Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 (NCT03879655) | Clinical Trial Compass
TerminatedPhase 2/3
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
Stopped: Terminated by previous Sponsor decision
Costa Rica2 participantsStarted 2019-12-02
Plain-language summary
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B \[NCT02534844\] and Part C \[NCT04958642\]) with neurologic manifestations of Niemann-Pick Type C1 (NPC1) disease.
Who can participate
Age range4 Years – 21 Years
SexALL
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Inclusion criteria
✓. Participant completed Part B of Study VTS301 (defined as having completed Visit 27/Week 52 or completed at least through Visit 13/Week 24 and required rescue option) and is continuing in Part C of Study VTS301.
✓. Participant, in the opinion of the Principal Investigator, should continue treatment with VTS-270.
✓. Females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception and must agree to continue use of this method for the duration of the study and for 30 days after participation in the study. Acceptable methods of contraception include barrier method with spermicide, intrauterine device, steroidal contraceptive in conjunction with a barrier method, abstinence, or same-sex partner.
✓. Participant or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to participate must also be sought from minor children.
Exclusion criteria
✕. Participants discontinued from Study VTS301 for AEs.
✕. Participant has an unresolved serious adverse event (SAE) for which treatment with VTS-270 has been halted.
✕. Female participants who are pregnant or nursing.
✕. Participants with suspected infection of the central nervous system or any systemic infection.
✕. Participants with a spinal deformity that could impact the ability to perform a LP.
✕. Participants with a skin infection in the lumbar region within 2 months of study entry.
What they're measuring
1
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)