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

X-Linked Adrenoleukodystrophy Clinical Trial

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)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03196765
• Other study ID #: NV1205-009
• Status: Withdrawn
• Phase: Phase 1/Phase 2
• Start date: August 2018
• Est. completion date: December 1, 2020

Study information

• Verified date: May 2018
• Source: NeuroVia, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 1, 2020
• Est. primary completion date: December 1, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• X-Linked Adrenoleukodystrophy

Intervention

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

Locations

Country	Name	City	State
Argentina	Hospital Austral	Buenos Aires
Argentina	Hospital General de ninos Pedro de Elizalde	Buenos Aires
Australia	Monash Health	Clayton	Victoria
Chile	Hospital Clínico San Borja Arriarán	Santiago
Chile	Hospital Dr. Luis Calvo Mackenna	Santiago
Colombia	Fundacion Cardioinfantil	Bogotá
France	Hôpital Bicêtre - Paris Sud	Paris
Russian Federation	Endocrinology Research Center	Moscow
Russian Federation	Moscow Morozov`s Children Clinical Hospital	Moscow
Russian Federation	Saint Petersburg State Pediatric Medical University	Saint Petersburg
Ukraine	National Children's Specialized Hospital 'OKHMATDET'	Kiev
United Kingdom	Great Ormond Street Hospital for Children	London
United Kingdom	Manchester Children's Hospital	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
NeuroVia, Inc.

Countries where clinical trial is conducted

Argentina,  Australia,  Chile,  Colombia,  France,  Russian Federation,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Loes score	Percent change from baseline in brain lesions assessed as Loes score will be calculated	2 years
Primary	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	Adverse events	2 years
Secondary	Area under the curve concentration of NV1205 in plasma	A sparse sampling approach will be utilized to estimate a 24-hr area under the curve of NV1205 plasma concentration	12 weeks