A Phase 2 interventional study of ORGN001 (formerly ALXN1101) in Molybdenum Cofactor Deficiency, Type A, sponsored by Origin Biosciences. Completed at 6 sites in 5 countries. Per ClinicalTrials.gov, last updated 2023-10-17.
Sponsored by Origin Biosciences · Phase 2, Interventional, and Treatment
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
Patients will receive daily IV infusions of ORGN001 (formerly ALXN1101) starting on Day 1. After a prescribed period, dosing will increase monthly based on defined patient safety measures. After Month 6, patients will continue daily dosing at their last tolerated dose.
Origin Biosciences is the lead sponsor of 5 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
daily IV infusions
Drug: ORGN001 (formerly ALXN1101)
IV infusion
Safety of ORGN001 (Formerly ALXN1101)
Treatment Emergent Serious Adverse Events
Time frame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints
Time frame: First 6 months at each dose level, where available
S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time
Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time.
Time frame: Baseline to Month 24 for all patients plus additional follow-up to Month 90
Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination
Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.
Time frame: Baseline to Month 24 for all patients plus additional follow-up until Month 30
Long-term Safety of ORGN001 (Formerly ALXN1101)
Change from baseline in Seizure frequency
Time frame: Baseline to Month 24 for all patients plus additional follow up until Month 72
| Milestone | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Started | 8 |
| Completed | 8 |
| Not completed | 0 |
Treatment Emergent Serious Adverse Events
| events | Patient Currently Receiving rcPMP Infusions at Baseline |
|---|---|
| Gastrointestinal disorders | 1 |
| General disorders and administration site conditions | 5 |
| Infections and infestations | 6 |
| Injury, poisoning and procedural complications | 2 |
| Metabolism and nutrition disorders | 2 |
| Musculoskeletal and connective tissue disorders | 1 |
| Nervous system disorders | 2 |
| Product issues | 1 |
| Respiratory, thoracic and mediastinal disorders | 2 |
| Skin and subcutaneous tissue disorders | 1 |
| Surgical and medical procedures | 1 |
| Vascular disorders | 2 |
ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints
| ng/mL | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Day 1 Pre-infusion (240 mcg/kg) | 3.95 ± 9.68 |
| Day 1 Pre-infusion (248 mcg/kg) | 0 ± NA |
| Day 1 EOI (240 mcg/kg) | 669.00 ± 236.06 |
| Day 1 EOI (248 mcg/kg) | 36.00 ± NA |
| Day 1 EOI (280 mcg/kg) | 770.00 ± NA |
| Day 7 EOI (240 mcg/kg) | 699.50 ± 246.72 |
| Day 7 EOI (248 mcg/kg) | 694.00 ± NA |
| Day 60 EOI (480 mcg/kg) | 880.84 ± 558.56 |
| Day 90 EOI (240 mcg/kg) | 1230.00 ± NA |
| Day 90 EOI (480 mcg/kg) | 762.00 ± NA |
| Day 90 EOI (720 mcg/kg) | 1888.33 ± 271.10 |
| Day 120 EOI (480 mcg/kg) | 2810.00 ± NA |
| Day 120 EOI (720 mcg/kg) | 671.00 ± NA |
| Day 120 EOI (960 mcg/kg) | 4213.33 ± 4520.31 |
| Day 150 EOI (720 mcg/kg) | 3810.00 ± NA |
| Day 150 EOI (960 mcg/kg) | 2045.67 ± 1009.84 |
| Day 150 EOI (1200 mcg/kg) | 3173.33 ± 656.53 |
Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time.
| umol/mmol | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Baseline | 21.1 ± 12.89 |
| Day 4 Change from Baseline | -1.7 ± 12.70 |
| Day 7 Change from Baseline | -7.8 ± 9.70 |
| Day 14 Change from Baseline | -2.2 ± 14.51 |
| Day 28 Change of Baseline | 2.8 ± 14.17 |
| Day 57 Change from Baseline | -3.0 ± 10.29 |
| Day 127 Change from Baseline | -12.2 ± 14.77 |
| Month 6 Change from Baseline | -13.6 ± 11.77 |
| Month 12 Change from Baseline | -8.6 ± 20.15 |
| Month 24 Change from Baseline | -14.3 ± 14.73 |
| Month 36 Change from Baseline | -13.2 ± 18.40 |
| Month 48 Change from Baseline | -6.7 ± 6.65 |
| Month 60 Change from Baseline | -17.9 ± 14.59 |
| Month 90 Change from Baseline | 1.7 ± 10.25 |
Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.
