Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients

NCT01908712 | Completed Phase 3

• 2 other identifiers: rhLAMAN-072013-000336-97
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamzede i.v. treatment of subjects with alpha-Mannosidosis.

Conditions

Alpha-Mannosidosis

Outcome Measures

Primary Outcomes (2)

• Change from baseline in Adverse events

  AE documented at every visit

  3 year

• Change from baseline in Lamazym antibodies

  AB measured every 12th week

  3 year

Secondary Outcomes (4)

• progress from baseline in number of steps climbed in 3 minutes

  1 year, 2 year and 3 year

• Progress from baseline in equivalent age

  1 year, 2 year and 3 year

• Progress from baseline in Forced Vital Capacity

  1 year, 2 year and 3 year

• Progress from baseline in distance walked in 6 minutes

  1 year, 2 year and 3 year

Study Arms (1)

Lamazym

EXPERIMENTAL

1 mg Lamazym/kg body weight

Drug: Lamazym

Interventions

Lamazym DRUG

ERT, i.v. infusions weekly

Also known as: rhLAMAN, recombinant human alpha-mannosidase

Lamazym

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• the subject must have participated in previous Lamazym-trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17
• Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
• The subject and his/her guardian(s) must have the ability to comply with the protocol

You may not qualify if:

• 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 circumstances that, in the opinion of the Investigator, would preclude participation in the trial
• Pregnancy. Pregnant woman is excluded. Before start fot he treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
• Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
• Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
• total IgE \> 800 IU/mL
• Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead

Study Sites (1)

Hôpital Femme Mére Enfant - CHU de Lyon

Bron, F-69677, France

Location

Related Links

• 12th international symposium on MPS and related diseases
• Results posted in EudraCT

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 Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2013
• First Posted: July 26, 2013
• Study Start: August 1, 2013
• Primary Completion: September 30, 2022
• Study Completion: September 30, 2022
• Last Updated: July 28, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)