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

Sponsor
NeuroVia, Inc. (Industry)
Overall Status
Withdrawn
CT.gov ID
NCT03196765
Collaborator
(none)
0
Enrollment
13
Locations
28
Anticipated Duration (Months)
0
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

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
  • 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 Design

Study Type:
Interventional
Actual Enrollment :
0 participants
Intervention Model:
Sequential Assignment
Intervention Model Description:
Sequential dose escalationSequential 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)
Anticipated Study Start Date :
Aug 1, 2018
Anticipated Primary Completion Date :
Dec 1, 2020
Anticipated Study Completion Date :
Dec 1, 2020

Outcome Measures

Primary Outcome Measures

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [2 years]

    Adverse events

Secondary Outcome Measures

  1. 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

Other Outcome Measures

  1. Loes score [2 years]

    Percent change from baseline in brain lesions assessed as Loes score will be calculated

Eligibility Criteria

Criteria

Ages Eligible for Study:
4 Years to 18 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Hospital Austral Buenos Aires Argentina
2 Hospital General de ninos Pedro de Elizalde Buenos Aires Argentina
3 Monash Health Clayton Victoria Australia 3168
4 Hospital Clínico San Borja Arriarán Santiago Chile
5 Hospital Dr. Luis Calvo Mackenna Santiago Chile
6 Fundacion Cardioinfantil Bogotá Colombia
7 Hôpital Bicêtre - Paris Sud Paris France
8 Endocrinology Research Center Moscow Russian Federation
9 Moscow Morozov's Children Clinical Hospital Moscow Russian Federation
10 Saint Petersburg State Pediatric Medical University Saint Petersburg Russian Federation
11 National Children's Specialized Hospital 'OKHMATDET' Kiev Ukraine
12 Great Ormond Street Hospital for Children London United Kingdom
13 Manchester Children's Hospital Manchester United Kingdom

Sponsors and Collaborators

  • NeuroVia, Inc.

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
NeuroVia, Inc.
ClinicalTrials.gov Identifier:
NCT03196765
Other Study ID Numbers:
  • NV1205-009
First Posted:
Jun 23, 2017
Last Update Posted:
Mar 7, 2019
Last Verified:
May 1, 2018
Individual Participant Data (IPD) Sharing Statement:
No
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

Study Results

No Results Posted as of Mar 7, 2019