Molybdenum Cofactor Deficiency, Type APhase 2NCT02047461
Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
A Phase 2 study testing ORGN001 (formerly ALXN1101) in people with Molybdenum Cofactor Deficiency, Type A, run by Origin Biosciences at 6 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Molybdenum Cofactor Deficiency, Type A
- What is being tested?
- ORGN001 (formerly ALXN1101)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 6 sitesin 5 countries
- Study dates
- Apr 2014to Oct 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
- Currently treated with rcPMP infusions
Who cannot join
- Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
United Kingdom2 sites
Royal Hospital for Sick Children
Glasgow
Manchester University Hospitals NHS Foundation Trust
Manchester
United States1 site
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
Read the detailed description
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.
- Conditions
- Molybdenum Cofactor Deficiency, Type A
- Being tested
- ORGN001 (formerly ALXN1101)
- Keywords
- Molybdenum cofactor deficiency (MoCD), molybdenum cofactor (MoCo) biosynthesis, sulfite oxidase (SO), xanthine dehydrogenase, aldehyde oxidase, S sulfocysteine (SSC)
- Medical subject headings
- Molybdenum Cofactor Deficiency, Complementation Group A, Molybdenum cofactor deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ORGN001 (formerly ALXN1101)Drug
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| ORGN001 (formerly ALXN1101) | Experimental | daily IV infusions Receives: Drug: ORGN001 (formerly ALXN1101) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of ORGN001 (Formerly ALXN1101)
Treatment Emergent Serious Adverse Events
Measured: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Secondary outcomes (4)
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints
Measured: 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.
Measured: 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.
Measured: 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
Measured: Baseline to Month 24 for all patients plus additional follow up until Month 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2014 (estimated)
- Study startApr 2014
- Primary completionAug 2022
- Study completionOct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Molybdenum Cofactor Deficiency, Type AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Origin Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02047461
- Sponsor's protocol ID
- ALXN1101-MCD-201
- Other IDs
- 2013-002701-56
- Status last verified
- Sep 2023
- First submitted
- 9 Jan 2014
- Last updated
- 17 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
- ClinicalTrials.gov ID
- NCT02047461
- Acronym / sponsor ID
- ALXN1101-MCD-201
- Conditions
- Molybdenum Cofactor Deficiency, Type A
- Keywords
- Molybdenum cofactor deficiency (MoCD), molybdenum cofactor (MoCo) biosynthesis, sulfite oxidase (SO), xanthine dehydrogenase, aldehyde oxidase, S sulfocysteine (SSC)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Origin BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Apr 2014 (actual)
- Primary completion
- Aug 2022 (actual)
- Study completion
- Oct 2022 (actual)
- First posted
- 28 Jan 2014 (estimated)
- Results
- Posted 17 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ORGN001 (formerly ALXN1101) | Experimental | Drug: ORGN001 (formerly ALXN1101)daily IV infusions |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
- Currently treated with rcPMP infusions
Key exclusion criteria (1 listed)
- Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ORGN001 (formerly ALXN1101)IV infusion | Drug | ORGN001 (formerly ALXN1101) |
Primary outcome
Safety of ORGN001 (Formerly ALXN1101)
Time frame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Treatment Emergent Serious Adverse Events
4 secondary outcomes
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
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
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
Time frame: Baseline to Month 24 for all patients plus additional follow-up until Month 30
Long-term Safety of ORGN001 (Formerly ALXN1101)
Time frame: Baseline to Month 24 for all patients plus additional follow up until Month 72
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Molybdenum Cofactor Deficiency, Type A
Active, not recruitingObservational study
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Spinal Muscular Atrophy, Fragile X Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ORGN001 (formerly ALXN1101) in people with Molybdenum Cofactor Deficiency, Type A, run by Origin Biosciences at 6 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United Kingdom, United States, Australia, Netherlands, Tunisia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of ORGN001 (Formerly ALXN1101) (time frame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Oct 2023; AzTrials last refreshed its copy on 8 Nov 2026.