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

NCT03196765 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: NV1205-009
• Study Type: interventional
• Target: N/A
• Locations: 8 countries
• Sites: 13

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.

Conditions

X-Linked Adrenoleukodystrophy

Keywords

X-ALD

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  Adverse events

  2 years

Secondary Outcomes (1)

• Area under the curve concentration of NV1205 in plasma

  12 weeks

Other Outcomes (1)

• Loes score

  2 years

Interventions

Sobetirome (NV1205) DRUG

Once a day oral dose of the study drug

Also known as: NV1205

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• NeuroVia, Inc.: lead

Study Sites (13)

Hospital Austral

Buenos Aires, Argentina

Location

Hospital General de ninos Pedro de Elizalde

Buenos Aires, Argentina

Location

Monash Health

Clayton, Victoria, 3168, Australia

Location

Hospital Clínico San Borja Arriarán

Santiago, Chile

Location

Hospital Dr. Luis Calvo Mackenna

Santiago, Chile

Location

Fundacion Cardioinfantil

Bogotá, Colombia

Location

Hôpital Bicêtre - Paris Sud

Paris, France

Location

Endocrinology Research Center

Moscow, Russia

Location

Moscow Morozov's Children Clinical Hospital

Moscow, Russia

Location

Saint Petersburg State Pediatric Medical University

Saint Petersburg, Russia

Location

National Children's Specialized Hospital 'OKHMATDET'

Kiev, Ukraine

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Manchester Children's Hospital

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Adrenoleukodystrophy

Interventions

GC 1 compound

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• John Henderson, MD

  NeuroVia, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential dose escalation

Study Record Dates

• First Submitted: May 5, 2017
• First Posted: June 23, 2017
• Study Start: August 1, 2018
• Primary Completion: December 1, 2020
• Study Completion: December 1, 2020
• Last Updated: March 7, 2019

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

UA (1); CO (1); CL (2); GB (2); RU (3); AU (1); FR (1); AR (2)