Molybdenum Cofactor DeficiencyNCT01640717
A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
An observational study of Molybdenum Cofactor Deficiency, run by Origin Biosciences at 13 sites in 6 countries.
Trial summary
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD). Read more about this study
- Who is it for?
- Ages 1 day and older
- Conditions the study is for
- Molybdenum Cofactor Deficiency
- How many people
- 15 participants
- Where
- 13 sitesin 6 countries
- Study dates
- Nov 2012to Oct 2014
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 day and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients who received only intravenous cPMP under named-patient use will be eligible
Who may be able to join
- Male or female of any age.
- Patient with MoCD type A, suspected type A, or type B.
- Patient previously received cPMP only by intravenous route of administration.
- Parent(s) or legal guardian(s), depending on local regulations, has voluntarily provided written informed consent for the Investigator, Investigator's designee, or Sponsor designee to review, collect, transmit, and analyze data extracted from the medical record. In the case of a deceased patient for whom the parents or legal guardians could not be located, the appropriate ethical review committee may assign another person as legal representative to provide consent, where applicable per local and country regulations.
Who cannot join
- Patient's parent(s) or legal guardian(s) are unable to understand the nature and scope of the study.
Trial locations and contacts
13 sites in 6 countries.
13 sites in 6 countries
Australia3 sites
Neonatologist, Department of Paediatrics, Mercy Hospital for Women
Heidelberg
Monash Medical Centre
Melbourne
Western Sydney Genetics Program & Sydney Medical School
Westmead
Germany3 sites
University Hospital of Cologne
Cologne
Frankfurt Children's Hospital
Frankfurt
Akademisches Lehrkrankenhaus der Johannes Gutenberg
Koblenz
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham
Royal Hospital for Sick Children
Glasgow
Manchester Academic Health Science Centre
Manchester
United States2 sites
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232
Turkey (Türkiye)1 site
TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi
Gaziantep
About this study
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).
- Conditions
- Molybdenum Cofactor Deficiency
- Medical subject headings
- Molybdenum cofactor deficiency
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Efficacy
This is a noninterventional, observational, retrospective study to collect data on pediatric patients with MoCD who have received E. coli derived cPMP by intravenous only administration. The study will neither provide treatment with cPMP nor alter any ongoing treatment schedules; rather, its objective is to retrospectively collect data on MoCD history and previous treatment with intravenous E. coli derived cPMP, which is documented in the medical records of patients who have received treatment according to a named patient treatment plan.
Measured: For up to 60 months from the initial date of treatment with cPMP
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2012 (estimated)
- Study startNov 2012
- Primary completionOct 2014
- Study completionOct 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Molybdenum Cofactor DeficiencyStudies 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
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 15 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
- NCT01640717
- Sponsor's protocol ID
- ALX-MCD-501
- Other IDs
- ALX-MCD-501
- Status last verified
- Mar 2019
- First submitted
- 12 Jul 2012
- Last updated
- 19 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
- ClinicalTrials.gov ID
- NCT01640717
- Acronym / sponsor ID
- ALX-MCD-501
- Conditions
- Molybdenum Cofactor Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Origin BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Nov 2012
- Primary completion
- Oct 2014 (actual)
- Study completion
- Oct 2014 (actual)
- First posted
- 16 Jul 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).
Study design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Ages
- 1 day and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients who received only intravenous cPMP under named-patient use will be eligible
Key inclusion criteria (4 listed)
- Male or female of any age.
- Patient with MoCD type A, suspected type A, or type B.
- Patient previously received cPMP only by intravenous route of administration.
- Parent(s) or legal guardian(s), depending on local regulations, has voluntarily provided written informed consent for the Investigator, Investigator's designee, or Sponsor designee to review, collect, transmit, and analyze data extracted from the medical record. In the case of a deceased patient for whom the parents or legal guardians could not be located, the appropriate ethical review committee may assign another person as legal representative to provide consent, where applicable per local and country regulations.
Key exclusion criteria (1 listed)
- Patient's parent(s) or legal guardian(s) are unable to understand the nature and scope of the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Safety and Efficacy
Time frame: For up to 60 months from the initial date of treatment with cPMP
This is a noninterventional, observational, retrospective study to collect data on pediatric patients with MoCD who have received E. coli derived cPMP by intravenous only administration. The study will neither provide treatment with cPMP nor alter any ongoing treatment schedules; rather, its objective is to retrospectively collect data on MoCD history and previous treatment with intravenous E. coli derived cPMP, which is documented in the medical records of patients who have received treatment according to a named patient treatment plan.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2019.
Related trials for Molybdenum Cofactor Deficiency
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
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.
13 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Molybdenum Cofactor Deficiency, run by Origin Biosciences at 13 sites in 6 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 4 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 19 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Australia, Germany, United Kingdom, United States, Netherlands and 1 more country. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 and Efficacy (time frame: For up to 60 months from the initial date of treatment with cPMP). 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 19 Mar 2019.