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

NCT03879655 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: VTS-270-302
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Niemann-Pick Disease, Type C

Keywords

Niemann-Pick; NPC

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

  Baseline up to Week 156

Study Arms (1)

VTS-270

EXPERIMENTAL

Eligible participants who transition into this study will receive treatment with VTS-270 at the last dose level administered in Study VTS301, administered IT via LP infusion every 2 weeks, for up to a total duration of 3 years or until the investigator considers VTS-270 to be no longer beneficial to the participant, VTS-270 receives marketing authorization, or the VTS-270 development program is discontinued.

Drug: VTS-270

Interventions

VTS-270 DRUG

Administered IT via LP infusion of VTS-270

Also known as: 2-hydroxypropyl-β-cyclodextrin, Cyclodextrin, Adrabetadex

VTS-270

Eligibility Criteria

• Age: 4 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• A participant is ineligible for study participation if, at the Baseline Visit:
• 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.
• Any of the following laboratory abnormalities at the Baseline Visit:
• Neutropenia, defined as an absolute neutrophil count of less than 1.5 × 10\^9/liter (L).
• Thrombocytopenia (platelet count of less than 75 × 10\^9/L).
• 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.
• Aspartate aminotransferase or alanine aminotransferase (ALT) greater than 4 × ULN.
• Anemia: hemoglobin greater than 2 standard deviations below normal for age and gender.
• 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.
• Evidence of obstructive hydrocephalus or normal pressure hydrocephalus.

Sponsors & Collaborators

• Mandos LLC: lead

Study Sites (1)

Hospital Clinica Biblica

San José, 10101, Costa Rica

Location

MeSH Terms

Conditions

Niemann-Pick Disease, Type C

Interventions

Cyclodextrins; 2-Hydroxypropyl-beta-cyclodextrin

Condition Hierarchy (Ancestors)

Niemann-Pick Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Macrocyclic Compounds; Polycyclic Compounds; Dextrins; Starch; Dietary Carbohydrates; Carbohydrates; Glucans; Polysaccharides; beta-Cyclodextrins

Limitations and Caveats

Study was terminated by the Sponsor and only 2 participants were enrolled in the study.

Results Point of Contact

• Title: Executive Vice President, Regulatory Affairs
• Organization: Mandos, LLC

jspinella@mandoshealth.com

619-905-0489

Study Officials

• Clinical Study Lead

  Mandos LLC

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2018
• First Posted: March 19, 2019
• Study Start: December 2, 2019
• Primary Completion: October 18, 2021
• Study Completion: October 18, 2021
• Last Updated: December 26, 2023
• Results First Posted: December 26, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

CO (1)