Natalizumab in Inclusion Body Myositis (IBM)

NCT02483845 | Unknown Phase 1

• 2 other identifiers: IBM-2013117571
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

Muscle biopsies of patients with Inclusion Body Myositis (IBM) have demonstrated a T-cell predominant inflammatory infiltrate, therefore, new agents targeting T -cell mediated cell death may be a novel treatment for IBM. Such an agent capable of preventing T-cell movement out of the vasculature, such as natalizumab, may be beneficial in IBM patients. Six patients will be recruited to participate in this phase I trial.

Conditions

Inclusion Body Myositis (IBM)

Outcome Measures

Primary Outcomes (2)

• Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies)

  muscle biopsies to determine inflammation

  12 months

• Determine if natalizumab is effective and safe in the treatment of patients with IBM (manual muscle testing)

  MMT - manual muscle testing to determine strength

  12 months

Secondary Outcomes (3)

• Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (blood levels)

  12 months

• Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (pain scores)

  12 months

• Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (functional rating score scale)

  12 months

Study Arms (1)

Natalizumab

EXPERIMENTAL

Natalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks

Drug: Natalizumab

Interventions

Natalizumab DRUG

Also known as: Tysabri

Natalizumab

Eligibility Criteria

• Age: 21 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Definite diagnosis of sporadic IBM through previous muscle biopsies
• Age 21-85
• FVC\> 50%
• Muscle function adequate for quantitative muscle testing
• JC virus negative at screening

You may not qualify if:

• Previous therapy with natalizumab.
• Treatment with other immunosuppressive agents within the last 12 months
• Quadriceps strength less than or equal to 2/5 at baseline
• Known malignancy
• Pregnancy or breastfeeding
• History of abnormal laboratory results indicative of any significant medical disease that would preclude the use of natalizumab
• Any clinically significant infectious illness in the 30 days before enrollment

Sponsors & Collaborators

• Phoenix Neurological Associates, LTD: lead

Study Sites (1)

Phoenix Neurological Insitutute

Phoenix, Arizona, 85018, United States

Location

MeSH Terms

Conditions

Myositis, Inclusion Body

Interventions

Natalizumab

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Todd Leveine, MD

  Phoenix Neurological

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 31, 2013
• First Posted: June 29, 2015
• Study Start: May 1, 2013
• Primary Completion: November 1, 2017
• Study Completion: November 1, 2017
• Last Updated: February 9, 2017

Record last verified: 2017-02

Locations

US (1)