Trial Outcomes & Findings for Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A (NCT NCT02629393)
NCT ID: NCT02629393
Last Updated: 2023-10-17
Results Overview
Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
5 participants
Primary outcome timeframe
Through last observation (average of 24 months)
Results posted on
2023-10-17
Participant Flow
Participant milestones
| Measure |
Patients Treated With ORGN001 (Formerly ALXN1101)
All patients who received at least one dose of ORGN001 (formerly ALXN1101)
|
|---|---|
|
Overall Study
STARTED
|
5
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Patients Treated With ORGN001 (Formerly ALXN1101)
All patients who received at least one dose of ORGN001 (formerly ALXN1101)
|
|---|---|
|
Overall Study
MOCD Type A not genetically confirmed
|
2
|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
Baseline characteristics by cohort
| Measure |
Patients Treated With ORGN001 (Formerly ALXN1101)
n=3 Participants
All patients who received at least one dose of ORGN001 (formerly ALXN1101)
|
|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
|
Age, Continuous
|
1 days old
n=93 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
Norway
|
1 participants
n=93 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=93 Participants
|
|
Region of Enrollment
Israel
|
1 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Through last observation (average of 24 months)Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.
Outcome measures
| Measure |
ORGN001 (Formerly ALXN1101)
n=3 Participants
ORGN001 (formerly ALXN1101)
|
|---|---|
|
Overall Survival
|
3 Participants
|
SECONDARY outcome
Timeframe: At Month 12 visitNumber of patients who can feed orally
Outcome measures
| Measure |
ORGN001 (Formerly ALXN1101)
n=3 Participants
ORGN001 (formerly ALXN1101)
|
|---|---|
|
Feeding Pattern
Oral feeding at Baseline · Able to feed orally
|
2 Participants
|
|
Feeding Pattern
Oral feeding at Baseline · Not able to feed orally
|
1 Participants
|
|
Feeding Pattern
Oral feeding at Month 12 · Able to feed orally
|
3 Participants
|
|
Feeding Pattern
Oral feeding at Month 12 · Not able to feed orally
|
0 Participants
|
Adverse Events
Patients Treated With ORGN001 (Formerly ALXN1101)
Serious events: 4 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Patients Treated With ORGN001 (Formerly ALXN1101)
n=5 participants at risk
All patients who received at least one dose of ORGN001 (formerly ALXN1101)
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Apneoa
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Complication associated with device
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Bacteraemia
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Gastroenteritis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Pneumonia
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Catheter site swelling
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Viral tonsillitis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Viral infection
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Gastrointestinal viral
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Surgical and medical procedures
Central venous catheterisation
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Product Issues
Device leakage
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Cardiac disorders
Cardiac failure
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Nervous system disorders
Seizures
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Device related sepsis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Device related infection
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
Other adverse events
| Measure |
Patients Treated With ORGN001 (Formerly ALXN1101)
n=5 participants at risk
All patients who received at least one dose of ORGN001 (formerly ALXN1101)
|
|---|---|
|
Renal and urinary disorders
Haematuria
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Nasopharyngitis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Oral herpes
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Conjunctivitis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Pneumonia
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
40.0%
2/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
40.0%
2/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Gastrointestinal disorders
Anal fissure
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Eye disorders
Conjunctival haemorrhage
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Pathogen resistance
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Blood and lymphatic system disorders
Anaemia
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Pyrexia
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Catheter site infection
|
40.0%
2/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Gastrointestinal disorders
Diarrhoea
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Congenital, familial and genetic disorders
Chiari network
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Gastroenteritis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
2/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Investigations
Staphylococcus test positive
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Injury, poisoning and procedural complications
Fall
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Catheter site rash
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Injury, poisoning and procedural complications
Contusion
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Otitis media acute
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Catheter site erythema
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Catheter site haemorrhage
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
General disorders
Catheter site swelling
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Viral infection
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Tonsillitis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
COVID-19
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Cardiac disorders
Cardiac failure
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Congenital, familial and genetic disorders
Ventricular septal defect
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Eye disorders
Eye discharge
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Seizure
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
|
Infections and infestations
Bronchitis
|
20.0%
1/5 • Adverse Event data were collected from Day 1 with ORGN001 treatment until study completion, up to Month 36 or study termination or completion.
All-cause mortality is zero in this study because there were no deaths that occurred in patients with confirmed MOCD Type A. All SAE data presented.
|
Additional Information
Business Development and Operations
Origin Biosciences (affiliate of BridgeBio)
Phone: 650-391-9740
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Institution/Investigator shall have the right to publish or present the results of Institution's and Investigator's activities including that Study Data which was obtained or derived at Institution. Institution/Investigator agree to submit any proposed publication/presentation to Sponsor for review at least 60 days prior to submitting any such proposed publication. Within 30 days of its receipt, Sponsor shall advise if any changes are needed to protect confidentiality.
- Publication restrictions are in place
Restriction type: OTHER