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

Alpha Mannosidosis Clinical Trial

Official title:

A Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01268358
• Other study ID #: rhLAMAN-02
• Secondary ID: 2010-022084-36
• Status: Completed
• Phase: Phase 1
• Start date: October 2010
• Est. completion date: January 2011

Study information

• Verified date: July 2020
• Source: Chiesi Farmaceutici S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all patients will continue weekly treatment at the designated dose until the Safety Committee approves transfer to the rhLAMAN-03 protocol.

It is the hypothesis that Lamazym is safe to use.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: January 2011
• Est. primary completion date: January 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 20 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 = 20 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
Lamazym, ERT, infusion weekly

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Zymenex A/S	European Commission

References & Publications (3)

Borgwardt L, Dali CI, Fogh J, Månsson JE, Olsen KJ, Beck HC, Nielsen KG, Nielsen LH, Olsen SO, Riise Stensland HM, Nilssen O, Wibrand F, Thuesen AM, Pearl T, Haugsted U, Saftig P, Blanz J, Jones SA, Tylki-Szymanska A, Guffon-Fouiloux N, Beck M, Lund AM. E — View Citation

Borgwardt L, Stensland HM, Olsen KJ, Wibrand F, Klenow HB, Beck M, Amraoui Y, Arash L, Fogh J, Nilssen Ø, Dali CI, Lund AM. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation. Orphanet J Rare Dis. 2015 J — View Citation

Borgwardt L, Thuesen AM, Olsen KJ, Fogh J, Dali CI, Lund AM. Cognitive profile and activities of daily living: 35 patients with alpha-mannosidosis. J Inherit Metab Dis. 2015 Nov;38(6):1119-27. doi: 10.1007/s10545-015-9862-4. Epub 2015 May 28. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the safety profile of rhLAMAN (Lamazym)		1-5 weeks
Secondary	To determine the PK profile of rhLAMAN (Lamazym) in patients with alpha-mannosidosis as measured by rhLAMAN levels in plasma		1 dosis
Secondary	To collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03)		1 week

External Links

•   Study Record on EU Clinical Trials Register including results