
nurse professional or deputy at the local site and reported immediately to the parent site
for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.
For logistical reasons home infusions will be permitted as long as AEs, infusion details and
concomitants medications are reported directly to the parent site by an authorised deputy
according to local guidelines. A Safety Review Committee (SRC) consisting of an independent
advisor, the medical monitor, and the principal investigator will be established to review
AEs and laboratory data throughout the study.
Inclusion Criteria:
- Completion of study CTD‐TCNPC‐101 with no safety concerns at the final visit
- Negative urine pregnancy test for females of child bearing potential
- Patients must be legally resident in the USA with access to healthcare
- Written, informed consent
Exclusion Criteria:
- Inability to comply with the proposed protocol assessments or any uncertainty about
their ability to give meaningful, informed consent (legal guardian may give consent
with subject assent)
- Concurrent medical conditions representing a contraindication to any of the study
medications
- Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate
(eGFR) < 60mL/min/1.73m2
- Clinical evidence of acute liver disease including symptoms of jaundice or right upper
quadrant pain or International Normalised Ratio (INR) >1.8
- Male patients and female patients of childbearing potential who are not willing to use
appropriate birth control (i.e. double barrier birth control) from enrollment until
the follow‐up visit
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