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Protein Stabilizer

Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

Phase 3
Waitlist Available
Led By Michael Hanna
Research Sponsored by Orphazyme
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 change from baseline to month 20
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is designed to compare the efficacy and safety of starting arimoclomol earlier versus later in the treatment of sIBM.

Eligible Conditions
  • Inclusion Body Myositis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~change from baseline to month 20
This trial's timeline: 3 weeks for screening, Varies for treatment, and change from baseline to month 20 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
Secondary study objectives
Change in Hand Grip Strength
Change in Modified Timed Up and Go (mTUG)
Change in Quadriceps Muscle Strength
+3 more
Other study objectives
Magnetic Resonance Imaging (MRI) whole fat fraction of the thigh
Number of Treatment Emergent Adverse Events (TEAEs) in the open label extension

Side effects data

From 2012 Phase 2 & 3 trial • 24 Patients • NCT00769860
50%
Fall/contusion
38%
Musculoskeletal pain
31%
Upper respiratory tract infection
25%
Constipation
19%
Headache
19%
Throat irritation
19%
Post-biopsy pain
13%
Nausea
13%
Dry mouth
13%
Hyponatremia
13%
Loose stools
13%
Sinus infection
13%
Rash
13%
Hypertension
13%
Edema
13%
Lower respiratory tract infection
13%
Dizziness/tinnitus
6%
Palpitations
6%
Bowel movement problems
6%
Hematuria
6%
Eye pain
6%
Epigastralgia
6%
Gas pain
6%
Post-biopsy fatigue
6%
High thyroxine levels
6%
Paresthesia
6%
Cough
6%
Tooth extraction
6%
Dizziness
6%
Pyrosis
6%
Weight loss
6%
Erysipelas
6%
Cramps
6%
Rheumatoid arthritis flare
6%
Heat and soreness of proximal lower limbs
6%
Insect bite with erythema
6%
Cold sores
6%
Loss of consciousness
6%
Rosacea
6%
Sinus surgery
6%
Solar lentigines removal
6%
Tooth infection
6%
Conjunctivitis
6%
Geographic tongue
6%
Vomiting
6%
Worsening of restless leg syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arimoclomol
Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: ArimoclomolExperimental Treatment1 Intervention
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Arimoclomol
2008
Completed Phase 3
~460

Find a Location

Who is running the clinical trial?

OrphazymeLead Sponsor
7 Previous Clinical Trials
641 Total Patients Enrolled
ZevraDenmarkLead Sponsor
7 Previous Clinical Trials
641 Total Patients Enrolled
University of Kansas Medical CenterOTHER
517 Previous Clinical Trials
177,320 Total Patients Enrolled
KemPharm Denmark A/SLead Sponsor
7 Previous Clinical Trials
641 Total Patients Enrolled
University College, LondonOTHER
863 Previous Clinical Trials
38,752,907 Total Patients Enrolled
Michael HannaPrincipal InvestigatorUniversity College, London
Mazen M DimachkiePrincipal InvestigatorUniversity of Kansas Medical Center
~18 spots leftby Dec 2025
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