| Participants | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Quality of Spontaneous Movement at Baseline — Normal | 3 |
| Quality of Spontaneous Movement at Baseline — Abnormal | 5 |
| Quality of Spontaneous Movement at Month 30 — Normal | 4 |
| Quality of Spontaneous Movement at Month 30 — Abnormal | 4 |
| Dystonic at Baseline — Normal | 4 |
| Dystonic at Baseline — Abnormal | 4 |
| Dystonic at Month 30 — Normal | 4 |
| Dystonic at Month 30 — Abnormal | 4 |
| Opistonic at Baseline — Normal | 7 |
| Opistonic at Baseline — Abnormal | 1 |
| Opistonic at Month 30 — Normal | 6 |
| Opistonic at Month 30 — Abnormal | 2 |
| Truncal Tone at Baseline — Normal | 2 |
| Truncal Tone at Baseline — Abnormal | 5 |
| Truncal Tone at Month 30 — Normal | 2 |
| Truncal Tone at Month 30 — Abnormal | 6 |
| Appendicular Tone at Baseline — Normal | 2 |
| Appendicular Tone at Baseline — Abnormal | 6 |
| Appendicular Tone at Month 30 — Normal | 1 |
| Appendicular Tone at Month 30 — Abnormal | 7 |
| Deep Tendon reflexes at Baseline — Normal | 4 |
| Deep Tendon reflexes at Baseline — Abnormal | 4 |
| Deep Tendon reflexes at Month 30 — Normal | 3 |
| Deep Tendon reflexes at Month 30 — Abnormal | 5 |
| Primitive reflexes at Baseline — Normal | 5 |
| Primitive reflexes at Baseline — Abnormal | 1 |
| Primitive reflexes at Month 30 — Normal | 6 |
| Primitive reflexes at Month 30 — Abnormal | 1 |
| Clonus presence at Baseline — Normal | 7 |
| Clonus presence at Baseline — Abnormal | 1 |
| Clonus presence at Month 30 — Normal | 6 |
| Clonus presence at Month 30 — Abnormal | 2 |
| Ambulation at Baseline — Normal | 4 |
| Ambulation at Baseline — Abnormal | 4 |
| Ambulation at Month 30 — Normal | 4 |
| Ambulation at Month 30 — Abnormal | 4 |
Change from baseline in Seizure frequency
| Participants | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Screening | 3 |
| Baseline to Month 6 | 4 |
| Month 6 to Month 12 | 3 |
| Month 12 to Month 24 | 3 |
| Month 24 to Month 36 | 3 |
| Month 36 to Month 48 | 3 |
| Month 48 to Month 60 | 3 |
| Month 60 to Month 72 | 3 |
Collected over Adverse event data were collected from Day 1 with ORGN001 treatment until Month 24 for all patients; additional data included for patients treated up to Month 90. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 | 0/8 (0%) | 8/8 (100%) | 8/8 (100%) |
| Event | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Complication associated with deviceGeneral disorders | 4/8 |
| PyrexiaGeneral disorders | 2/8 |
| Catheter site infectionsInfections and infestations | 2/8 |
| Device related infectionInfections and infestations | 2/8 |
| PneumoniaInfections and infestations | 2/8 |
| Lower respiratory tract infectionInfections and infestations | 2/8 |
| Vascular device infectionInfections and infestations | 2/8 |
| Event | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| VomitingGastrointestinal disorders | 7/8 |
| PyrexiaGeneral disorders | 7/8 |
| Complication associated with deviceGeneral disorders | 6/8 |
| Viral infectionInfections and infestations | 5/8 |
| InfluenzaInfections and infestations | 4/8 |
| PneumoniaInfections and infestations | 4/8 |
| CoughRespiratory, thoracic and mediastinal disorders | 4/8 |
| DiarrhoeaGastrointestinal disorders | 3/8 |
| Lower respiratory tract infectionInfections and infestations | 3/8 |
| Otitis mediaInfections and infestations | 3/8 |
Patients currently receiving rcPMP infusions at Baseline and transitioned to ORGN001.
| Age, Categorical(Participants) | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| <=18 years | 8 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(Months) | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Mean | 45.2 ± 22.96 |
| Sex: Female, Male(Participants) | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Female | 5 |
| Male | 3 |
| Race (NIH/OMB)(Participants) | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 3 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 5 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Netherlands | 2 |
| United States | 1 |
| United Kingdom | 3 |
| Australia | 1 |
| Tunisia | 1 |
Documents are hosted by the registry — open the source record to download them.
This study is completed, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Metal Metabolism, Inborn Errors
Origin Biosciences