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Enzyme Replacement Therapy

ORGN001 (formerly ALXN1101) for Molybdenum Cofactor Deficiency

Phase 2
Waitlist Available
Research Sponsored by Origin Biosciences
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
Currently treated with rcPMP infusions
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to month 24 for all patients plus additional follow-up to month 90
Awards & highlights

Study Summary

This trial will have three stages: a screening period, a 6-month treatment period, and a long-term extension period that is expected to last around 72 months.

Eligible Conditions
  • Molybdenum Cofactor Deficiency

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to month 24 for all patients plus additional follow-up to month 90
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 24 for all patients plus additional follow-up to month 90 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety of ORGN001 (Formerly ALXN1101)
Secondary outcome measures
Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination
Long-term Safety of ORGN001 (Formerly ALXN1101)
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
+1 more

Side effects data

From 2022 Phase 2 & 3 trial • 5 Patients • NCT02629393
40%
Vomiting
40%
Hypoglycaemia
40%
Hypocalcaemia
40%
Catheter site infection
20%
Device related infection
20%
Haematuria
20%
Bacteraemia
20%
Gastrointestinal viral
20%
Seizures
20%
Catheter site haemorrhage
20%
Gastroenteritis
20%
Bronchitis
20%
COVID-19
20%
Eczema
20%
Pathogen resistance
20%
Staphylococcus test positive
20%
Device related sepsis
20%
Conjunctival haemorrhage
20%
Hyperbilirubinaemia
20%
Seizure
20%
Viral infection
20%
Nasopharyngitis
20%
Catheter site swelling
20%
Pneumonia
20%
Anaemia
20%
Pyrexia
20%
Device leakage
20%
Tonsillitis
20%
Fall
20%
Conjunctivitis
20%
Central venous catheterisation
20%
Skin laceration
20%
Catheter site erythema
20%
Eye discharge
20%
Oral herpes
20%
Otitis media acute
20%
Cough
20%
Diarrhoea
20%
Cardiac failure
20%
Ventricular septal defect
20%
Respiratory syncytial virus infection
20%
Rhinorrhea
20%
Chiari network
20%
Anal fissure
20%
Catheter site rash
20%
Apneoa
20%
Dermatitis
20%
Complication associated with device
20%
Contusion
20%
Viral tonsillitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Patients Treated With ORGN001 (Formerly ALXN1101)

Trial Design

1Treatment groups
Experimental Treatment
Group I: ORGN001 (formerly ALXN1101)Experimental Treatment1 Intervention
daily IV infusions
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ORGN001 (formerly ALXN1101)
2016
Completed Phase 3
~20

Find a Location

Who is running the clinical trial?

Origin BiosciencesLead Sponsor
4 Previous Clinical Trials
109 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~1 spots leftby Mar 2025