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

NCT ID: NCT03196765

Last Updated: 2019-03-07

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1/PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-08-31
• Study Completion Date: 2020-12-01

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.

Conditions

X-Linked Adrenoleukodystrophy

Study Design

• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

Sobetirome (NV1205)

Once a day oral dose of the study drug

Intervention Type: DRUG

Other Intervention Names

NV1205

Eligibility Criteria

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

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

NeuroVia, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John Henderson, MD

Role: STUDY_DIRECTOR

NeuroVia, Inc.

Locations

Hospital Austral

Buenos Aires, , Argentina

Hospital General de ninos Pedro de Elizalde

Buenos Aires, , Argentina

Monash Health

Clayton, Victoria, Australia

Hospital Clínico San Borja Arriarán

Santiago, , Chile

Hospital Dr. Luis Calvo Mackenna

Santiago, , Chile

Fundacion Cardioinfantil

Bogotá, , Colombia

Hôpital Bicêtre - Paris Sud

Paris, , France

Endocrinology Research Center

Moscow, , Russia

Moscow Morozov's Children Clinical Hospital

Moscow, , Russia

Saint Petersburg State Pediatric Medical University

Saint Petersburg, , Russia

National Children's Specialized Hospital 'OKHMATDET'

Kiev, , Ukraine

Great Ormond Street Hospital for Children

London, , United Kingdom

Manchester Children's Hospital

Manchester, , United Kingdom

Countries

Argentina; Australia; Chile; Colombia; France; Russia; Ukraine; United Kingdom

Other Identifiers

NV1205-009

Identifier Type: -

Identifier Source: org_study_id