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Enzyme Replacement Therapy

ORGN001 (formerly ALXN1101) for Molybdenum Cofactor Deficiency

Phase 2 & 3
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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through last observation (average of 24 months)
Awards & highlights

Study Summary

This trial is studying a drug called ORGN001 in newborn babies with a rare genetic disorder called MoCD Type A.

Eligible Conditions
  • Molybdenum Cofactor Deficiency

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through last observation (average of 24 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and through last observation (average of 24 months) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Overall Survival
Secondary outcome measures
Feeding Pattern

Side effects data

From 2022 Phase 2 & 3 trial • 5 Patients • NCT02629393
40%
Hypoglycaemia
40%
Catheter site infection
40%
Hypocalcaemia
40%
Vomiting
20%
Complication associated with device
20%
Pyrexia
20%
Viral infection
20%
Device related infection
20%
Gastrointestinal viral
20%
Device leakage
20%
Anal fissure
20%
Catheter site rash
20%
Nasopharyngitis
20%
Conjunctival haemorrhage
20%
Chiari network
20%
Ventricular septal defect
20%
Pneumonia
20%
Catheter site swelling
20%
Cardiac failure
20%
Dermatitis
20%
Eye discharge
20%
Seizure
20%
Central venous catheterisation
20%
Pathogen resistance
20%
Haematuria
20%
Gastroenteritis
20%
Tonsillitis
20%
Hyperbilirubinaemia
20%
Viral tonsillitis
20%
Seizures
20%
Otitis media acute
20%
COVID-19
20%
Catheter site haemorrhage
20%
Bronchitis
20%
Rhinorrhea
20%
Diarrhoea
20%
Skin laceration
20%
Bacteraemia
20%
Apneoa
20%
Cough
20%
Oral herpes
20%
Anaemia
20%
Fall
20%
Eczema
20%
Staphylococcus test positive
20%
Conjunctivitis
20%
Respiratory syncytial virus infection
20%
Device related sepsis
20%
Catheter site erythema
20%
Contusion
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
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
112 Total Patients Enrolled
Liza Squires, M.D.Study DirectorOrigin Biosciences

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
2016. "Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02629393.
~1 spots leftby Apr 2025
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