Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

NCT01681940 | Completed Phase 2

• 2 other identifiers: rhLAMAN-042011-004355-40
• Study Type: interventional
• Target: 10
• Locations: 4 countries
• Sites: 4

Brief 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

Conditions

Alpha-Mannosidosis

Outcome Measures

Primary Outcomes (1)

• Reduction of Oligosaccharides in blood serum

  Efficacy endpoint evaluation as change from baseline

  6 months

Secondary Outcomes (8)

• The number of steps climbed in 3 minutes (3-minute stair climb)

  6 months

• Reduction of Oligosaccharides in CSF

  6 months

• The distance walked in 6 minutes (6-minute walk test)

  6 months

• Pulmonary function

  6 months

• Adverse events

  1 week

Study Arms (1)

Lamazym

EXPERIMENTAL

1 mg/kg body weight

Drug: Lamazym

Interventions

Lamazym DRUG

ERT, i.v. infusions weekly

Also known as: rhLAMAN, recombinant human alpha-mannosidase

Lamazym

Eligibility Criteria

• Age: 5 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Zymenex A/S: lead
• European Commission: collaborator

Study Sites (4)

Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101

Brussels, 1090, Belgium

Location

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

Copenhagen, DK-2100, Denmark

Location

Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn

Córdoba, 14004, Spain

Location

Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,

Manchester, M13 9WL, United Kingdom

Location

Related Links

• Study Record on EU Clinical Trials Register including results

MeSH Terms

Conditions

alpha-Mannosidosis

Condition Hierarchy (Ancestors)

Mannosidase Deficiency Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Allan M Lund, MD

  Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics

  PRINCIPAL INVESTIGATOR

• Jens Fogh

  Zymenex A/S

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2012
• First Posted: September 10, 2012
• Study Start: January 1, 2012
• Primary Completion: August 1, 2012
• Study Completion: September 1, 2013
• Last Updated: March 29, 2017

Record last verified: 2017-03

Locations

BE (1); ES (1); DK (1); GB (1)