NCT01359319

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

A Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Single and Repeat Doses of Sialic Acid Extended Release (SA-ER) Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

Hereditary Inclusion Body Myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA), a critical component of many muscle proteins, resulting in a deficiency in SA in the muscles of HIBM patients. The effective replacement of the missing SA substrate is theoretically simple, and, in animal models, replacement with SA showed significant restoration of sialylation biochemistry and excellent reduction in muscle disease. These data show that replacement can achieve significant clinical benefit in muscle pathology, function, and survival.

Conditions

• Hereditary Inclusion Body Myopathy (HIBM)

Interventions

Type	Name	Description
DRUG	Sialic Acid Extended Release (SA-ER) Tablets	Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.
DRUG	Sialic Acid Extended Release (SA-ER) Tables	PPatients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.
DRUG	Sialic Acid Extended Release (SA-ER) Tablets	Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.
DRUG	Sialic Acid Extended Release (SA-ER) Tablets	Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.
DRUG	Sialic Acid Extended Release (SA-ER) Tablets	Patients will receive SA-ER tablets orally at one of five (5) dose levels in the single-dose phase,phase and one of four (4) dose levels in the repeat-dose phase. During repeat dosing, the total daily dose will be divided evenly into three doses given in the morning, in the evening, and at bedtime (qHS). No placebo or active comparator will be administered and the study drug will be administered on an open-label basis.

Timeline

• Start date: 2011-07-01
• Primary completion: 2012-04-01
• Completion: 2012-05-01
• First posted: 2011-05-24
• Last updated: 2012-05-21

Locations

2 sites across 1 country: United States