Crossover Participants for Inclusion Body Myopathy

Phase 2

Waitlist Available

Research Sponsored by Ultragenyx Pharmaceutical Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up part i: baseline (study ux001-cl201 week 48), month 6; part ii-iv: baseline, months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination

Awards & highlights

No Placebo-Only Group

Summary

The safety objectives of the study are to: evaluate additional long-term safety of SA-ER treatment of participants with GNE myopathy previously treated with SA-ER at dose of 6g/day (Part I); evaluate the safety of 12g /day SA (delivered by 1.5g of SA-ER tablets and 1.5g of SA-IR capsules 4 times per day) in the treatment of participants with GNE myopathy (Part II) over a 6 month treatment period; evaluate the safety of SA treatment at both 6g/day and 12 g/day (Part III \[SA-ER/SA-IR\] and Part IV \[SA-ER\]).

Eligible Conditions

Inclusion Body Myopathy

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ part i: baseline (study ux001-cl201 week 48), month 6; part ii-iv: baseline, months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~part i: baseline (study ux001-cl201 week 48), month 6; part ii-iv: baseline, months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination

This trial's timeline: 3 weeks for screening, Varies for treatment, and part i: baseline (study ux001-cl201 week 48), month 6; part ii-iv: baseline, months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit?

Therapeutic procedure

Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit?

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Naïve ParticipantsExperimental Treatment2 Interventions

Treatment naïve participants with GNE myopathy were enrolled into Part II of the study: * Part II: 12 g/day SA (1.5 g of SA-ER and 1.5 g of SA-IR treatment QID) for 36 months * Part III: 6 g/day or 12 g/day SA (both SA-ER and SA-IR) * Part IV: 6 g/day or 12 g/day SA (SA-ER only)

Group II: Crossover ParticipantsExperimental Treatment2 Interventions

Participants completing the 48-week study (UX001-CL201; NCT01517880) were enrolled into Part I of the study: * Part I: participants continued on 6 g/day SA-ER for 12 weeks * Part II: 12 g/day SA (1.5 g of SA-ER and 1.5 g of SA-IR treatment 4 times per day \[QID\]) for 36 months * Part III: 6 g/day or 12 g/day SA (both SA-ER and SA-IR) * Part IV: 6 g/day or 12 g/day SA (SA-ER only)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SA-IR 500 mg

2013

Completed Phase 2

~60

SA-ER 500 mg

2013

Completed Phase 2

~60