Clinical trial NCT02486744: Safety and Efficacy of Acthar Gel in an Outpatient Dialysis Population
Official title: Safety and Efficacy of Acthar Gel in an Outpatient Dialysis Population
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: End Stage Renal Disease
Keywords: Acthar
Dialysis
Kidney
Non-Diabetic
Lead sponsor: Youngstown State University
Sponsor class: OTHER
First posted: 2015-07-01
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This will be a prospective observational study of Acthar Gel in Non-Diabetic Hemodialysis \[NDHD\] patients receiving dialysis for ≤ 2 years. The project will aimed at providing proof-of-concept data that 80 U two times \[2x\] week Acthar for 6 months is a safe and effective therapy for NDHD. Effectiveness of lower dose 40 U 2X week will also be determined. Therefore the study will be a repeated measures design (Time x condition) comparison of improvement in renal function, nutritional status, quality of life and physical performance resulting from Acthar therapy.

Detailed description: Recent reports demonstrated Acthar gel is effective to induce remission of proteinuria in the Nephrotic Syndrome patients (Bomback 2011, 2012). There are limited published reports documenting the clinical response (creatinine, proteinuria, serum albumin and cholesterol) to ACTHAR therapy in a non-diabetic hemodialysis population. In addition to renal dysfunction and high risk for mortality, individuals who receive dialysis also have decreased strength, low exercise capacity, poor physical functioning, and a low quality of life (Edgell 1996, Johansen, 2001). Strength deficits are well documented in those with End Stage Renal Disease \[ESRD\] (Cheema 2010, Yoda 2012). There are several reports documenting an association between strength and gait deficits and other measures of physical performance in persons with ESRD (Bohannon 1994, Fitts 1997, Segura-Orti 2011). It is well known that measures of physical performance may predict risk for fall and hospitalization in older individuals (Guralnik JM, 2000).

Deficits to physical performance may be further compounded by poor nutritional status. Persons with chronic renal disease, particularly in the context of hemodialysis or chronic renal replacement therapy, are often malnourished and/or are affected by abnormal micronutrient status (McMahon 2012). Furthermore, previous studies have shown that more than a third of acute-care, nephrology-related admissions are characterized by malnutrition (Lim 2012), and that it affects from 23% - 76% of all patients receiving hemodialysis (including outpatients)(Blumenkrantz 1980, Ikizler 1996, Pecoits-Filho 2002).

Decreased functional status and concomitant malnutrition contribute to increased hospital readmissions, prolonged length of stay (and inevitably, increased medical costs), and increased morbidity and mortality patients (Isabel 2003, Lim 2012). Given the high prevalence of these characteristics in persons with renal disease, it is important to prioritize identification of novel and effective means by which to sustain and improve the functional capability of these patients, and to maintain their nutritional status and attenuate malnutrition.

To date, there are no reports of the impact of Acthar gel therapy on renal function, strength, physical performance, nutritional status and quality of life in NDHD patients. This study will determine if Acthar gel therapy will maintain or improve overall kidney function as measured with 24 hour urine study at baseline and at the end of study period. In addition this study will determine if Acthar gel therapy will improve nutritional, physical and biochemical status in an outpatient non-diabetic hemodialysis population.

Eligibility criteria: Inclusion Criteria:

* Inclusion criteria will be individuals requiring dialysis for treatment of ESRD who have had poor response to immunosuppressive strategies.
* Non-Diabetic
* Adults age 18-80

Exclusion Criteria:

* receiving hemodialysis for \> 5 years
* diabetic, less than 18 years of age
* are pregnant
* have a history of cancer in the last 5 years
* have an active infection
* have recently had a myocardial infarction (within 6 weeks)
* have malignant arrhythmias, unstable angina, uncontrolled hypertension (SBP\> 180 and/or DBP \> 105)
* recent hospitalization (\< 30 days),
* ocular disease,
* accelerated osteoporosis,
* gastrointestinal diseases (ulcerative colitis, diverticulitis, myasthenia gravis)
* any disorder that may be exacerbated by short periods of activity.
* cognitive dysfunction
* neurological deficits leading to limited ambulation.

Primary outcomes:
measure: Renal Function
timeFrame: 6 months
Secondary outcomes:
measure: Hyperparathyroidism
timeFrame: 6 months
measure: Anemia
timeFrame: 6 months
measure: Inflammatory blood markers
timeFrame: 6 months
measure: Vitamin D uptake
timeFrame: 6 months
measure: Hypercalcemia
timeFrame: 6 months
measure: Phosphate
timeFrame: 6 months
measure: Total Iron Binding Capacity
timeFrame: 6 months
measure: Epogen Dose
timeFrame: 6 months
measure: Fall risk
timeFrame: 6 months
measure: Walking endurance
timeFrame: 6 months
measure: Gait speed
timeFrame: 6 months
measure: Mobility disablement risk
timeFrame: 6 months
measure: Habitual Physical Activity
timeFrame: 6 months
measure: Calf Strength
timeFrame: 6 months
measure: Quality of life
timeFrame: 6 months
measure: Fear of Falling
timeFrame: 6 months
measure: Self- Report Physical Activity
timeFrame: 6 months
measure: Nutritional Status
timeFrame: 6 months
measure: Lean body mass
timeFrame: 6 months
measure: Caloric intake
timeFrame: 6 months
measure: Strength
timeFrame: 6 months
measure: Nutritional Indices
timeFrame: 6 months
Other outcomes:
Locations:
city: Canfield
country: United States
facility: Center for Dialysis Care 3695 Stutz Drive
state: Ohio
Study officials:
affiliation: Renal Group
name: Erdal Sarac, MD
role: PRINCIPAL_INVESTIGATOR
References: