Systemic Lupus Erythematosus (SLE)Phase 4Acthar SLENCT02779153
Acthar SLE (Systemic Lupus Erythematosus)
A Phase 4 study testing Acthar low dose (40 U) and Acthar high dose (80 U) in people with Systemic Lupus Erythematosus (SLE) and Repository Corticotropin Injection, run by NYU Langone Health.
Trial summary
This is a randomized study exploring the efficacy, safety and steroid sparing ability of two doses (40 U and 80 U) of Acthar in SLE patients with immune mediated hematologic manifestations requiring steroid use for a minimum of 2 weeks prior to screening. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Lupus Erythematosus (SLE), Repository Corticotropin Injection
- What is being tested?
- Acthar low dose (40 U) and Acthar high dose (80 U)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2016to 22 May 2019
- Phase
- Phase 4
- 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
- Able to provide informed consent.
- Diagnosis of SLE according to the American College of Rheumatology revised criteria (fulfilled ≥ 4 criteria).
- Hematologic BILAG scores due to hemolytic anemia or thrombocytopenia of BILAG A (including hemolytic anemia with hemoglobin < 8.0 g/dL or platelet count < 25 x 109 l) or BILAG B (including hemolytic anemia with hemoglobin 8.0 - 9.9 g/dDL or platelet count 25 - 49 x 109 l) based on screening laboratory assessment.
- Currently taking prednisone ≥ 7.5 mg or equivalent for hematologic SLE for at least 2 weeks prior to screening.
- Use of antimalarials and NSAIDs (stable regimen within the 4 weeks prior to screening), as well as methotrexate, azathioprine, and mycophenolate mofetil (stable regimen within the 4 weeks prior to screening) are permitted but not required. If used, the regimen must remain stable through the study. An increase or addition of SLE medication at any time during the study is not permitted.
- Ability to comply with study procedures, which include SC injections of study medication, adhering to concomitant medication restrictions, and attending scheduled office visits.
Who cannot join
- Patients with a recent history (< 2 week prior to screening) of starting prednisone or equivalent.
- Patients with active nephritis, defined as serum creatinine > 2.5 mg/dL or protein/creatinine ratio (PCR) > 1.5 g/g, or patients that required hemodialysis within 3 months prior to screening.
- Active central nervous system (CNS) lupus (including seizures, psychosis, organic brain syndrome, cerebrovascular accident, cerebritis, or CNS vasculitis), requiring therapeutic intervention within 3 months prior to screening.
- Type 1 or type 2 diabetes mellitus (history of gestational diabetes is not an exclusion), or patients currently taking hypoglycemic medication.
- Patients with a history of concomitant medication use as follows:
- Receipt of the following within 1 month prior to screening: i. Any steroid injection (IM, intraarticular, or IV) b. Receipt of any of the following within 3 months prior to screening: i. Cyclosporine ii. Any non-biologic investigational drug c. Receipt of the following within 4 months prior to screening: i. IVIg ii. Plasmapheresis d. Receipt of cyclophosphamide within 6 months prior to screening e. Receipt of the following within 12 months prior to screening: i. B cell targeted therapy (rituximab or other anti-CD20 agent, anti-CD22 [epratuzumab], anti-CD52 [alentuzumab], or belimumab) ii. Abatacept iii. Any biologic investigational agent
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- Contraindication per Acthar Prescribing Information: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction.
- For the purposes of this study, osteoporosis is defined as evidence of vertebral or long bone fracture, or lumbar spine T-score > 2.0 standard deviations below the mean of the reference population.
- For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening.
- For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.
- For the purposes of this study, uncontrolled hypertension is defined as a mean systolic blood pressure ≥ 140 mm Hg and/or diastolic blood pressure ≥ 90 mm Hg on ≥ 3 seated readings taken at least 5 minutes apart during the screening period.
- Reproductive status:
- Women who are pregnant,
- Women who are breastfeeding,
- Women of childbearing potential who are unwilling or unable to use an acceptable method of birth control to avoid pregnancy for the entire study period, as evaluated by the Investigator (women of non-childbearing potential are those that have a history of hysterectomy, bilateral oophorectomy, or are postmenopausal with no history of menstrual flow for ≥ 12 months prior to screening).
- Immune System: Known immunocompromised status (not related to SLE or therapies for SLE), including individuals who have undergone organ transplantation or who are known to be positive for the human immunodeficiency virus.
