Safety and efficacy of ORGN001 in pediatric patients with Molybdenum Cofactor Deficiency Type A

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)

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-002701-56-GB

Enrollment

Registered

2013-12-24

Start date

2014-04-16

Completion date

Unknown

Last updated

2020-02-01

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

Conditions

Molybdenum Cofactor Deficiency (MoCD) Type A MedDRA version: 20.1 Level: PT Classification code 10069687 Term: Molybdenum cofactor deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: Cyclic Pyranopterin Monophosphate Monohydrobromide Dihydrate Product Code: ORGN001 (formerly ALXN1101) Pharmaceutical Form: Powder for solution for infusi

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation) - Currently treated with rcPMP infusions Are the trial subjects under 18? yes Number of subjects for this age range: 8 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: - 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
Main Objective: safety;Secondary Objective: PK, long-term safety and efficacy;Primary end point(s): safety;Timepoint(s) of evaluation of this end point: Trial duration	—

Secondary

Measure	Time frame
Secondary end point(s): - PK parameters - Change from baseline in urine and blood SSC levels - Change from baseline in clinical findings from neurologic examination, cognitive and motor assessment, seizure frequency, neuroimaging, growth parameters and feeding patterns ;Timepoint(s) of evaluation of this end point: Trial duration	—

Countries

Australia, Netherlands, Tunisia, United Kingdom, United States

Contacts

Public ContactHead of Clinical Operations

Origin Biosciences, Inc.

+1857 350 3841

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026