AzTrials
AzTrials

Sporadic Inclusion Body Myositis (sIBM)Phase 2/3BYM338NCT02250443

Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis

A Phase 2/3 study testing BYM338 (Bimagrumab) in people with Sporadic Inclusion Body Myositis (sIBM), run by Novartis Pharmaceuticals at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study is an open-label, long-term study for those patients who participated in the prior proof-of-concept protocol, in which the preliminary efficacy for BYM338 in patients with sIBM was demonstrated after a single 30 mg/kg i.v. dose of BYM338. Read more about this study

Who is it for?
Ages 40 to 75 years
Conditions the study is for
Sporadic Inclusion Body Myositis (sIBM)
What is being tested?
BYM338 (Bimagrumab)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
2 sitesUnited States
Study dates
11 Mar 2014to 23 Aug 2016
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 40 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Written informed consent must be obtained before any assessment is performed.
  • Patients who participated in the CBYM338X2205 study. A patient is defined as participating in the study if they were enrolled and received study medication.
  • Able to communicate well with the investigator, to understand and comply with the requirements of the study.

Who cannot join

  • Patients for whom the treating physician considers it inappropriate for continuation into the study, including consideration of physical and laboratory assessment of the patient at screening.
  • Patients who were non-compliant or demonstrated a serious protocol deviation in the previous study.
  • Use of other investigational drugs at the time of enrollment, within 30 days or 5 half-lives of enrollment, or until the expected PD effect has returned to baseline, whichever is longer; or longer if required by local regulations.
  • History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes
  • Swallowing difficulty or other reason that precludes adequate intake of energy and protein, defined as at least 20 kcal/kg/day and 0.6 g protein/kg/day as determined by the investigators assessment.
  • On the Columbia-Suicide Severity Rating Scale, a score of 4 or 5 on the Suicidal Ideation item or any "yes" on the Suicidal Behavior item that is related to suicidal behavior occurring during the last 2 years.
Show all 23Show fewer
  • Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
  • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, must use highly effective contraception during the study and for 5 half-lives (14 weeks) after stopping treatment. Highly effective contraception is defined as either:
  • Total abstinence: When this is in line with the preferred and usual lifestyle of the subject. [Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception].
  • Sterilization: have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
  • Male partner sterilization (with the appropriate post-vasectomy documentation of the absence of sperm in the ejaculate). [For female study subjects, the vasectomized male partner should be the sole partner for that patient].
  • Use of a combination of any two of the following (a+b or a+c or b+c):
  • <!-- -->
  • Use of oral, injected or implanted hormonal methods of contraception.
  • Placement of an intrauterine device (IUD) or intrauterine system (IUS).
  • Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository.
  • In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment. Postmenopausal females must have had no regular menstrual bleeding for at least one (1) year prior to initial dosing. Menopause will be confirmed by a plasma FSH level of > 40 IU/L (or as determined by the cut off used by the local clinical laboratory) at screening. Female patients who report surgical sterilization must have had the procedure at least six (6) months prior to initial dosing. Surgical sterilization procedures should be supported with clinical documentation made available to the sponsor and noted in the Relevant Medical History / Current Medical Conditions section of the eCRF. All female patients must have negative pregnancy test results at screening and baseline.
  • Use of oral beta agonists, oral corticosteroids, androgens or androgen inhibitors (including LHRH agonists), or intravenous gamma globulin (IVIG) within the previous 6 months. Short-term corticosteroids for unrelated indications, defined as < 20 mg/d for < 30 days and ending at least 60 days prior to screening, are acceptable.
  • Patients with known bleeding disorders, or who are under treatment with anti-coagulants.
  • A presence or history of clinically relevant ECG abnormalities, or including but not limited to Long QT syndrome, ischemic changes that cannot be shown to be stable for at least 6 months by previous ECG, or 2nd degree Mobitz II or 3rd degree heart block.
  • History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
  • Significant illness which has not resolved within two (2) weeks prior to initial dosing.
  • A positive HIV, Hepatitis B surface antigen or Hepatitis C test result. No additional exclusions may be applied by the investigator, in order to ensure that the study population will be representative of all eligible patients.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Novartis Investigative Site

    Boston, Massachusetts, 02115

  • Novartis Investigative Site

    Phoenix, Arizona, 85013

Other trials in United States

About this study

Official title: An Open-label, Long-term Study to Evaluate the Safety and Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis

This study is an open-label, long-term study for those patients who participated in the prior proof-of-concept protocol, in which the preliminary efficacy for BYM338 in patients with sIBM was demonstrated after a single 30 mg/kg i.v. dose of BYM338. This study is designed to confirm the efficacy, safety and tolerability of BYM338 in sIBM with long-term dosing. However due to lack of efficacy in patients with sIBM, the study was terminated early.

