Combination Therapy in Amyotrophic Lateral Sclerosis (ALS) (PNA)
The purpose of the study is to determine the safety and the efficacy of Tretinoin and Pioglitazone HCL in patients with ALS who are currently on Riluzole.
Amyotrophic Lateral Sclerosis
Drug: Pioglitazone and Tretinoin
Drug: Tretinoin and Pioglitazone HCL
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||Phase IIA Trial: Tretinoin and Pioglitazone HCL Combination Therapy in Amyotrophic Lateral Sclerosis|
- To evaluate the safety and efficacy of Tretinoin and Pioglitazone HCL in patients with ALS [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
- To determine if cerebrospinal fluid tau levels decline over the course of treatment and if the level of tau decline correlates with response in treatment measured by ALSFRS [ Time Frame: 3 years ] [ Designated as safety issue: No ]
|Study Start Date:||June 2008|
|Study Completion Date:||March 2012|
|Primary Completion Date:||November 2009 (Final data collection date for primary outcome measure)|
Active Comparator: Tretionoin and Pioglitazone HCL
20 patients will be randomized blindedly to Tretinoin and Pioglitazone HCL
Drug: Pioglitazone and Tretinoin
Tretinoin: 1 pill twice a day (10 mg/bid) Pioglitazone HCL: 1 pill once a day (30 mg/qd)Drug: Tretinoin and Pioglitazone HCL
Tretinoin 10 mg 1 pill po twice a day Pioglitazone HCL 30 mg 1 pill po once a day
Placebo Comparator: Sugar Pill
10 Patients will randomly receive placebo
Physicians at Phoenix Neurological Associates (PNA) are looking for individuals diagnosed with ALS to participate in a double-blind, placebo controlled study of Tretinoin and Pioglitazone HCL, used in combination with Riluzole, for treating ALS. This investigator initiated trial conducted by Dr. Todd Levine and Dr. David Saperstein will help determine whether Tretinoin and Pioglitazone HCL, in combination, can slow the progression of ALS.
At present, there is little to no effective therapy for ALS, nor is there a known cause. Therefore there is a tremendous unmet need for more effective therapy for this disease and that is why the physicians at PNA have been very interested in the role of developing a more active anti-excitotoxic cocktail for patients with ALS. Since recent data have suggested that inflammatory mechanisms may interact with and promote neurodegeneration (where cells in the spinal cord and brain are lost), there have been a number of anti-inflammatory treatment strategies that have been evaluated in animal models. Some studies have shown that mice that were given Pioglitazone HCL demonstrated improved muscle strength and body weight, exhibited a delayed disease onset as well as had a longer survival rate compared to non treated mice.
In addition to these studies, retinoic acids have been studied extensively in various models of the injured nervous system. These studies have shown that retinoic acids, such as Tretinoin may be neuroprotective and support axonal growth, which could in turn slow disease progression.
The purpose of this study is to determine if such a drug "cocktail" could offer the best chance of attaining a significant reduction in disease progression by utilizing currently available FDA-approved agents
|United States, Arizona|
|Phoenix Neurological Associates, LTD|
|Phoenix, Arizona, United States, 85018|
|Principal Investigator:||Todd D Levine, MD||Phoenix Neurological Associates, LTD|