Alpha-MannosidosisPhase 3rhLAMAN-10NCT02478840

Evaluation of Long-term Efficacy of Treatment With Lamazym

A Phase 3 study testing Lamazym in people with Alpha-Mannosidosis, run by Zymenex A/S at 1 site in Denmark.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently receiving the treatment according to the AfterCare Program. Read more about this study

Who is it for?: Any age
Conditions the study is for: Alpha-Mannosidosis
What is being tested?: Lamazym
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 18 participants
Where: 1 siteDenmark
Study dates: Feb 2015to Jun 2015
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 the phase 1 trial (EudraCT number: 2010-022084-36), phase 2a trial (EudraCT number: 2010-022085-26), phase 2b trial (EudraCT number: 2011-004355-40) or phase 3 trial (EudraCT number: 2012-000979-17)
The subject must still be receiving weekly intravenous infusions of Lamazym according to the AfterCare Program
The 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

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. Subjects unable to perform the motor tests independently from support are permitted to participate in the trial and will be evaluated for the remnant non motor endpoints
Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial
Pregnant and/or lactating women cannot participate in the trial. Concerning women of child bearing potential (WOCBP), 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.
Participation in other interventional trials testing IMP, including rhLAMAN-07 (EudraCT number: 2013-000336-97) and rhLAMAN-09 (EudraCT number: 2013-000321-31) trials with Lamazym
Pause of the IMP for 2 consecutive weeks during the last month. Subjects are allowed to be re-screened

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

Other trials in Denmark

About this study

Official title: A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym Trials

Read the detailed description

The primary objective of the trial is to evaluate the impact of the long-term treatment with Lamazym upon the level of biomarker oligosaccharides in serum and upon the endurance as measured by the change from baseline in the number of steps climbed in 3 minutes (3MSCT).

As secondary objectives, the long term efficacy of Lamazym will be investigated upon endurance as measured by the change from baseline in the number of meters walked in six minutes (6MWT), upon pulmonary function, motor proficiency by BOT-2 and hearing capability by audiometry. In addition, cognitive development will be assessed by Leiter-R test. CNS involvement will be evaluated with MRI/MRS (for patients who previously participated in rhLAMAN-02 trial), CSF biomarkers (Tau, NFL, GFAp) and CSF biomarkers oligosaccharides. Clearance of oligosaccharides in urine will be measured.

Long-term safety and Pharmaco-Kinetic (PK) profile after long-term treatment as measured by rhLAMAN levels in plasma will be assessed as well.

Quality of life will be assessed by questionnaires (CHAQ and EQ-5D-5L).

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

Study groups in NCT02478840
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 reduction of oligosaccharides in serum
Primary Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Change from baseline in 3 Minutes Stair Climb Test (3MSCT)
Primary Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment

Secondary outcomes (16)

6 Minute Walk Test (6MWT)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pulmonary function: Forced Vital Capacity (FVC)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pulmonary function: Forced Expiratory Volume during first second (FEV1)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pulmonary function: Peak Expiratory Flow Rate (PEF)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Functional capacity according to Bruininks-Oseretsky test of Motor Proficiency (BOT-2)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pure Tone Audiometry (PTA)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Equivalence age measured by Leiter International Performance Scale-Revised (Leiter-R)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Assessment of mannose-rich oligosaccharides in brain tissue as measured by Magnetic Resonance Spectroscopy (MRS) visual score (for patients who previously participated in rhLAMAN-02)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Assessment of mannose-rich oligosaccharides in brain tissue as measured by Magnetic Resonance Imaging (MRI) diffusion coefficient (for patients who previously participated in rhLAMAN-02)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid biomarkers: Oligosaccharides in Cerebrospinal Fluid (CSF)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid neuro-degeneration biomarkers: Tau Protein (Tau) in Cerebrospinal Fluid (CSF)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid neuro-degeneration biomarkers: Neurofilament Protein Light (NFL) in Cerebrospinal Fluid (CSF)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid neuro-degeneration biomarkers: Glial Fibrillary Acidic protein (GFAp) in Cerebrospinal Fluid (CSF)
Endpoint evaluation as change
Measured: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Drug exposure by Pharmaco Kinetic (PK) sampling profile on plasma
Evaluation of steady state Pharmaco Kinetics
Measured: 1 week
Measurement of in vivo biological activity of Lamazym in blood before and after Infusion of Lamazym
Comparing with Anti Body (AB) and PK measurements. Measuring unit is mU/mL
Measured: 1 week
Oligosaccharides in urine
Evaluation of steady state
Measured: 1 week

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Feb 2015Jun 2015

First posted23 Jun 2015 (estimated)
Study startFeb 2015
Primary completionJun 2015
Study completionJun 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (4)Papers the sponsor has linked to this study.
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 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02478840
Sponsor's protocol ID: rhLAMAN-10
Acronym: rhLAMAN-10
Status last verified: Nov 2020
First submitted: 9 Feb 2015
Last updated: 20 Nov 2020
Results posted: No
Data monitoring committee: No

Publications

Phillips D, Hennermann JB, Tylki-Szymanska A, Borgwardt L, Gil-Campos M, Guffon N, Amraoui Y, Geraci S, Ardigo D, Cattaneo F, Lund AM. Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis. Mol Genet Metab Rep. 2020 Apr 8;23:100586. doi: 10.1016/j.ymgmr.2020.100586. eCollection 2020 Jun.
PubMed 32292699
Borgwardt L, Guffon N, Amraoui Y, Jones SA, De Meirleir L, Lund AM, Gil-Campos M, Van den Hout JMP, Tylki-Szymanska A, Geraci S, Ardigò D, Cattaneo F, Harmatz P, Phillips D. Health Related Quality of Life, Disability, and Pain in Alpha Mannosidosis: Long-Term Data of Enzyme Replacement Therapy With Velmanase Alfa (Human Recombinant Alpha Mannosidase). Journal of Inborn Errors of Metabolism & Screening 2018, Volume 6: 1-12
Lund AM, Borgwardt L, Cattaneo F, Ardigo D, Geraci S, Gil-Campos M, De Meirleir L, Laroche C, Dolhem P, Cole D, Tylki-Szymanska A, Lopez-Rodriguez M, Guillen-Navarro E, Dali CI, Heron B, Fogh J, Muschol N, Phillips D, Van den Hout JMH, Jones SA, Amraoui Y, Harmatz P, Guffon N. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. J Inherit Metab Dis. 2018 Nov;41(6):1225-1233. doi: 10.1007/s10545-018-0175-2. Epub 2018 May 3.
PubMed 29725868
Harmatz P, Cattaneo F, Ardigo D, Geraci S, Hennermann JB, Guffon N, Lund A, Hendriksz CJ, Borgwardt L. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis. Mol Genet Metab. 2018 Jun;124(2):152-160. doi: 10.1016/j.ymgme.2018.04.003. Epub 2018 Apr 18.
PubMed 29716835

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Alpha-Mannosidosis trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym Trials
ClinicalTrials.gov ID: NCT02478840
Acronym / sponsor ID: rhLAMAN-10 / rhLAMAN-10
Conditions: Alpha-Mannosidosis
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: Zymenex A/SIndustry (pharma, biotech, devices)
Study start: Feb 2015
Primary completion: Jun 2015 (actual)
Study completion: Jun 2015 (actual)
First posted: 23 Jun 2015 (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.

Detailed description

Long-term safety and Pharmaco-Kinetic (PK) profile after long-term treatment as measured by rhLAMAN levels in plasma will be assessed as well.

Quality of life will be assessed by questionnaires (CHAQ and EQ-5D-5L).

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 (4 listed)

The subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36), phase 2a trial (EudraCT number: 2010-022085-26), phase 2b trial (EudraCT number: 2011-004355-40) or phase 3 trial (EudraCT number: 2012-000979-17)
The subject must still be receiving weekly intravenous infusions of Lamazym according to the AfterCare Program
The 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 (6 listed)

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. Subjects unable to perform the motor tests independently from support are permitted to participate in the trial and will be evaluated for the remnant non motor endpoints
Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial
Pregnant and/or lactating women cannot participate in the trial. Concerning women of child bearing potential (WOCBP), 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.
Participation in other interventional trials testing IMP, including rhLAMAN-07 (EudraCT number: 2013-000336-97) and rhLAMAN-09 (EudraCT number: 2013-000321-31) trials with Lamazym

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: rhLAMANrecombinant human alpha-mannosidase	Drug	Lamazym

Primary outcome

Change from baseline in reduction of oligosaccharides in serum
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Primary Endpoint evaluation as change
Change from baseline in 3 Minutes Stair Climb Test (3MSCT)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Primary Endpoint evaluation as change

16 secondary outcomes

6 Minute Walk Test (6MWT)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pulmonary function: Forced Vital Capacity (FVC)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pulmonary function: Forced Expiratory Volume during first second (FEV1)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pulmonary function: Peak Expiratory Flow Rate (PEF)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Functional capacity according to Bruininks-Oseretsky test of Motor Proficiency (BOT-2)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Pure Tone Audiometry (PTA)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Equivalence age measured by Leiter International Performance Scale-Revised (Leiter-R)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Assessment of mannose-rich oligosaccharides in brain tissue as measured by Magnetic Resonance Spectroscopy (MRS) visual score (for patients who previously participated in rhLAMAN-02)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Assessment of mannose-rich oligosaccharides in brain tissue as measured by Magnetic Resonance Imaging (MRI) diffusion coefficient (for patients who previously participated in rhLAMAN-02)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid biomarkers: Oligosaccharides in Cerebrospinal Fluid (CSF)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid neuro-degeneration biomarkers: Tau Protein (Tau) in Cerebrospinal Fluid (CSF)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid neuro-degeneration biomarkers: Neurofilament Protein Light (NFL) in Cerebrospinal Fluid (CSF)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Cerebrospinal fluid neuro-degeneration biomarkers: Glial Fibrillary Acidic protein (GFAp) in Cerebrospinal Fluid (CSF)
Time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment
Drug exposure by Pharmaco Kinetic (PK) sampling profile on plasma
Time frame: 1 week
Measurement of in vivo biological activity of Lamazym in blood before and after Infusion of Lamazym
Time frame: 1 week
Oligosaccharides in urine
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 20 Nov 2020.
Background: PubMed 32292699Phillips D, Hennermann JB, Tylki-Szymanska A, Borgwardt L, Gil-Campos M, Guffon N, Amraoui Y, Geraci S, Ardigo D, Cattaneo F, Lund AM. Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis. Mol Genet Metab Rep. 2020 Apr 8;23:100586. doi: 10.1016/j.ymgmr.2020.100586. eCollection 2020 Jun.
BackgroundBorgwardt L, Guffon N, Amraoui Y, Jones SA, De Meirleir L, Lund AM, Gil-Campos M, Van den Hout JMP, Tylki-Szymanska A, Geraci S, Ardigò D, Cattaneo F, Harmatz P, Phillips D. Health Related Quality of Life, Disability, and Pain in Alpha Mannosidosis: Long-Term Data of Enzyme Replacement Therapy With Velmanase Alfa (Human Recombinant Alpha Mannosidase). Journal of Inborn Errors of Metabolism & Screening 2018, Volume 6: 1-12
Results publication: PubMed 29725868Lund AM, Borgwardt L, Cattaneo F, Ardigo D, Geraci S, Gil-Campos M, De Meirleir L, Laroche C, Dolhem P, Cole D, Tylki-Szymanska A, Lopez-Rodriguez M, Guillen-Navarro E, Dali CI, Heron B, Fogh J, Muschol N, Phillips D, Van den Hout JMH, Jones SA, Amraoui Y, Harmatz P, Guffon N. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. J Inherit Metab Dis. 2018 Nov;41(6):1225-1233. doi: 10.1007/s10545-018-0175-2. Epub 2018 May 3.
Results publication: PubMed 29716835Harmatz P, Cattaneo F, Ardigo D, Geraci S, Hennermann JB, Guffon N, Lund A, Hendriksz CJ, Borgwardt L. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis. Mol Genet Metab. 2018 Jun;124(2):152-160. doi: 10.1016/j.ymgme.2018.04.003. Epub 2018 Apr 18.
Study Record on EU Clinical Trials Register including resultsLink provided by the sponsor.

Related trials for Alpha-Mannosidosis

Active, not recruitingPhase 2
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RecruitingObservational study
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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 of Clinical Genetics

Jens M Fogh, DVM 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 3 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 4 inclusion and 6 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 20 Nov 2020. 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 reduction of oligosaccharides in serum (time frame: Baseline evaluation prior to first dose compared to evaluation after one, two or four years of treatment). 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?

On the ClinicalTrials.gov record for NCT02478840, which also lists 4 linked publications.

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 20 Nov 2020.