- Patients with acute or chronic hepatitis C, or active hepatitis B infection, positive interferon gamma release assay (IGRA) or signs or symptoms concerning for active tuberculosis (TB); or use of antibiotics (antibacterial, antiviral, antifungal, or antiparasitic agent) within 4 weeks of randomization for treatment of an active infection.
- Presence of any other clinically significant disease or disorder which, in the opinion of the Investigator (by its nature or by being inadequately controlled), might put the patient at risk due to participation in the study, or may influence the results of the study or the patient's ability to complete the study.
- Any clinically significant laboratory abnormality, based on the Investigator's judgment. The following laboratory exclusions apply for all patients:
- AST > 2.5 times the upper limit of normal (ULN)
- ALT > 2.5 times ULN
- Hemoglobin A1c > 6.5%
About this study
Official title: Efficacy, Safety, and Steroid Sparing Effect of Acthar in Patients With Hematologic Manifestations of Systemic Lupus Erythematosus
This is a randomized study exploring the efficacy, safety and steroid sparing ability of two doses (40 U and 80 U) of Acthar in SLE patients with immune mediated hematologic manifestations requiring steroid use for a minimum of 2 weeks prior to screening.
Read the detailed description
Acthar is currently labeled for use during an exacerbation or for maintenance therapy in selected SLE cases, however data from prospective trials on hematologic manifestations of SLE are not available. Due to the potential effect of lowering or eliminating autoantibodies, the absence of bone marrow suppression, and a steroid sparing effect, Acthar may represent a novel therapeutic option in recalcitrant cases of hematologic SLE.
- Being tested
- Acthar low dose (40 U), Acthar high dose (80 U)
- Keywords
- SLE, H.P. Acthar Gel
- Medical subject headings
- Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acthar low dose (40 U)Drug
Also called Repository Corticotropin Injection
The 40U group will administer 0.5 mL of study medication once a day. Patients will taper study medication during Week 2 through the remainder of the study and will administer 0.5 mL of study medication twice a week. The stable Acthar regimen should be maintained for the remainder of the study. However, dose adjustments may be implemented if needed based on safety.
Acthar high dose (80 U)Drug
Also called Repository Corticotropin Injection
The 80U group will administer 1.0 mL of study medication every day. If a patient meets dose reduction criteria, their assigned volume will be adjusted from 1.0 mL to 0.5 mL. Patients will taper study medication during Week 2 through the remainder of the study. However, dose adjustments may be implemented if needed based on safety.
| Study group | Role | What they receive |
|---|---|---|
| Acthar low dose (40 U) | Experimental | Not described Receives: Drug: Acthar low dose (40 U) |
| Acthar high dose (80 U) | Experimental | Not described Receives: Drug: Acthar high dose (80 U) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) Score
Disease activity is based on 24 questions, weighted across nine organ systems, combined into a total score that can range from 0 to 105, but are generally < 20, even with very active disease.
Measured: Baseline to Week 24
British Isles Lupus Assessment Group (BILAG) disease activity index Score
Items are rated as 0 (not present), 1 (improving), 2 (same), 3 (worse), or 4 (new) in the last 4 weeks compared with the previous 4 weeks. The numerical scores are used to categorize each organ system by an alphabetical score: 'A' reflecting severe disease requiring increases in prednisone to > 20 mg daily and/or addition of immunosuppressive agents, 'B' indicated less active disease, requiring low-dose prednisone and/or symptomatic treatment with NSAIDs and/or antimalarials, 'C' reflecting mild disease requiring only symptomatic therapy, 'D' reflecting previous organ system involvement without current disease activity, and 'E' reflecting no prior or no current disease involvement in that organ system.
Measured: Baseline to Week 24
Physician's Global Assessment Score
The Physician's Global Assessment is a 10 cm visual analogue scale (VAS) anchored at 0 (none) and 3 (severe) with intermediate lines at 1 (mild) and 2 (moderate). It is designed for the physician to indicate the patient's overall disease activity at a particular visit
Measured: Baseline to Week 24
SELENA Flare Index Score
The SELENA Flare Index categorizes SLE flare as 'mild or moderate' or 'severe' based on six variables
- Change in SLEDAI score from the most recent assessment to current.
- Change in signs or symptoms of disease activity.
- Change in prednisone dosage.
- Use of new medication for disease activity or hospitalization.
- Change in PGA score.
- Hospitalization for SLE activity (severe flare only).
Measured: Baseline to Week 24
SLICC/ACR Damage Index
The SLICC/ACR Damage Index measures irreversible organ damage from either the disease process or treatment, which has been present for ≥ 6 months, in 12 organ systems. It is an important predictor of long-term mortality and is an independent outcome measure separate from the SLEDAI.
Measured: Baseline to Week 24
Secondary outcomes (3)
Medical Outcomes Survey Short Form-36
The SF-36 has been validated in SLE and includes eight domains: physical functioning, role physical, bodily pain, general health perceptions, vitality (which includes fatigue, energy and vigor), social functioning, role emotional and mental-health impact, combined into two summary scores (physical component summary scores [PCS] and mental component summary scores [MCS])
Measured: Baseline to Week 24
FACIT Fatigue Scale
The FACIT-F scale is a 13-item questionnaire to evaluate self-reported fatigue and its impact upon daily activities and function. The responses are measured on a 4-point Likert scale, with the total score ranging from 0 to 52. The FACIT-F scale has been validated in SLE patients.
Measured: Baseline to Week 24
Patient's Global Assessment
Subjects rate their global assessment of SLE disease activity on each visit, in response to the question "Considering all the ways your SLE affects you, please mark a vertical line on the scale below for how are you feeling today?" using a 100mm visual analogue scale, where 0 is "very good, no symptoms" and 100 is "very poor, very severe symptoms."
Measured: Baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2016 (estimated)
- Study startOct 2016
- Primary completion22 May 2019
- Study completion22 May 2019
Why it stopped: closed in agreement of the sponsor and institution due to lack of patient accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Lupus Erythematosus (SLE)Studies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe 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
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor
- Investigators
- Amit Saxena, MD, Principal investigator, New York University Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02779153
- Sponsor's protocol ID
- 15-00295
- Acronym
- Acthar SLE
- Status last verified
- Sep 2019
- First submitted
- 18 May 2016
- Last updated
- 13 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy, Safety, and Steroid Sparing Effect of Acthar in Patients With Hematologic Manifestations of Systemic Lupus Erythematosus
- ClinicalTrials.gov ID
- NCT02779153
- Acronym / sponsor ID
- Acthar SLE / 15-00295
- Keywords
- SLE, H.P. Acthar Gel
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: closed in agreement of the sponsor and institution due to lack of patient accrual.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- Oct 2016
- Primary completion
- 22 May 2019 (actual)
- Study completion
- 22 May 2019 (actual)
- First posted
- 20 May 2016 (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.
This is a randomized study exploring the efficacy, safety and steroid sparing ability of two doses (40 U and 80 U) of Acthar in SLE patients with immune mediated hematologic manifestations requiring steroid use for a minimum of 2 weeks prior to screening.
Detailed description
Acthar is currently labeled for use during an exacerbation or for maintenance therapy in selected SLE cases, however data from prospective trials on hematologic manifestations of SLE are not available. Due to the potential effect of lowering or eliminating autoantibodies, the absence of bone marrow suppression, and a steroid sparing effect, Acthar may represent a novel therapeutic option in recalcitrant cases of hematologic SLE.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Acthar low dose (40 U) | Experimental | Drug: Acthar low dose (40 U) |
| Acthar high dose (80 U) | Experimental | Drug: Acthar high dose (80 U) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Able to provide informed consent.
- Diagnosis of SLE according to the American College of Rheumatology revised criteria (fulfilled ≥ 4 criteria).
- Hematologic BILAG scores due to hemolytic anemia or thrombocytopenia of BILAG A (including hemolytic anemia with hemoglobin < 8.0 g/dL or platelet count < 25 x 109 l) or BILAG B (including hemolytic anemia with hemoglobin 8.0 - 9.9 g/dDL or platelet count 25 - 49 x 109 l) based on screening laboratory assessment.
- Currently taking prednisone ≥ 7.5 mg or equivalent for hematologic SLE for at least 2 weeks prior to screening.
- Use of antimalarials and NSAIDs (stable regimen within the 4 weeks prior to screening), as well as methotrexate, azathioprine, and mycophenolate mofetil (stable regimen within the 4 weeks prior to screening) are permitted but not required. If used, the regimen must remain stable through the study. An increase or addition of SLE medication at any time during the study is not permitted.
