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

PHASE1CompletedINTERVENTIONAL
Enrollment

26

Participants

Timeline

Start Date

July 31, 2011

Primary Completion Date

April 30, 2012

Study Completion Date

May 31, 2012

Conditions
Hereditary Inclusion Body Myopathy (HIBM)
Interventions
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.

Trial Locations (2)

10019

Clinilabs, New York

92626

West Coast Clinical Trials, Costa Mesa

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT01359319 - Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) | Biotech Hunter | Biotech Hunter