A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (formerly ALXN1101) in Pediatric Patients with Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated with Recombinant Escherichia coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)

A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (formerly ALXN1101) in Pediatric Patients with Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated with Recombinant Escherichia coli-derived Cyclic Pyranopterin Monophosphate (rcPMP) - ALXN1101-MCD-201

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON55625

Enrollment

Registered

2014-01-03

Start date

2015-04-30

Completion date

Unknown

Last updated

2024-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

and aldehyde oxidase Combined deficiency of sulfite oxidase xanthine dehydrogenase

Interventions

ORGN001 (formerly ALXN1101) is a synthetic form of cPMP. Patients will begin IV infusions of ORGN001 (formerly ALXN1101) at the same dose as their current dose of rcPMP. After the first 2 months of

Eligibility

Age

No minimum to 17 Years

Inclusion criteria

Inclusion criteria: 1) Male of female patients with genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation) and who are currently treated with rcPMP infusions. 2) Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures.

Exclusion criteria

Exclusion criteria: Current or planned treatment with another investigational drug or device, with the exception of rcPMP treatment through Day -1

Design outcomes

Primary

Measure	Time frame
Safety over the first 6 months of treatment	—

Secondary

Measure	Time frame
Safety: • Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) • Incidence of clinical laboratory abnormalities • Change from baseline in clinical laboratory assessments • Change from baseline in clinical findings from physical examination • Change from baseline in vital sign measurements • Change from baseline in EEG results Efficacy: • Change from baseline in urine and blood SSC levels • Change from baseline in clinical findings from neurologic examination • Change from baseline in age-appropriate motor and cognitive assessments (Bayley Scales of Infant Development-Third Edition [Bayley-III], The Gross Motor Function Classification System [GMFCS], Wechsler Preschool and Primary Scale of Intelligence [WPPSI]) • Change from baseline in seizure frequency • Change from baseline in neuroimaging • Changes in growth parameters (body weight, body length, head circumference) • Change from baseline in feeding patterns Pharmacokinetic: • PK parameters of ORGN001 (formerly ALXN1101) including, but not limited to, maximum observed plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), area under the plasma concentration-time curve (AUC) and if possible, terminal half-life (t*), and dose linearity Exploratory: • Change from baseline in MoCD-associated urine and blood biomarker levels including, but not limited to, uric acid and xanthine	—

Measure

Time frame

Safety: • Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) • Incidence of clinical laboratory abnormalities • Change from baseline in clinical laboratory assessments • Change from baseline in clinical findings from physical examination • Change from baseline in vital sign measurements • Change from baseline in EEG results Efficacy: • Change from baseline in urine and blood SSC levels • Change from baseline in clinical findings from neurologic examination • Change from baseline in age-appropriate motor and cognitive assessments (Bayley Scales of Infant Development-Third Edition [Bayley-III], The Gross Motor Function Classification System [GMFCS], Wechsler Preschool and Primary Scale of Intelligence [WPPSI]) • Change from baseline in seizure frequency • Change from baseline in neuroimaging • Changes in growth parameters (body weight, body length, head circumference) • Change from baseline in feeding patterns Pharmacokinetic: • PK parameters of ORGN001 (formerly ALXN1101) including, but not limited to, maximum observed plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), area under the plasma concentration-time curve (AUC) and if possible, terminal half-life (t*), and dose linearity Exploratory: • Change from baseline in MoCD-associated urine and blood biomarker levels including, but not limited to, uric acid and xanthine

—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)