Molybdenum Cofactor Deficiency (MoCD)Phase 1NCT01894165
Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
A Phase 1 study testing ALXN1101 in people with Molybdenum Cofactor Deficiency (MoCD) and Rare Autosomal Recessive Disorder, run by Origin Biosciences at 1 site in United States.
Trial summary
Phase 1 single dose study of ALXN1101 in healthy volunteers. Read more about this study
- Who is it for?
- Ages 18 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- What is being tested?
- ALXN1101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2013to Sep 2013
- Phase
- Phase 1
- 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 18 to 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Male or female subjects ≥ 18 and ≤ 60 years of age and weight ≥ 55 kg and ≤ 100 kg
- Willing and able to give written informed consent
- Female subjects of child bearing potential must have a negative serum pregnancy test or must be practicing an approved contraceptive regimen for the duration of the study
- Male subjects must be practicing an acceptable barrier method of contraception
Who cannot join
- Pregnant or nursing female subjects
- QTcF > 450 msec for males and > 470 msec for females, or a family history of Long QT Syndrome.
- CrCl < 80 mL/min
- CBC in acceptable range; SGOT or SGPT above the ULN
- HIV, Hepatitis B or Hepatitis C virus infection
- Other active systemic infection or malignancy
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- Investigational drug study within 60 days
- Major surgery within the prior 90 days
- History of illicit drug use or chronic alcohol dependence within 2 years prior to this study
- Positive urine drug toxicology screen or serum alcohol test
- Alcohol consumption within 48 hours prior to study drug administration
- Recently donated or lost ≥ 499 mL of blood
- Recent hormone replacement therapy or use of prescription medications
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Blinded, Placebo-Controlled, Single-Dose, Sequential-Cohort, Dose-Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ALXN1101 in Healthy Adult Subjects
Phase 1 single dose study of ALXN1101 in healthy volunteers.
Read the detailed description
This is a first-in-human (FIH), randomized, blinded, placebo-controlled, single-dose, sequential-cohort, dose-escalation study to evaluate the safety, tolerability, and pharmacokinetics (PK) of a single dose of ALXN1101 in healthy adult subjects.
- Conditions
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- Being tested
- ALXN1101, Placebo
- Keywords
- molybdenum cofactor deficiency (MoCD), deficiency of activity of molybdenum-dependent enzymes
- Medical subject headings
- Molybdenum cofactor deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1101Drug
Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
PlaceboDrug
Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
| Study group | Role | What they receive |
|---|---|---|
| ALXN1101 | Experimental | Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV. Receives: Drug: ALXN1101 |
| Placebo | Placebo comparator | Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of single dose of ALXN1101 in healthy adult subjects
Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events.
Measured: following the Day 30 visit for the last study subject
Secondary outcomes (1)
PK parameters of ALXN1101
PK parameters of ALXN1101 will be estimated including, but not limited to,maximum observed plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), terminal elimination halflife (t½), area under the plasma concentration-time curve (AUC), total body clearance (CL), and volume of distribution (Vd).
Measured: following the Day 5 visit for the last study subject
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2013 (estimated)
- Study startJun 2013
- Primary completionSep 2013
- Study completionSep 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Molybdenum Cofactor Deficiency (MoCD)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Origin Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ronald Goldwater, MD, Principal investigator, Parexel Baltimore Early Phase Clinical Unit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01894165
- Sponsor's protocol ID
- ALXN1101-MCD-101
- Status last verified
- Mar 2019
- First submitted
- 27 Jun 2013
- 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 Phase 1, Randomized, Blinded, Placebo-Controlled, Single-Dose, Sequential-Cohort, Dose-Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ALXN1101 in Healthy Adult Subjects
- ClinicalTrials.gov ID
- NCT01894165
- Acronym / sponsor ID
- ALXN1101-MCD-101
- Conditions
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- Keywords
- molybdenum cofactor deficiency (MoCD), deficiency of activity of molybdenum-dependent enzymes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Origin BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Jun 2013
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 10 Jul 2013 (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.
Phase 1 single dose study of ALXN1101 in healthy volunteers.
Detailed description
This is a first-in-human (FIH), randomized, blinded, placebo-controlled, single-dose, sequential-cohort, dose-escalation study to evaluate the safety, tolerability, and pharmacokinetics (PK) of a single dose of ALXN1101 in healthy adult subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALXN1101 | Experimental | Drug: ALXN1101Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV. |
| Placebo | Placebo comparator | Drug: PlaceboFour cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Male or female subjects ≥ 18 and ≤ 60 years of age and weight ≥ 55 kg and ≤ 100 kg
- Willing and able to give written informed consent
- Female subjects of child bearing potential must have a negative serum pregnancy test or must be practicing an approved contraceptive regimen for the duration of the study
- Male subjects must be practicing an acceptable barrier method of contraception
Key exclusion criteria (13 listed)
- Pregnant or nursing female subjects
- QTcF > 450 msec for males and > 470 msec for females, or a family history of Long QT Syndrome.
- CrCl < 80 mL/min
- CBC in acceptable range; SGOT or SGPT above the ULN
- HIV, Hepatitis B or Hepatitis C virus infection
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 |
|---|---|---|
| ALXN1101Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level. | Drug | ALXN1101 |
| PlaceboRandomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level. | Drug | Placebo |
Primary outcome
Safety and tolerability of single dose of ALXN1101 in healthy adult subjects
Time frame: following the Day 30 visit for the last study subject
Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events.
1 secondary outcome
PK parameters of ALXN1101
Time frame: following the Day 5 visit for the last study subject
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.
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.
Ronald Goldwater, MD principal investigator
Parexel Baltimore Early Phase Clinical Unit
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALXN1101 in people with Molybdenum Cofactor Deficiency (MoCD) and Rare Autosomal Recessive Disorder, run by Origin Biosciences at 1 site in United States.
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 13 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 1 site in United States. 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 tolerability of single dose of ALXN1101 in healthy adult subjects (time frame: following the Day 30 visit for the last study subject). 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.