DermatomyositisEarly Phase 1NCT02271165
Subcutaneous Immunoglobulin (Hizentra) in Patients With Dermatomyositis: A Proof of Concept Study
A Early Phase 1 study testing Immunoglobulin (Hizentra) in people with Dermatomyositis, run by Thomas Jefferson University at 1 site in United States.
Trial summary
The purpose of the study is to evaluate the effectiveness and safety of human immunoglobulin SCIg in the form of Hizentra (Immune globulin Subcutaneous) in patients with Dermatomyositis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dermatomyositis
- What is being tested?
- Immunoglobulin (Hizentra)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2014to 2 Mar 2017
- Phase
- Early 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men or woman aged >18 years
- Diagnosis of DM based on standard criteria
- Receiving the equivalent of at least 0.4 g/kg IVIg every 4 weeks (IVIg group only)
- Established response to IVIg or dependence on IVIg to maintain status established either by symptomatic worsening of condition at the end of the inter-dose interval for both groups or by worsening after reduction of the dose within the previous 12 months (IVIg group only)
- IVIg regimen stable for 12 weeks while on IVIg (minor changes are permitted provided that the dose change is 15% or less) (IVIg group only)
- Stable dosing with steroids and/or other immunosuppressives for 12 weeks with no changes schedule or intended.
Who cannot join
- Pregnancy, planned pregnancy, breast feeding or unwillingness to practice contraception
- Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
- Initiation or immunomodulatory treatment other than IVIg in the past 24 weeks or modification of immunomodulatory treatment other than IVIg in the past 12 weeks.
- Participation in trial of an investigational medicinal product in the past 12 weeks
- Presence of skin infection unrelated to dermatomyositis, severe skin involvement
- Presence of any other medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
About this study
The purpose of the study is to evaluate the effectiveness and safety of human immunoglobulin SCIg in the form of Hizentra (Immune globulin Subcutaneous) in patients with Dermatomyositis. Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body) in patients with primary immunodeficiency. At present, patients with steroid resistant dermatomyositis can only be treated with IVIg (The healthy antibodies in IVIG can block the damaging antibodies that attack muscle and skin in dermatomyositis) treatment. An evaluation can then be made to see if SCIg is a suitable replacement and exerts immunomodulatory effect on complement antibodies.
- Conditions
- Dermatomyositis
- Being tested
- Immunoglobulin (Hizentra)
- Medical subject headings
- Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Immunoglobulin (Hizentra)Drug
Also called Immunoglobulin subcutaneous (human) 20% liquid, Hizentra
| Study group | Role | What they receive |
|---|---|---|
| IVIg naive | Active comparator | Patients naïve to IVIg who have active disease responding to corticosteroids or are corticosteroid-dependent, will be also included. Before these patients enter the study, they will receive 3 monthly infusions of IVIg starting with the standard dose of 2gram/kg/month and followed with monthly maintenance of 1 or 2gram/kg according to their response. Receives: Drug: Immunoglobulin (Hizentra) |
| non-IVIg naive | Active comparator | Participants already on IVIg will be observed for 12 weeks under their existing IVIg regimen and will undergo measurements of their muscle strength, skin changes, assessments of their daily activities and quality of life (QoL) every 4 weeks at scheduled visits for monthly maintenance IVIg infusions (weeks 0,4,8,12). Receives: Drug: Immunoglobulin (Hizentra) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
Measured: The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
Secondary outcomes (1)
The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Secondary outcome measures will be a) patient preference, based on the number of participants who prefer SCIg to IVIg (a home-made questionnaire will be utilized to capture preference in the most unbiased way; b) average change in Quality of life scores between week 12 and 28; c) effect on the complement consumption in serum; d) Immunological parameters on the repeated skin biopsies; and e) adverse events as reported at each visit. Accordingly, information will be obtained on the superiority of SCIg vs. IVIg
Measured: The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2014 (estimated)
- Study startNov 2014
- Primary completion2 Mar 2017
- Study completion2 Mar 2017
Why it stopped: Difficult recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DermatomyositisStudies of the same condition, open and under way.
- More trials run by Thomas Jefferson UniversityThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, marinos dalakas, Thomas Jefferson University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02271165
- Sponsor's protocol ID
- 13P.192
- Status last verified
- May 2018
- First submitted
- 15 Oct 2014
- Last updated
- 16 May 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Subcutaneous Immunoglobulin (Hizentra) in Patients With Dermatomyositis: A Proof of Concept Study
- ClinicalTrials.gov ID
- NCT02271165
- Acronym / sponsor ID
- 13P.192
- Conditions
- Dermatomyositis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficult recruitment
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Thomas Jefferson UniversityUniversity, hospital or other
- Study start
- Nov 2014
- Primary completion
- 2 Mar 2017 (actual)
- Study completion
- 2 Mar 2017 (actual)
- First posted
- 22 Oct 2014 (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.
The purpose of the study is to evaluate the effectiveness and safety of human immunoglobulin SCIg in the form of Hizentra (Immune globulin Subcutaneous) in patients with Dermatomyositis. Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body) in patients with primary immunodeficiency. At present, patients with steroid resistant dermatomyositis can only be treated with IVIg (The healthy antibodies in IVIG can block the damaging antibodies that attack muscle and skin in dermatomyositis) treatment. An evaluation can then be made to see if SCIg is a suitable replacement and exerts immunomodulatory effect on complement antibodies.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IVIg naive | Active comparator | Drug: Immunoglobulin (Hizentra)Patients naïve to IVIg who have active disease responding to corticosteroids or are corticosteroid-dependent, will be also included. Before these patients enter the study, they will receive 3 monthly infusions of IVIg starting with the standard dose of 2gram/kg/month and followed with monthly maintenance of 1 or 2gram/kg according to their response. |
| non-IVIg naive | Active comparator | Drug: Immunoglobulin (Hizentra)Participants already on IVIg will be observed for 12 weeks under their existing IVIg regimen and will undergo measurements of their muscle strength, skin changes, assessments of their daily activities and quality of life (QoL) every 4 weeks at scheduled visits for monthly maintenance IVIg infusions (weeks 0,4,8,12). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Men or woman aged >18 years
- Diagnosis of DM based on standard criteria
- Receiving the equivalent of at least 0.4 g/kg IVIg every 4 weeks (IVIg group only)
- Established response to IVIg or dependence on IVIg to maintain status established either by symptomatic worsening of condition at the end of the inter-dose interval for both groups or by worsening after reduction of the dose within the previous 12 months (IVIg group only)
- IVIg regimen stable for 12 weeks while on IVIg (minor changes are permitted provided that the dose change is 15% or less) (IVIg group only)
Key exclusion criteria (6 listed)
- Pregnancy, planned pregnancy, breast feeding or unwillingness to practice contraception
- Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
- Initiation or immunomodulatory treatment other than IVIg in the past 24 weeks or modification of immunomodulatory treatment other than IVIg in the past 12 weeks.
- Participation in trial of an investigational medicinal product in the past 12 weeks
- Presence of skin infection unrelated to dermatomyositis, severe skin involvement
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 |
|---|---|---|
| Immunoglobulin (Hizentra)Also: Immunoglobulin subcutaneous (human) 20% liquid, Hizentra | Drug | IVIg naive, non-IVIg naive |
Primary outcome
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
Time frame: The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
1 secondary outcome
The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Time frame: The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Results and safety
The study was stopped early (reason given: Difficult recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2018.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Immunoglobulin (Hizentra) in people with Dermatomyositis, run by Thomas Jefferson University 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 6 inclusion and 6 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: stopped early, as of the sponsor's last update on 16 May 2018. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Difficult recruitment). 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?
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28. (time frame: The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.). 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 16 May 2018; AzTrials last refreshed its copy on 8 Nov 2026.