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

Zymenex

Status and phase: Completed; Phase 2

Conditions: Alpha-Mannosidosis

Treatments: Drug: Lamazym

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01681940

2011-004355-40 (EudraCT Number)

rhLAMAN-04

Details and patient eligibility

About

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

Enrollment: 10 patients

Sex: All

Ages: 5 to 21 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Lamazym

Experimental group

Description:

1 mg/kg body weight

Treatment:

Drug: Lamazym