Alpha-MannosidosisPhase 2NCT01681940
Long-term Efficacy and Safety of Lamazym 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 4 sites in 4 countries.
Trial summary
The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age 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
- 4 sitesin 4 countries
- Study dates
- Jan 2012to Sep 2013
- 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 subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
- Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
- The subject and his/her guardian(s) must have the ability to comply with the protocol
Who cannot join
- The subject 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
- 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: Before the start of the treatment the investigators will decide whether or not there is a need for contraception. This assessment will be done through interviews with the patient and parents. The evaluation will be done continuously during the study
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- Psychosis within the last 3 months
- Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial
- Participation in other interventional trials testing IMP except for studies with Lamazym
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
Belgium1 site
Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
Brussels, 1090
Denmark1 site
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, DK-2100
Spain1 site
Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
Córdoba, 14004
United Kingdom1 site
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Manchester, M13 9WL
About this study
Official title: A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age 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, recombinant human alpha-mannosidase
ERT, i.v. infusions weekly
| Study group | Role | What they receive |
|---|---|---|
| Lamazym | Experimental | 1 mg/kg body weight Receives: Drug: Lamazym |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of Oligosaccharides in blood serum
Efficacy endpoint evaluation as change from baseline
Measured: 6 months
Secondary outcomes (8)
The number of steps climbed in 3 minutes (3-minute stair climb)
Efficacy endpoint evaluation as change from baseline
Measured: 6 months
Reduction of Oligosaccharides in CSF
Efficacy endpoint evaluation as change from baseline
Measured: 6 months
The distance walked in 6 minutes (6-minute walk test)
Efficacy endpoint evaluation as change from baseline
Measured: 6 months
Pulmonary function
Efficacy endpoint evaluation as change from baseline
Measured: 6 months
Adverse events
Safety endpoint assesed weekly throughout the trial
Measured: 1 week
Development of clinically significant changes in vital signs and change in physical examination
Safety endpoint assesed weekly throughout the trial
Measured: 1 week
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
Safety endpoint assesed weekly throughout the trial
Measured: 1 week
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
Safety endpoint assesed weekly throughout the trial
Measured: 1 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2012 (estimated)
- Study startJan 2012
- Primary completionAug 2012
- Study completionSep 2013
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
- Not applicable
- Intervention model
- Single group
- 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, Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical GeneticsJens Fogh, Study chair, Zymenex A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01681940
- Sponsor's protocol ID
- rhLAMAN-04
- Other IDs
- 2011-004355-40
- Status last verified
- Mar 2017
- First submitted
- 22 Aug 2012
- Last updated
- 29 Mar 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
- ClinicalTrials.gov ID
- NCT01681940
- Acronym / sponsor ID
- rhLAMAN-04
- Conditions
- Alpha-Mannosidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Zymenex A/SIndustry (pharma, biotech, devices)
- Collaborators
- European Commission
- Study start
- Jan 2012
- Primary completion
- Aug 2012 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 10 Sep 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 overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lamazym | Experimental | Drug: Lamazym1 mg/kg body weight |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- The subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
- Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
- The subject and his/her guardian(s) must have the ability to comply with the protocol
Key exclusion criteria (9 listed)
- The subject 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
- Any other medical condition or serious intercurrent illness, or extenuating circumstance 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: rhLAMAN, recombinant human alpha-mannosidaseERT, i.v. infusions weekly | Drug | Lamazym |
Primary outcome
Reduction of Oligosaccharides in blood serum
Time frame: 6 months
Efficacy endpoint evaluation as change from baseline
8 secondary outcomes
The number of steps climbed in 3 minutes (3-minute stair climb)
Time frame: 6 months
Reduction of Oligosaccharides in CSF
Time frame: 6 months
The distance walked in 6 minutes (6-minute walk test)
Time frame: 6 months
Pulmonary function
Time frame: 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: 1 week
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
Time frame: 1 week
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 29 Mar 2017.
- Study Record on EU Clinical Trials Register including resultsLink provided by the sponsor.
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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
Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
Jens Fogh study chair
Zymenex A/S
4 sites in 4 countries: 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 4 sites in 4 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 3 inclusion and 9 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 29 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Belgium, Denmark, Spain, United Kingdom. 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?
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?
Reduction of Oligosaccharides in blood serum (time frame: 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 29 Mar 2017.