Inclusion Body Myositis (IBM)Phase 1IBM-NATNCT02483845
Natalizumab in Inclusion Body Myositis (IBM)
A Phase 1 study testing Natalizumab in people with Inclusion Body Myositis (IBM), run by Phoenix Neurological Associates, LTD at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 to 85 years
- Conditions the study is for
- Inclusion Body Myositis (IBM)
- What is being tested?
- Natalizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- May 2013to Nov 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Phoenix Neurological Insitutute
Phoenix, Arizona, 85018
About this study
Official title: Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis
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.
Read the detailed description
This is a phase I open label, non-placebo controlled trial evaluating the safety and efficacy of natalizumab in patients with IBM. Pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells will be measured. The investigators will also assess quality of life, the inclusion body myositis functional rating score (IBM-FRS), and patient and physician global impression of change. Manual muscle testing and quantitative dynamometry will also be evaluated to see if patient's strength improves.
Patients who are eligible to participate and have signed a consent form will have a muscle biopsy performed at baseline and at the end of the study. Physical and neurological exams, as well as IBM-FRS, safety labs will be conducted and collected at monthly visits. Patients will start natalizumab therapy at 300mg intravenously every 4 weeks for 24 weeks. All study related procedures will be conducted at Phoenix Neurological Associates, as well as all infusions and muscle biopsies
- Conditions
- Inclusion Body Myositis (IBM)
- Being tested
- Natalizumab
- Medical subject headings
- Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NatalizumabDrug
Also called Tysabri
| Study group | Role | What they receive |
|---|---|---|
| Natalizumab | Experimental | Natalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks Receives: Drug: Natalizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies)
muscle biopsies to determine inflammation
Measured: 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
Measured: 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)
Safety labs - to determine blood levels
Measured: 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)
Pain scores on a visual analog scale
Measured: 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)
IM-FRS a functional rating score scale
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2015 (estimated)
- Study startMay 2013
- Primary completionNov 2017 (estimated)
- Study completionNov 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inclusion Body Myositis (IBM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Phoenix Neurological Associates, LTD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Todd Leveine, MD, Principal investigator, Phoenix Neurological
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02483845
- Sponsor's protocol ID
- IBM-2013
- Other IDs
- 117571
- Acronym
- IBM-NAT
- Status last verified
- Feb 2017
- First submitted
- 31 May 2013
- Last updated
- 9 Feb 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis
- ClinicalTrials.gov ID
- NCT02483845
- Acronym / sponsor ID
- IBM-NAT / IBM-2013
- Conditions
- Inclusion Body Myositis (IBM)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Phoenix Neurological Associates, LTDUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- Nov 2017 (estimated)
- Study completion
- Nov 2017 (estimated)
- First posted
- 29 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a phase I open label, non-placebo controlled trial evaluating the safety and efficacy of natalizumab in patients with IBM. Pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells will be measured. The investigators will also assess quality of life, the inclusion body myositis functional rating score (IBM-FRS), and patient and physician global impression of change. Manual muscle testing and quantitative dynamometry will also be evaluated to see if patient's strength improves.
Patients who are eligible to participate and have signed a consent form will have a muscle biopsy performed at baseline and at the end of the study. Physical and neurological exams, as well as IBM-FRS, safety labs will be conducted and collected at monthly visits. Patients will start natalizumab therapy at 300mg intravenously every 4 weeks for 24 weeks. All study related procedures will be conducted at Phoenix Neurological Associates, as well as all infusions and muscle biopsies
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Natalizumab | Experimental | Drug: NatalizumabNatalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks |
Who may qualify
- Ages
- 21 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NatalizumabAlso: Tysabri | Drug | Natalizumab |
Primary outcome
Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies)
Time frame: 12 months
muscle biopsies to determine inflammation
Determine if natalizumab is effective and safe in the treatment of patients with IBM (manual muscle testing)
Time frame: 12 months
MMT - manual muscle testing to determine strength
3 secondary outcomes
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)
Time frame: 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)
Time frame: 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)
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2017.
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The Aim of the Study is to Characterize Patients Exhibiting Muscular Atrophy and Weakness Who Require a Muscle Biopsy for Diagnosis (IBM vs Atypical MND), Through an Innovative Technique Which Enables the Evaluation of the Neuromuscular Junction Involvement in Each Condition
Inclusion Body Myositis (IBM), Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Todd Leveine, MD principal investigator
Phoenix Neurological
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Natalizumab in people with Inclusion Body Myositis (IBM), run by Phoenix Neurological Associates, LTD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies) (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2017.