Alpha MannosidosisPhase 2NCT01285700
Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis
A Phase 2 study testing Lamazym in people with Alpha Mannosidosis, run by Zymenex A/S at 1 site in Denmark.
Trial summary
This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis. Read more about this study
- Who is it for?
- Ages 5 to 21 years
- Conditions the study is for
- Alpha Mannosidosis
- What is being tested?
- Lamazym
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteDenmark
- Study dates
- Jan 2011to Nov 2012
- Phase
- Phase 2
- 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 5 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes
- The patient must have an age at the time of screening ≥ 5 year and ≤ 21 years
- The patient must have physical ability to perform 6-minutes walk test (6MWT), 3 minute-stair climb test (3MSCT) and pulmonary lung function test (spirometry, body plethysmography).
- The patient must have the ability to mentally cooperate in the cognitive and motor function tests
- The patient must have the ability to hear and follow a request. Hearing aids can be worn.
- Patient or patient's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
- The patient and his/her guardian(s) must have the ability to comply with the protocol
Who cannot join
- The patient cannot walk without support.
- Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
- History of bone marrow transplantation
- Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
- Presence of an ECHO with abnormalities within half a year that, in the opinion of the Investigator, would preclude participation in the trial
- Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial
- Pregnancy
- Psychosis within the last 3 months
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, 2100
About this study
Official title: A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.
This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis.
- Conditions
- Alpha Mannosidosis
- Being tested
- Lamazym
- Medical subject headings
- alpha-Mannosidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LamazymDrug
Also called rhLAMAN
ERT, infusion weekly
| Study group | Role | What they receive |
|---|---|---|
| Lamazym 25 | Active comparator | 25 U/kg Receives: Drug: Lamazym |
| Lamazym 50 | Active comparator | 50 U/kg Receives: Drug: Lamazym |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of Oligosaccharides in urine
Efficacy endpoint evaluation as change from baseline
Measured: 3 months (interim evaluation) + 6 months
Secondary outcomes (9)
Reduction of Oligosaccharides in serum
Efficacy endpoint evaluation as change from baseline
Measured: 3 months (interim evaluation) + 6 months
Reduction of Oligosaccharides in CSF
Efficacy endpoint evaluation as change from baseline
Measured: 3 months (interim evaluation) + 6 months
The distance walked in 6 minutes
Efficacy endpoint evaluation as change from baseline
Measured: 3 months (interim evaluation) + 6 months
The number of steps climbed in 3 minutes
Efficacy endpoint evaluation as change from baseline
Measured: 3 months (interim evaluation) + 6 months
Pulmonary Function
Efficacy endpoint evaluation as change from baseline
Measured: 3 months (interim evaluation) + 6 months
Adverse events
Safety endpoint assessed weekly throughout the trial
Measured: 1 week
Development of clinically significant changes in vital signs and change in physical examination
Safety endpoint assessed weekly throughout the trial
Measured: 1 week
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
Safety endpoint assessed every 4th week throughout the trial
Measured: 4 weeks
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
Safety endpoint assessed every other week throughout the trial
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2011 (estimated)
- Study startJan 2011
- Primary completionJan 2012
- Study completionNov 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha MannosidosisStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zymenex A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- European Commission
- Responsible party
- Sponsor
- Investigators
- Allan M. Lund, MD, Principal investigator, Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, DenmarkJens Fogh, Study chair, Zymenex A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01285700
- Sponsor's protocol ID
- rhLAMAN-03
- Other IDs
- 2010-022085-26
- Status last verified
- Sep 2012
- First submitted
- 25 Jan 2011
- Last updated
- 26 Sep 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.
- ClinicalTrials.gov ID
- NCT01285700
- Acronym / sponsor ID
- rhLAMAN-03
- Conditions
- Alpha Mannosidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Zymenex A/SIndustry (pharma, biotech, devices)
- Collaborators
- European Commission
- Study start
- Jan 2011
- Primary completion
- Jan 2012 (actual)
- Study completion
- Nov 2012 (estimated)
- First posted
- 28 Jan 2011 (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.
This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lamazym 25 | Active comparator | Drug: Lamazym25 U/kg |
| Lamazym 50 | Active comparator | Drug: Lamazym50 U/kg |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes
- The patient must have an age at the time of screening ≥ 5 year and ≤ 21 years
- The patient must have physical ability to perform 6-minutes walk test (6MWT), 3 minute-stair climb test (3MSCT) and pulmonary lung function test (spirometry, body plethysmography).
- The patient must have the ability to mentally cooperate in the cognitive and motor function tests
- The patient must have the ability to hear and follow a request. Hearing aids can be worn.
Key exclusion criteria (8 listed)
- The patient cannot walk without support.
- Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
- History of bone marrow transplantation
- Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
- Presence of an ECHO with abnormalities within half a year that, in the opinion of the Investigator, would preclude participation in the trial
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 |
|---|---|---|
| LamazymAlso: rhLAMANERT, infusion weekly | Drug | Lamazym 25, Lamazym 50 |
Primary outcome
Reduction of Oligosaccharides in urine
Time frame: 3 months (interim evaluation) + 6 months
Efficacy endpoint evaluation as change from baseline
9 secondary outcomes
Reduction of Oligosaccharides in serum
Time frame: 3 months (interim evaluation) + 6 months
Reduction of Oligosaccharides in CSF
Time frame: 3 months (interim evaluation) + 6 months
The distance walked in 6 minutes
Time frame: 3 months (interim evaluation) + 6 months
The number of steps climbed in 3 minutes
Time frame: 3 months (interim evaluation) + 6 months
Pulmonary Function
Time frame: 3 months (interim evaluation) + 6 months
Adverse events
Time frame: 1 week
Development of clinically significant changes in vital signs and change in physical examination
Time frame: 1 week
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
Time frame: 4 weeks
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
Time frame: 2 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2012.
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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.
Allan M. Lund, MD principal investigator
Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark
Jens Fogh study chair
Zymenex A/S
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lamazym in people with Alpha Mannosidosis, run by Zymenex A/S at 1 site in Denmark.
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 7 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 26 Sep 2012. 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 Denmark. 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?
Results are not available in the source record.
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?
Reduction of Oligosaccharides in urine (time frame: 3 months (interim evaluation) + 6 months). 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 26 Sep 2012.