Clinical Trial: Growth Hormone in Amyotrophic Lateral Sclerosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Efficacy, Safety and Tolerability of Growth Hormone in Patients With Amyotrophic Lateral Sclerosis as add-on Therapy to Riluzole

Brief Summary: Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline, etc. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients.

Detailed Summary:

Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. When administered to SOD-1 transgenic mice, IGF-I prolongs survival, ameliorates muscular strength, and reduces weight and motor neuron loss, astrocyte gliosis, and ubiquitin positive protein inclusions.

Two clinical trials have been performed in ALS patients with s.c. administration of IGF-I indicating a possible beneficial effect, and a third clinical trial is in progress. Methionyl growth hormone (mGH) showed no effect on survival, disease progression and muscular strength. MGH was administered at a fixed dose and peripheral production of IGF-I appeared to be normal. We propose a double-blind trial of Growth Hormone (GH) as add-on therapy to Riluzole, with an individually regulated dose based on the peripheral response of IGF-I. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients. As secondary objectives, effect of GH on mortality, QoL, and motor function will be assessed.


Sponsor: Federico II University

Current Primary Outcome: Primary endpoint is the N-acetylaspartate/Creatine ratio in the motor cortex assessed with magnetic resonance spectroscopy. [ Time Frame: 0, 6 and 12 months after treatment start ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Difference in mortality between groups [ Time Frame: 12 months ]
  • Difference in the ALS-FRS score (motor function scale) [ Time Frame: 0, 6, and 12 months after treatment start ]
  • Difference in the SF-36 score (quality of life ) [ Time Frame: 0, 6, and 12 monthst after treatmetn start ]
  • Safety and tolerability [ Time Frame: 12 months ]


Original Secondary Outcome: Same as current

Information By: Federico II University

Dates:
Date Received: March 3, 2008
Date Started: March 2007
Date Completion:
Last Updated: May 25, 2010
Last Verified: May 2010