Clinical trial NCT03196765: 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)
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
PHASE2
Conditions: X-Linked Adrenoleukodystrophy
Keywords: X-ALD
Lead sponsor: NeuroVia, Inc.
Sponsor class: INDUSTRY
First posted: 2017-06-23
Enrollment: 0
Sex: MALE
Minimum age (years): 4
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Intervention model: SEQUENTIAL
Intervention model description: Sequential dose escalation
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 2 years
Secondary outcomes:
measure: Area under the curve concentration of NV1205 in plasma
timeFrame: 12 weeks
Other outcomes:
measure: Loes score
timeFrame: 2 years
Locations:
city: Buenos Aires
country: Argentina
facility: Hospital Austral
city: Buenos Aires
country: Argentina
facility: Hospital General de ninos Pedro de Elizalde
city: Clayton
country: Australia
facility: Monash Health
state: Victoria
city: Santiago
country: Chile
facility: Hospital Clínico San Borja Arriarán
city: Santiago
country: Chile
facility: Hospital Dr. Luis Calvo Mackenna
city: Bogotá
country: Colombia
facility: Fundacion Cardioinfantil
city: Paris
country: France
facility: Hôpital Bicêtre - Paris Sud
city: Moscow
country: Russian Federation
facility: Endocrinology Research Center
city: Moscow
country: Russian Federation
facility: Moscow Morozov's Children Clinical Hospital
city: Saint Petersburg
country: Russian Federation
facility: Saint Petersburg State Pediatric Medical University
city: Kiev
country: Ukraine
facility: National Children's Specialized Hospital 'OKHMATDET'
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Manchester
country: United Kingdom
facility: Manchester Children's Hospital
Study officials:
affiliation: NeuroVia, Inc.
name: John Henderson, MD
role: STUDY_DIRECTOR
References: