Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

• Register: ClinicalTrials.gov
• Last refreshed on: 8 January 2024
• Main ID: NCT03879655
• Date of registration: 12/12/2018
• Prospective Registration: Yes
• Primary sponsor: Mandos LLC
• Public title: Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
• Scientific title: A Phase 2B/3 Open-label Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 Disease Previously Treated Under Protocol VTS301
• Date of first enrolment: December 2, 2019
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT03879655
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Costa Rica

Contacts

• Name: Clinical Study Lead
• Affiliation: Mandos LLC

Key inclusion & exclusion criteria

Key Inclusion Criteria:

To be eligible to participate in the study, at the Baseline Visit (except as noted below):

1. 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.

2. Participant, in the opinion of the Principal Investigator, should continue treatment
with VTS-270.

3. 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.

4. 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.

Key Exclusion Criteria:

A participant is ineligible for study participation if, at the Baseline Visit:

1. Participants discontinued from Study VTS301 for AEs.

2. Participant has an unresolved serious adverse event (SAE) for which treatment with
VTS-270 has been halted.

3. Female participants who are pregnant or nursing.

4. Participants with suspected infection of the central nervous system or any systemic
infection.

5. Participants with a spinal deformity that could impact the ability to perform a LP.

6. Participants with a skin infection in the lumbar region within 2 months of study
entry.

7. Any of the following laboratory abnormalities at the Baseline Visit:

1. Neutropenia, defined as an absolute neutrophil count of less than 1.5 ×
10^9/liter (L).

2. Thrombocytopenia (platelet count of less than 75 × 10^9/L).

3. Activated partial thromboplastin time or prothrombin time prolonged by greater
than 1.5 × the upper limit of normal (ULN) or known history of a bleeding
disorder.

4. Aspartate aminotransferase or alanine aminotransferase (ALT) greater than 4 ×
ULN.

5. Anemia: hemoglobin greater than 2 standard deviations below normal for age and
gender.

6. Estimated glomerular filtration rate less than 60 milliliters (mL)/minute/1.73
square meter (m^2) calculated using the modified Schwartz formula (Schwartz et
al., 2009) for participants aged 4 through 17 years old or using the Chronic
Kidney Disease Epidemiology Collaboration equation formula for participants aged
18 years or older.

8. Evidence of obstructive hydrocephalus or normal pressure hydrocephalus.

9. Recent use of anticoagulants (in past 2 weeks prior to first dose [Study Day 0]).

10. Active pulmonary disease, oxygen requirement, or clinically significant history of
decreased blood oxygen saturation, pulmonary therapy, or requiring active suction.

11. Participants who, in the opinion of the investigator, are unable to comply with the
protocol or have medical conditions that would potentially increase the risk of
participation.



Age minimum: 4 Years
Age maximum: 21 Years
Gender: All

Health Condition(s) or Problem(s) studied

Niemann-Pick Disease, Type C

Intervention(s)

Drug: VTS-270

Primary Outcome(s)

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time Frame: Baseline up to Week 156]

Secondary ID(s)

VTS-270-302

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/12/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03879655