Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

Phase 1

Withdrawn

• Conditions: X-Linked Adrenoleukodystrophy

• Registration Number: NCT03196765
• Lead Sponsor: NeuroVia, Inc.

Brief Summary

This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.

Detailed Description

This study is to evaluate the safety, pharmacokinetics, and efficacy of NV1205 in pediatric patients diagnosed with childhood cerebral adrenoleukodystrophy (CCALD).

The study consists of:

* Screening period: within 30 days of first dose

* Main treatment period of 12 weeks (Part 1- from Screening to Week 12)

* Long Term Treatment (LTT) period (Part 2- Week 13 through Week 96)

In Part 1, subjects will have an initial 4-week treatment period at the assigned dose and, if no safety concerns are noted, subjects continue for another 8 weeks of extended safety assessment.

There will be several cohorts of subjects enrolled. After each Cohort has completed the 4-week initial safety assessment, the safety data will be reviewed by an independent Data Safety Monitoring Board (DSMB) and, subject to DSMB recommendation, Cohort 2 will be enrolled and receive the next dose level. After Cohort 2 has completed 4 weeks of treatment, the DSMB will review all available safety data (Cohorts 1 and 2) and, subject to DSMB recommendation, Cohort 3 will be enrolled and receive the next dose level. Additional Cohorts may be enrolled at the recommendation of the DSMB with an incremental dose increase.

In Part 2, subjects will continue to receive treatment in the LTT period of the study.

Study Timeline

• Study First Submitted: 2017-05-05
• Study First Submitted QC: 2017-06-21
• Study First Posted: 2017-06-23
• Status Verified: 2018-05
• Study Start: 2018-08
• Last Update Submitted: 2019-03-06
• Last Update Posted: 2019-03-07
• Primary Completion: 2020-12-01
• Study Completion: 2020-12-01

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: Male
• Target Recruitment: Not specified

Inclusion Criteria

1. Males ≥4 years and <18 years of age
2. CCALD diagnosis confirmed by genetic testing
3. Loes score of >0 and ≤15
4. Patients on VLCFA lowering agents such as Lorenzo's oil must stop the medication and have high VLCFA levels before enrollment

Exclusion Criteria

• Significant medical conditions such as heart, thyroid, or liver disease
• HSCT recipients

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	2 years	Adverse events

Secondary Outcome Measures

Name	Time	Method
Area under the curve concentration of NV1205 in plasma	12 weeks	A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration

Trial Locations

Locations (13)

Great Ormond Street Hospital for Children; 🇬🇧 London, United Kingdom

Hospital Clínico San Borja Arriarán; 🇨🇱 Santiago, Chile

Saint Petersburg State Pediatric Medical University; 🇷🇺 Saint Petersburg, Russian Federation

Hospital Dr. Luis Calvo Mackenna; 🇨🇱 Santiago, Chile

Endocrinology Research Center; 🇷🇺 Moscow, Russian Federation

Manchester Children's Hospital; 🇬🇧 Manchester, United Kingdom

Hospital General de ninos Pedro de Elizalde; 🇦🇷 Buenos Aires, Argentina

Hospital Austral; 🇦🇷 Buenos Aires, Argentina

Monash Health; 🇦🇺 Clayton, Victoria, Australia

Fundacion Cardioinfantil; 🇨🇴 Bogotá, Colombia

Moscow Morozov's Children Clinical Hospital; 🇷🇺 Moscow, Russian Federation

Hôpital Bicêtre - Paris Sud; 🇫🇷 Paris, France

National Children's Specialized Hospital 'OKHMATDET'; 🇺🇦 Kiev, Ukraine