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

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Brief Title

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

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)

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 Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 Area under the curve concentration of NV1205 in plasma

Condition

X-Linked Adrenoleukodystrophy

Intervention

Sobetirome (NV1205)


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

0

Start Date

August 2018

Completion Date

December 1, 2020

Primary Completion Date

December 1, 2020

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
      

Gender

Male

Ages

4 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

John Henderson, MD, , 

Location Countries

Argentina

Location Countries

Argentina

Administrative Informations


NCT ID

NCT03196765

Organization ID

NV1205-009


Responsible Party

Sponsor

Study Sponsor

NeuroVia, Inc.


Study Sponsor

John Henderson, MD, Study Director, NeuroVia, Inc.


Verification Date

May 2018