Read the detailed description

This is a non-confirmatory, multicenter, open-label, non-randomized trial which will extend active treatment to those patients that participated in the preceding proof-of-concept study (CBYM338X2205) in order to collect long-term safety and tolerability data. Up to 14 patients with sIBM will be invited to enroll into the study to receive BYM338 open-label for a period of approximately 3 years or until BYM338 is commercially available, whichever comes first. The study consists of a maximum 28-day screening period, a 5-day baseline period, and a treatment period consisting of the site visits at 4-week intervals for treatment administration, safety and pharmacokinetic follow-up. All patients will be administered a medium level i.v. BYM338 dose regardless of their treatment allocation in the prior study.

Being tested
BYM338 (Bimagrumab)
Keywords
Inclusion Body Myositis (IBM)
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.

  • BYM338 (Bimagrumab)Drug

Study groups in NCT02250443
Study groupRoleWhat they receive
BYM338ExperimentalBYM338 Group
Receives: Drug: BYM338 (Bimagrumab)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Adverse Events as a Measure of Safety and Tolerability

    Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity

    Measured: Up to 29 month

Secondary outcomes (9)

  1. Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)

    To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.

    Measured: Baseline, Day 1, 57, 113, 169, 365, 533, and day 729

  2. Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing

    To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins \& 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration

    Measured: Day 29, 85, 169, 253, 337, 421, 505, 589, 673, 757, 1177

  3. Changes From Baseline in Physical Function Reported by Patients

    Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration. Due to the no-signal this analysis was cancelled.

    Measured: Baseline, Week 104

  4. Changes From Baseline in Muscle Strength.

    Quadriceps muscle strength was measured, Quadriceps Quantitative Muscle Testing (QMT) by portable fixed dynamometry (PFD). A negative change from baseline indicates deterioration

    Measured: Baseline, Day 1, 113, 169, 365, 533, 729

  5. Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)

    The effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry).

    Measured: Baseline,Day 1, 113, 169, 365, 533, 729

  6. Changes From Baseline in Muscle Function 6 Minute Walking Distance

    The effect of BYM338 on additional muscle function measures (6 minute walking distance). The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.

    Measured: Baseline,Day 1, 113, 169, 365, 533, 729

  7. Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan

    Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was equal or more than 2% at Week 8 and 16 were considered responders

    Measured: Baseline, Day 1, 57, 113

  8. Pharmacokinetics (PK) Parameter of Cmax

    To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins \& 4 hours post-dose on Day 1.

    Measured: Day 1

  9. Time to Reach the Maximum Concentration After Drug Administration (Tmax)

    The time to reach the maximum concentration after drug administration

    Measured: Day 1

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Sep 2014 (estimated)
  2. Study start11 Mar 2014
  3. Primary completion23 Aug 2016
  4. Study completion23 Aug 2016

Resources

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 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02250443
Sponsor's protocol ID
CBYM338X2205E1
Acronym
BYM338
Status last verified
Mar 2019
First submitted
20 Mar 2014
Last updated
30 Dec 2020

Publications

  • Sivakumar K, Cochrane TI, Sloth B, Ashar H, Laurent D, Tanko LB, Amato AA. Long-term safety and tolerability of bimagrumab (BYM338) in sporadic inclusion body myositis. Neurology. 2020 Oct 6;95(14):e1971-e1978. doi: 10.1212/WNL.0000000000010417. Epub 2020 Jul 20.

    PubMed 32690797

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing BYM338 (Bimagrumab) in people with Sporadic Inclusion Body Myositis (sIBM), run by Novartis Pharmaceuticals at 2 sites 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 3 inclusion and 23 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: finished, as of the sponsor's last update on 30 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Apr 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants With Adverse Events as a Measure of Safety and Tolerability (time frame: Up to 29 month). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02250443, which also lists 1 linked publication.

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 30 Dec 2020.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in