Phase 2 N=8 Treatment

Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP

Molybdenum Cofactor Deficiency, Type A

Bottom Line

View on ClinicalTrials.gov: NCT02047461 ↗

Enrolled (actual)

Serious AEs

100.0%

Results posted

Oct 2023

Primary outcome: Primary: Safety of ORGN001 (Formerly ALXN1101) — 1; 5; 6; 2 events

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ORGN001 (formerly ALXN1101) (Drug)
Age: Pediatric, Adult, Older Adult
Sex: All
Sponsor: Origin Biosciences
Primary completion: Aug 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety of ORGN001 (Formerly ALXN1101)	1; 5; 6; 2; 2; 1	—
SECONDARY Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)	3.95; 0; 669.00; 36.00; 770.00; 699.50	—
SECONDARY S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time	21.1; -1.7; -7.8; -2.2; 2.8; -3.0	—
SECONDARY Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination	3; 5; 4; 4; 4; 4	—
SECONDARY Long-term Safety of ORGN001 (Formerly ALXN1101)	3; 4; 3; 3; 3; 3	—

Summary

This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.

Eligibility Criteria

Inclusion Criteria

Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
Currently treated with rcPMP infusions

Exclusion Criteria

Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02047461). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.