Alpha-MannosidosisPhase 3rhLAMAN-09NCT01908725
Lamazym Aftercare Study
A Phase 3 study testing Lamazym in people with Alpha-Mannosidosis, run by Chiesi Farmaceutici S.p.A. at 1 site in Denmark.
Trial summary
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial. Read more about this study
- Who is it for?
- Any age
- 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
- 8 participants
- Where
- 1 siteDenmark
- Study dates
- Jun 2013to 30 Sep 2022
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- the subject must have participated in previous Lamazym-trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17
- Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
- The subject and his/her guardian(s) must have the ability to comply with the protocol
Who cannot join
- 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 circumstances that, in the opinion of the Investigator, would preclude participation in the trial
- Pregnancy. Pregnant woman is excluded. Before start fot he treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
- Psychosis; any psychotic disease, also in remission, is an exclusion criteria
- Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, DK-2100
About this study
Official title: A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
Read the detailed description
This protocol only concern subjects where compassionate use program was not accepted. Efficacy will be evaluated once yearly to follow the subject's progress in clinical parameters.
- 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 Lamazym/kg body weight Receives: Drug: Lamazym |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Adverse events
AE documented at all visits
Measured: 3 year
Change from baseline in Lamazym antibodies
AB measured every 12th week
Measured: 3 year
Secondary outcomes (4)
progress from baseline in number of steps climbed in 3 minutes
Measured: 1 year, 2 year and 3 year
Progress from baseline in equivalent age
Measured: 1 year, 2 year and 3 year
Progress from baseline in Forced Vital Capacity
Measured: 1 year, 2 year and 3 year
Progress from baseline in distance walked in 6 minutes
Measured: 1 year, 2 year and 3 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2013 (estimated)
- Study startJun 2013
- Primary completion30 Sep 2022
- Study completion30 Sep 2022
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.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01908725
- Sponsor's protocol ID
- rhLAMAN-09
- Other IDs
- 2013-000321-31
- Acronym
- rhLAMAN-09
- Status last verified
- Jul 2023
- First submitted
- 12 Jun 2013
- Last updated
- 28 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials
- ClinicalTrials.gov ID
- NCT01908725
- Acronym / sponsor ID
- rhLAMAN-09 / rhLAMAN-09
- Conditions
- Alpha-Mannosidosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- Jun 2013
- Primary completion
- 30 Sep 2022 (actual)
- Study completion
- 30 Sep 2022 (actual)
- First posted
- 26 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.
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
Detailed description
This protocol only concern subjects where compassionate use program was not accepted. Efficacy will be evaluated once yearly to follow the subject's progress in clinical parameters.
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 Lamazym/kg body weight |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- the subject must have participated in previous Lamazym-trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17
- Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
- The subject and his/her guardian(s) must have the ability to comply with the protocol
Key exclusion criteria (5 listed)
- 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 circumstances that, in the opinion of the Investigator, would preclude participation in the trial
- Pregnancy. Pregnant woman is excluded. Before start fot he treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
- Psychosis; any psychotic disease, also in remission, is an exclusion criteria
- Planned major surgery 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
Change from baseline in Adverse events
Time frame: 3 year
AE documented at all visits
Change from baseline in Lamazym antibodies
Time frame: 3 year
AB measured every 12th week
4 secondary outcomes
progress from baseline in number of steps climbed in 3 minutes
Time frame: 1 year, 2 year and 3 year
Progress from baseline in equivalent age
Time frame: 1 year, 2 year and 3 year
Progress from baseline in Forced Vital Capacity
Time frame: 1 year, 2 year and 3 year
Progress from baseline in distance walked in 6 minutes
Time frame: 1 year, 2 year and 3 year
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 28 Jul 2023.
- 12th international symposium on MPS and related diseasesLink provided by the sponsor.
- Results posted in EudraCTLink 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lamazym in people with Alpha-Mannosidosis, run by Chiesi Farmaceutici S.p.A. 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 3 inclusion and 5 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 28 Jul 2023. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Change from baseline in Adverse events (time frame: 3 year). The study lists 2 primary outcomes in all. 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 28 Jul 2023.