Safety and Efficacy of Acthar Gel in an Outpatient Dialysis Population

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.

Study Overview

• Status: Terminated
• Conditions: End Stage Renal Disease
• Intervention / Treatment: Drug: Acthar Gel

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.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Canfield, Ohio, United States, 44406
      • Center for Dialysis Care 3695 Stutz Drive

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: 80 Units of Acthar; Subjects assigned by random assignment to receive 80 Units of Acthar Gel 2x weekly for 6 months	Drug: Acthar Gel; Subjects will be given an injection subcutaneously 2x week for 6 months. Dosing will be randomly assigned as either 80 U or 40 U; Other Names: repository corticotropin injection
Active Comparator: 40 Units of Acthar; Subjects assigned by random assignment to receive 40 Units of Acthar Gel 2x weekly for 6 months	Drug: Acthar Gel; Subjects will be given an injection subcutaneously 2x week for 6 months. Dosing will be randomly assigned as either 80 U or 40 U; Other Names: repository corticotropin injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Renal Function	Maintain or improve renal function [Creatinine clearance and Proteinuria] by ≥ 20% , as measured by eGFR or 24 hour urine study.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hyperparathyroidism	Change in serum parathyroid hormone levels	6 months
Anemia	Change in hemoglobin levels	6 months
Inflammatory blood markers	Change in serum C-Reactive Protein and Creatine Kinase	6 months
Vitamin D uptake	Change in Vitamin D dosing requirements	6 months
Hypercalcemia	Change in serum calcium levels	6 months
Phosphate	Change in serum phosphate levels	6 months
Total Iron Binding Capacity	Change in Total Iron Binding Capacity in serum	6 months
Epogen Dose	Change in epogen dosing requirements	6 months
Fall risk	Timed Up and Go Test will improve	6 months
Walking endurance	2 minute walk distance	6 months
Gait speed	10 meter gait speed test	6 months
Mobility disablement risk	Short Performance Physical Battery	6 months
Habitual Physical Activity	Physical Activity Scale for the Elderly	6 months
Calf Strength	Heel Rise Test	6 months
Quality of life	Medical Outcome Study Short Form 36	6 months
Fear of Falling	ABC Scale [Activities-specific Balance Confidence Scale-Powell]. Scores to be above 67.	6 months
Self- Report Physical Activity	RAPA [Rapid Assessment of Physical Activity-Univ of Washington]	6 months
Nutritional Status	Body Mass Index	6 months
Lean body mass	Body Composition examination using Noori (2012) regression equation for lean body mass.	6 months
Caloric intake	Food Frequency Questionnaire (indicator of habitual intake)	6 months
Strength	Muscle force measures in 8 muscle groups	6 months
Nutritional Indices	Pre-Albumin	6 months

Collaborators and Investigators

• Sponsor: Youngstown State University
• Investigators: Principal Investigator: Erdal Sarac, MD, Renal Group

Study record dates

Study Major Dates

• Study Start: March 1, 2015
• Primary Completion (Actual): July 1, 2016
• Study Completion (Actual): July 1, 2016

Study Registration Dates

• First Submitted: June 8, 2015
• First Submitted That Met QC Criteria: June 26, 2015
• First Posted (Estimate): July 1, 2015

Study Record Updates

• Last Update Posted (Actual): February 10, 2017
• Last Update Submitted That Met QC Criteria: February 8, 2017
• Last Verified: February 1, 2017

More Information

Terms related to this study

• Keywords: Dialysis; Kidney; Acthar; Non-Diabetic
• Additional Relevant MeSH Terms: Kidney Diseases; Urologic Diseases; Renal Insufficiency; Renal Insufficiency, Chronic; Kidney Failure, Chronic; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Adrenocorticotropic Hormone
• Other Study ID Numbers: 014-2015

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO