NCT01753401|CompletedPhase 4Results

Acthar for the Treatment of Systemic Lupus Erythematosus (SLE) in Patients With a History of Persistently Active Disease

A Two-part Study Exploring the Efficacy, Safety, and Pharmacodynamics of Acthar in Systemic Lupus Erythematosus Patients With a History of Persistently Active Disease

Mallinckrodt ClinicalTrials.gov

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1 other identifier

QSC01-SLE-01

Study Type

interventional

Target

Locations

1 country

Sites

Timeline

RegisteredDec 2012

StartedJan 2013

CompletionOct 2015

Brief Summary

This Phase 4 study is being performed to examine the effects of Acthar for the indicated use of treatment of SLE. This study will enroll patients with steroid-dependent, persistently active SLE with arthritic and/or cutaneous involvement. The study will involve two periods: an 8-week double-blind period, to provide placebo-controlled safety, efficacy, and pharmacodynamic data, and an optional open-label period, to examine the prolonged effects of Acthar maintenance.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_4

Timeline

Completed

Started Jan 2013

Typical duration for phase_4

Geographic Reach

1 country

22 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.7 years study duration

First Submitted

Initial submission to the registry

December 17, 2012

Completed

3 days until next milestone

First Posted

Study publicly available on registry

December 20, 2012

Completed

12 days until next milestone

Study Start

First participant enrolled

January 1, 2013

Completed

2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2015

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2015

Completed

4.4 years until next milestone

Results Posted

Study results publicly available

February 27, 2020

Completed

Last Updated

February 27, 2020

Status Verified

August 1, 2017

Enrollment Period

2.7 years

First QC Date

December 17, 2012

Results QC Date

December 6, 2019

Last Update Submit

February 14, 2020

Conditions

Systemic Lupus Erythematosus (SLE)

Keywords

SLELupus

Outcome Measures

Primary Outcomes (1)

Number of Participants Who Meet the Definition of a Responder Within 4 Weeks
Participants are counted as responders based on two SLE indices: the Systemic Lupus Erythematosus Disease Activity Index amended by the SELENA group (SELENA-SLEDAI) and the British Isles Lupus Assessment Group (BILAG) Index. * decrease in SELENA-SLEDAI score from 4 to 0 for the arthritis descriptor (highest possible score is 4) and no worsening in other organ systems based on BILAG OR * decrease in SELENA-SLEDAI score from 2 to 0 for rash (highest possible score is 2) and no worsening in other organ systems based on BILAG The BILAG is a transitional index that captures changing severity of clinical manifestations. It has an ordinal scale scoring system by design that produces an overview of disease activity across eight systems. The individual system scores were not intended to be summated into a global score.
within 4 weeks

Secondary Outcomes (20)

Number of Participants Who Meet the Definition of a Responder Within 8 Weeks
within 8 weeks
Score on the SELENA-SLEDAI Within 8 Weeks
within 8 weeks
BILAG Total Score Within 8 Weeks
within 8 weeks
Physician's Global Assessment (PGA) of Disease Severity at Baseline
at Baseline
Physician's Global Assessment (PGA) of Disease Severity at Week 4
at Week 4
+15 more secondary outcomes

Study Arms (4)

Period 2: Placebo/Acthar

EXPERIMENTAL

Participants receive Placebo in Part 1, but after completion of Week 8 in the double-blind phase, patients who received Placebo may choose to participate in the optional open-label phase where they will receive an Acthar maintenance regimen for 44 weeks. The initial Acthar regimen will be assigned based on the study medication regimen the patient received at the completion of the double-blind phase (Visit 6, Week 8). Acthar regimen during Part 2 may be adjusted based on the Investigator's judgment with the goal of achieving a stable Acthar regimen no later than Week 28. Once the stable Acthar regimen is achieved, the Investigator should consider tapering the steroid regimen to a low daily dose or completely discontinue.

