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

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ClinicalTrials.gov Identifier: NCT03196765
Recruitment Status : Recruiting
First Posted : June 23, 2017
Last Update Posted : June 28, 2018
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
X-Linked Adrenoleukodystrophy Drug: Sobetirome (NV1205) Phase 1 Phase 2

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Sequential Assignment
Intervention Model Description: Sequential dose escalation
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)
Estimated Study Start Date : June 2018
Estimated Primary Completion Date : December 1, 2020
Estimated Study Completion Date : December 1, 2020



Intervention Details:
  • Drug: Sobetirome (NV1205)
    Once a day oral dose of the study drug
    Other Name: NV1205


Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 2 years ]
    Adverse events


Secondary Outcome Measures :
  1. Area under the curve concentration of NV1205 in plasma [ Time Frame: 12 weeks ]
    A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration


Other Outcome Measures:
  1. Loes score [ Time Frame: 2 years ]
    Percent change from baseline in brain lesions assessed as Loes score will be calculated



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03196765


Contacts
Contact: Program Director 415-870-6967 patients@neurovia-inc.com

Locations
Argentina
Hospital Austral Not yet recruiting
Buenos Aires, Argentina
Hospital General de ninos Pedro de Elizalde Not yet recruiting
Buenos Aires, Argentina
Australia, Victoria
Monash Health Recruiting
Clayton, Victoria, Australia, 3168
Contact: Michael Fahey, M.D.       michael.fahey@monash.edu   
Chile
Hospital Clínico San Borja Arriarán Not yet recruiting
Santiago, Chile
Hospital Dr. Luis Calvo Mackenna Recruiting
Santiago, Chile
Contact: Constanza Ruiz Berger    56972174121    cruizberger@gmail.com   
Contact: Patricia Acuna Rosales    56982482811    patricia.ar78@gmail.com   
Principal Investigator: Ricardo Erazo Torricelli, MD         
Colombia
Fundacion Cardioinfantil Not yet recruiting
Bogotá, Colombia
France
Hôpital Bicêtre - Paris Sud Not yet recruiting
Paris, France
Russian Federation
Endocrinology Research Center Not yet recruiting
Moscow, Russian Federation
Moscow Morozov`s Children Clinical Hospital Not yet recruiting
Moscow, Russian Federation
Saint Petersburg State Pediatric Medical University Not yet recruiting
Saint Petersburg, Russian Federation
Ukraine
National Children's Specialized Hospital 'OKHMATDET' Not yet recruiting
Kiev, Ukraine
United Kingdom
Great Ormond Street Hospital for Children Not yet recruiting
London, United Kingdom
Manchester Children's Hospital Not yet recruiting
Manchester, United Kingdom
Sponsors and Collaborators
NeuroVia, Inc.
Investigators
Study Director: Masoud Mokhtarani, MD NeuroVia, Inc.

Responsible Party: NeuroVia, Inc.
ClinicalTrials.gov Identifier: NCT03196765     History of Changes
Other Study ID Numbers: NV1205-009
First Posted: June 23, 2017    Key Record Dates
Last Update Posted: June 28, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by NeuroVia, Inc.:
X-ALD

Additional relevant MeSH terms:
Adrenoleukodystrophy
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
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases