NCT01908725

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jun 2013

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2013

Completed
11 days until next milestone

First Submitted

Initial submission to the registry

June 12, 2013

Completed
1 month until next milestone

First Posted

Study publicly available on registry

July 26, 2013

Completed
9.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2022

Completed
Last Updated

July 28, 2023

Status Verified

July 1, 2023

Enrollment Period

9.3 years

First QC Date

June 12, 2013

Last Update Submit

July 26, 2023

Conditions

Outcome Measures

Primary Outcomes (2)

  • Change from baseline in Adverse events

    AE documented at all visits

    3 year

  • Change from baseline in Lamazym antibodies

    AB measured every 12th week

    3 year

Secondary Outcomes (4)

  • progress from baseline in number of steps climbed in 3 minutes

    1 year, 2 year and 3 year

  • Progress from baseline in equivalent age

    1 year, 2 year and 3 year

  • Progress from baseline in Forced Vital Capacity

    1 year, 2 year and 3 year

  • Progress from baseline in distance walked in 6 minutes

    1 year, 2 year and 3 year

Study Arms (1)

Lamazym

EXPERIMENTAL

1 mg Lamazym/kg body weight

Drug: Lamazym

Interventions

ERT, i.v. infusions weekly

Also known as: rhLAMAN, recombinant human alpha-mannosidase
Lamazym

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9

Copenhagen, DK-2100, Denmark

Location

Related Links

MeSH Terms

Conditions

alpha-Mannosidosis

Condition Hierarchy (Ancestors)

Mannosidase Deficiency DiseasesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 12, 2013

First Posted

July 26, 2013

Study Start

June 1, 2013

Primary Completion

September 30, 2022

Study Completion

September 30, 2022

Last Updated

July 28, 2023

Record last verified: 2023-07

Locations