Drug: ActharDrug: PlaceboDrug: Steroid Drug

Period 2: Acthar/Acthar

EXPERIMENTAL

After completion of Week 8 in the double-blind phase, patients who received Acthar may choose to participate in the optional open-label phase where they will receive an Acthar maintenance regimen for 44 weeks. The initial Acthar regimen will be assigned based on the study medication regimen the patient received at the completion of the double-blind phase (Visit 6, Week 8). Acthar regimen may be adjusted based on the Investigator's judgment with the goal of achieving a stable Acthar regimen no later than Week 28. Once the stable Acthar regimen is achieved, the Investigator should consider tapering the steroid regimen to a low daily dose or completely discontinue.

Drug: ActharDrug: Steroid Drug

Period 1: Placebo

PLACEBO COMPARATOR

Participants receive matching placebo (in 0.5 mL daily or in 1 mL every other day) for 4 weeks. In Weeks 5-8, they will taper the study medication. Participants will continue on their stable steroid regimen during this phase of the study. After completion of Week 8 in the double-blind phase, they may choose to participate in the optional open-label phase. Participants will continue on their stable steroid regimen during this phase of the study.

Drug: PlaceboDrug: Steroid Drug

Period 1: Acthar

EXPERIMENTAL

Participants receive Acthar (40 units in 0.5 mL daily or 80 units in 1 mL every other day) for 4 weeks. In Weeks 5-8, they will taper the study medication. Participants will continue on their stable steroid regimen during this phase of the study. After completion of Week 8 in the double-blind phase, they may choose to participate in the optional open-label phase. Participants will continue on their stable steroid regimen during this phase of the study.

Drug: ActharDrug: Steroid Drug

Interventions

ActharDRUG

Acthar is given by subcutaneous (SC) injection (shot under the skin), at a dose of 40 units daily or 80 units every other day

Also known as: H.P. Acthar Gel, Repository Corticotropin Injection

Period 1: ActharPeriod 2: Acthar/ActharPeriod 2: Placebo/Acthar

PlaceboDRUG

Placebo contains the same inactive ingredients as Acthar, and is given by SC injection

Also known as: Matching Placebo

Period 1: PlaceboPeriod 2: Placebo/Acthar

Steroid DrugDRUG

The patient's steroid regimen 7.5 to 30 mg/day of prednisone or equivalent, chronic/stable within the 4 weeks prior to screening.

Also known as: Prednisone or equivalent

Period 1: ActharPeriod 1: PlaceboPeriod 2: Acthar/ActharPeriod 2: Placebo/Acthar

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Male or female ≥ 18 years of age at screening who are able to provide informed consent
Diagnosis of SLE according to the American College of Rheumatology revised criteria (fulfilled ≥ 4 criteria)
Active SLE with arthritic and/or cutaneous involvement as demonstrated by a SELENA-SLEDAI score ≥ 2 (clinical manifestation must include rash and/or arthritis)
Moderate to severe rash and/or arthritis as demonstrated by BILAG score A or B in the mucocutaneous and/or musculoskeletal body systems
Documented history of autoantibodies to at least one of the following: anti-dsDNA, anti-Smith, or anti-cardiolipin
Documented history of positive antinuclear antibody (ANA)
Currently on a stable dose of prednisone (7.5 to 30 mg/day of prednisone or equivalent within the 4 weeks prior to screening). The prednisone regimen must remain stable through the double-blind phase and until the stable Acthar regimen is attained in the open-label phase.

You may not qualify if:

Patients with a recent history (≤ 2 months prior to screening) of starting prednisone (or equivalent) use
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
History of using certain medications prior to screening:
oral prednisone (or equivalent) \> 30 mg/day, any steroid injection, cyclosporine, or non-biologic investigational drug within 3 months prior to screening
intravenous immunoglobulin (IVIg) or plasmapheresis within 4 months prior to screening
cyclophosphamide within 6 months prior to screening; and/or
B-cell targeted therapy, abatacept, or any biologic investigational agent within 12 months prior to screening
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 vertebral T-score \> 2.0
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

Contact the study team to confirm eligibility.