rhLAMAN-04

• Research type

  Research Study

• Full title

  A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis

• IRAS ID

  92681

• Contact name

  Simon Jones

• Sponsor organisation

  Zymenex A/S

• Eudract number

  2011-004355-40

• ISRCTN Number

  0

• Clinicaltrials.gov Identifier

  0

• Research summary

  Alpha-Mannosidosis is a rare lysosomal storage disorder caused by deficiency of the lysosomal enzyme alpha-mannosidase. The effect of the enzyme deficiency is accumulation of mannose-rich glyco-proteins in the tissues. It is inherited in an autosomal recessive manner. The typical symptoms include facial characteristics, mental retardation, ataxia, hearing impairment, impaired speech, recurrent infections, skeletal abnormalities, muscular pain and weakness.There is a slow progression of symptoms over several decades. The long-term prognosis is generally poor. The clinical variation is considerable, encompassing a continuum from mild to severe. Most of the patients are diagnosed in their first or second decade of life. Increased levels of mannose-rich oligosaccharides in urine, reduced activity of alpha-mannosidase in leukocytes and finding of two pathogenic mutations in MAN2B1 identify the patients. Currently no effective clinical treatment for alpha-Mannosidosis exists. Medical treatments are confined to be supportive and symptomatic. Lamazym is recombinant human lysosomal alpha-Mannosidase developed as an intravenous enzyme replacement therapy (ERT) for the treatment of alpha-Mannosidosis. The current trial is being performed to evaluate the long-term efficacy, safety and tolerability of Lamazym treatment in patients with alpha-Mannosidosis. If successful, the enzyme replacement therapy is expected to be given life-long, with the aim of normalizing the mannose-rich oligosaccharide levels in the tissues, altering the progression of the disease and thereby preventing or reducing abnormalities from being developed and hopefully improving the patient??s condition and quality of life.

• REC name

  North West - Haydock Research Ethics Committee

• REC reference

  12/NW/0119

• Date of REC Opinion

  28 Mar 2012

• REC opinion

  Further Information Favourable Opinion