Phase 2
Completed N=8
Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
Molybdenum Cofactor Deficiency, Type A
Source: ClinicalTrials.gov NCT02047461 ↗
Enrolled (actual)
8
Serious AEs
100.0%
Results posted
Oct 2023
Primary outcomePrimary: Safety of ORGN001 (Formerly ALXN1101) — 1; 5; 6; 2 events
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.
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 | — |
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.
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. Informational only — not medical advice.