Key exclusion criteria (22 listed)
- Patients with a recent history (< 2 week prior to screening) of starting prednisone or equivalent.
- Patients with active nephritis, defined as serum creatinine > 2.5 mg/dL or protein/creatinine ratio (PCR) > 1.5 g/g, or patients that required hemodialysis within 3 months prior to screening.
- Active central nervous system (CNS) lupus (including seizures, psychosis, organic brain syndrome, cerebrovascular accident, cerebritis, or CNS vasculitis), requiring therapeutic intervention within 3 months prior to screening.
- Type 1 or type 2 diabetes mellitus (history of gestational diabetes is not an exclusion), or patients currently taking hypoglycemic medication.
- Patients with a history of concomitant medication use as follows:
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 |
|---|---|---|
| Acthar low dose (40 U)Also: Repository Corticotropin InjectionThe 40U group will administer 0.5 mL of study medication once a day. Patients will taper study medication during Week 2 through the remainder of the study and will administer 0.5 mL of study medication twice a week. The stable Acthar regimen should be maintained for the remainder of the study. However, dose adjustments may be implemented if needed based on safety. | Drug | Acthar low dose (40 U) |
| Acthar high dose (80 U)Also: Repository Corticotropin InjectionThe 80U group will administer 1.0 mL of study medication every day. If a patient meets dose reduction criteria, their assigned volume will be adjusted from 1.0 mL to 0.5 mL. Patients will taper study medication during Week 2 through the remainder of the study. However, dose adjustments may be implemented if needed based on safety. | Drug | Acthar high dose (80 U) |
Primary outcome
Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) Score
Time frame: Baseline to Week 24
Disease activity is based on 24 questions, weighted across nine organ systems, combined into a total score that can range from 0 to 105, but are generally < 20, even with very active disease.
British Isles Lupus Assessment Group (BILAG) disease activity index Score
Time frame: Baseline to Week 24
Items are rated as 0 (not present), 1 (improving), 2 (same), 3 (worse), or 4 (new) in the last 4 weeks compared with the previous 4 weeks. The numerical scores are used to categorize each organ system by an alphabetical score: 'A' reflecting severe disease requiring increases in prednisone to > 20 mg daily and/or addition of immunosuppressive agents, 'B' indicated less active disease, requiring low-dose prednisone and/or symptomatic treatment with NSAIDs and/or antimalarials, 'C' reflecting mild disease requiring only symptomatic therapy, 'D' reflecting previous organ system involvement without current disease activity, and 'E' reflecting no prior or no current disease involvement in that organ system.
Physician's Global Assessment Score
Time frame: Baseline to Week 24
The Physician's Global Assessment is a 10 cm visual analogue scale (VAS) anchored at 0 (none) and 3 (severe) with intermediate lines at 1 (mild) and 2 (moderate). It is designed for the physician to indicate the patient's overall disease activity at a particular visit
SELENA Flare Index Score
Time frame: Baseline to Week 24
The SELENA Flare Index categorizes SLE flare as 'mild or moderate' or 'severe' based on six variables * Change in SLEDAI score from the most recent assessment to current. * Change in signs or symptoms of disease activity. * Change in prednisone dosage. * Use of new medication for disease activity or hospitalization. * Change in PGA score. * Hospitalization for SLE activity (severe flare only).
SLICC/ACR Damage Index
Time frame: Baseline to Week 24
The SLICC/ACR Damage Index measures irreversible organ damage from either the disease process or treatment, which has been present for ≥ 6 months, in 12 organ systems. It is an important predictor of long-term mortality and is an independent outcome measure separate from the SLEDAI.
3 secondary outcomes
Medical Outcomes Survey Short Form-36
Time frame: Baseline to Week 24
FACIT Fatigue Scale
Time frame: Baseline to Week 24
Patient's Global Assessment
Time frame: Baseline to Week 24
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2019.
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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.
Amit Saxena, MD principal investigator
New York University Medical School
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Acthar low dose (40 U) and Acthar high dose (80 U) in people with Systemic Lupus Erythematosus (SLE) and Repository Corticotropin Injection, run by NYU Langone Health.
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 22 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: withdrawn, as of the sponsor's last update on 13 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 withdrawn before enrolling participants, so there are no results.
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?
Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) Score (time frame: Baseline to Week 24). The study lists 5 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 13 Sep 2019.