Childhood Cerebral Adrenoleukodystrophy (CCALD)
Conditions
Interventions
Product Name: Sobetirome
Product Code: NV1205
Pharmaceutical Form: Oral solution
INN or Proposed INN: Sobetirome
CAS Number: 211110-63-3
Sponsors
Neurov Acquisition LLC
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Males =4 and 0 and =15 4. Not eligible for HSCT, does not have access to HSCT, or opts not to receive HSCT, and is not expected to receive HSCT 5. VLCFA levels above normal limit in plasma or blood spot test a. For subjects not on VLCFA lowering agents such as Lorenzo’s oil, a test within 3 months of enrollment is acceptable. b. Patients on VLCFA lowering agents such as Lorenzo’s oil must stop the medication and have high VLCFA levels before enrollment 6. Signed informed consent by subject’s parent(s)/legally acceptable representative(s). In addition, signed children’s assent form according to local requirements. 7. Use of adequate contraception for subjects of reproductive potential during the study and for 93 days after the last dose of the study drug a. Adequate contraception include vasectomy, true abstinence when this is in line with the preferred and usual lifestyle of the subject or consistent use of condom by subjects, or the use of effective contraceptive methods such as hormonal contraception, intrauterine device or hormone-releasing system, or bilateral tubal ligations by subject’s partners. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception. Are the trial subjects under 18? yes Number of subjects for this age range: 6 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Electrocardiographic (ECG) evidence of a previous myocardial infarction (MI) or ECG changes that likely represent cardiac ischemia, an unstable cardiac condition within the last 3 months (e.g., MI, revascularization, congestive heart failure, acute coronary syndrome), or ventricular tachycardia, unexplained syncope or syncope due to a cardiac etiology, long QT syndrome, or a family history of sudden cardiac death 2. History of cerebrovascular accident 3. QTcF >450 msec; significant ST-T wave changes that make interpretation of the QT interval challenging. PR >240 msec; resting heart rate 100 beats per minute 4. History of cancer within the past 5 years 5. Expected to initiate a major dietary change known to affect levels of VLCFA 6. Use of prohibited medications, including QTc prolonging drugs, oncologic chemotherapy for cancer treatment, metformin, statins, or Lorenzo's Oil within 14 days of first dose or at least 5.5 known half-lives of the relevant medication 7. Use of any investigational drug or procedures within 30 days of first dose of NV1205 8. Evidence of cataract development 9. Other significant medical conditions judged by the Investigator to preclude entry 10. HSCT recipient or receiving conditioning treatment for HSCT
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary: To evaluate the safety and pharmacokinetics of NV1205 in pediatric subjects diagnosed with CCALD. ; Secondary Objective: Exploratory: To assess the effect of NV1205 on expression of the ABCD2 gene, levels of very long chain fatty acids (VLCFAs), beta oxidation rate of VLCFA, markers of lipid peroxidation, brain lesions as measured by magnetic resonance imaging (MRI), and brain metabolic status as measured by magnetic resonance spectroscopy (MRS). ; Primary end point(s): Safety Endpoints: • Adverse events • Thyroid function tests • Liver function tests • Markers of bone metabolism • Hematology and chemistry laboratory tests • 12 lead electrocardiogram (ECG) evaluation ;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacodynamic (PD) Endpoints: • Spectroscopically measured concentration of N-acetylaspartate (NAA), choline, and creatine in brain • Plasma VLCFA • Markers of oxidative stress in plasma and urine. MRI and Clinical Endpoints: • Brain MRI (Loes score) and presence of gadolinium-enhanced lesions • Neurological function score ;Timepoint(s) of evaluation of this end point: 2 years | — |
Countries
Argentina, Australia, Chile, Colombia, France, Russian Federation, Ukraine, United Kingdom
Contacts
Public ContactPresident
Neurov Acquisition LLC
Outcome results
None listed