Alpha-Mannosidosis Clinical Trial
— rhLAMAN-07Official title:
A Multi-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials
Verified date | July 2023 |
Source | Chiesi Farmaceutici S.p.A. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 13 |
Est. completion date | September 30, 2022 |
Est. primary completion date | September 30, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: - 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 Exclusion Criteria: - 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 - Psychosis; any psychotic disease, also in remission, is an exclusion criteria - 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) |
Country | Name | City | State |
---|---|---|---|
France | Hôpital Femme Mére Enfant - CHU de Lyon | Bron |
Lead Sponsor | Collaborator |
---|---|
Chiesi Farmaceutici S.p.A. |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change from baseline in Adverse events | AE documented at every visit | 3 year | |
Primary | Change from baseline in Lamazym antibodies | AB measured every 12th week | 3 year | |
Secondary | progress from baseline in number of steps climbed in 3 minutes | 1 year, 2 year and 3 year | ||
Secondary | Progress from baseline in equivalent age | 1 year, 2 year and 3 year | ||
Secondary | Progress from baseline in Forced Vital Capacity | 1 year, 2 year and 3 year | ||
Secondary | Progress from baseline in distance walked in 6 minutes | 1 year, 2 year and 3 year |
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