NCT01285700

Brief 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.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2011

Geographic Reach
1 country

1 active site

Status
unknown

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

January 1, 2011

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

January 25, 2011

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 28, 2011

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2012

Completed
10 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2012

Completed
Last Updated

September 26, 2012

Status Verified

September 1, 2012

Enrollment Period

1 year

First QC Date

January 25, 2011

Last Update Submit

September 25, 2012

Conditions

Alpha Mannosidosis

Outcome Measures

Primary Outcomes (1)

  • Reduction of Oligosaccharides in urine

    Efficacy endpoint evaluation as change from baseline

    3 months (interim evaluation) + 6 months

Secondary Outcomes (9)

  • Reduction of Oligosaccharides in serum

    3 months (interim evaluation) + 6 months

  • Reduction of Oligosaccharides in CSF

    3 months (interim evaluation) + 6 months

  • The distance walked in 6 minutes

    3 months (interim evaluation) + 6 months

  • The number of steps climbed in 3 minutes

    3 months (interim evaluation) + 6 months

  • Pulmonary Function

    3 months (interim evaluation) + 6 months

  • +4 more secondary outcomes

Study Arms (2)

Lamazym 25

ACTIVE COMPARATOR

25 U/kg

Drug: Lamazym

Lamazym 50

ACTIVE COMPARATOR

50 U/kg

Drug: Lamazym

Interventions

LamazymDRUG

ERT, infusion weekly

Also known as: rhLAMAN
Lamazym 25Lamazym 50

Eligibility Criteria

Age5 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9

Copenhagen, 2100, Denmark

Location

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 Officials

  • Allan M. Lund, MD

    Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark

    PRINCIPAL INVESTIGATOR

  • Jens Fogh

    Zymenex A/S

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 25, 2011

First Posted

January 28, 2011

Study Start

January 1, 2011

Primary Completion

January 1, 2012

Study Completion

November 1, 2012

Last Updated

September 26, 2012

Record last verified: 2012-09

Locations

DK(1)