Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis

Alpha Mannosidosis Clinical Trial

Official title:

A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.

Clinical Trial Details — Status: Unknown status

Administrative data

• NCT number: NCT01285700
• Other study ID #: rhLAMAN-03
• Secondary ID: 2010-022085-26
• Status: Unknown status
• Phase: Phase 2
• First received: January 25, 2011
• Last updated: September 25, 2012
• Start date: January 2011
• Est. completion date: November 2012

Study information

• Verified date: September 2012
• Source: Zymenex A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Unknown status
• Enrollment: 10
• Est. completion date: November 2012
• Est. primary completion date: January 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 21 Years

Eligibility

Inclusion Criteria:

1. The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes

2. The patient must have an age at the time of screening = 5 year and = 21 years

3. 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).

4. The patient must have the ability to mentally cooperate in the cognitive and motor function tests

5. The patient must have the ability to hear and follow a request. Hearing aids can be worn.

6. 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)

7. The patient and his/her guardian(s) must have the ability to comply with the protocol

Exclusion Criteria:

1. The patient cannot walk without support.

2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis

3. History of bone marrow transplantation

4. 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

5. Presence of an ECHO with abnormalities within half a year that, in the opinion of the Investigator, would preclude participation in the trial

6. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the investigator, would preclude participation in the trial

7. Pregnancy

8. Psychosis within the last 3 months

Related Conditions & MeSH terms

• Alpha Mannosidosis
• alpha-Mannosidosis

Intervention

Drug:
• Lamazym
ERT, infusion weekly

Locations

Country	Name	City	State
Denmark	Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9	Copenhagen

Sponsors (2)

Lead Sponsor	Collaborator
Zymenex A/S	European Commission

Country where clinical trial is conducted

Denmark, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reduction of Oligosaccharides in urine	Efficacy endpoint evaluation as change from baseline	3 months (interim evaluation) + 6 months
Secondary	Reduction of Oligosaccharides in serum	Efficacy endpoint evaluation as change from baseline	3 months (interim evaluation) + 6 months
Secondary	Reduction of Oligosaccharides in CSF	Efficacy endpoint evaluation as change from baseline	3 months (interim evaluation) + 6 months
Secondary	The distance walked in 6 minutes	Efficacy endpoint evaluation as change from baseline	3 months (interim evaluation) + 6 months
Secondary	The number of steps climbed in 3 minutes	Efficacy endpoint evaluation as change from baseline	3 months (interim evaluation) + 6 months
Secondary	Pulmonary Function	Efficacy endpoint evaluation as change from baseline	3 months (interim evaluation) + 6 months
Secondary	Adverse events	Safety endpoint assessed weekly throughout the trial	1 week
Secondary	Development of clinically significant changes in vital signs and change in physical examination	Safety endpoint assessed weekly throughout the trial	1 week
Secondary	Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)	Safety endpoint assessed every 4th week throughout the trial	4 weeks
Secondary	Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies	Safety endpoint assessed every other week throughout the trial	2 weeks