Growth Hormone in Amyotrophic Lateral Sclerosis

May 25, 2010 updated by: Federico II University

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Anticipated)

40

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Naples, Italy, 80131
        • Diparimento di Scienze Neurologiche
      • Naples, Italy, 80131
        • Istituto Biostrutture e Bioimmagini, Consiglio Nazionale delle Ricerche

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

40 years to 85 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Definite/probable ALS according to El Escorial criteria
  • Aged > 40, < 85 years
  • Progression from onset
  • Disease duration ≤3 years
  • Treatment with Riluzole

Exclusion Criteria:

  • Rapid disease progression in the first 6 months after diagnosis
  • Patients with tracheostomy and/or Gastrostomy
  • Disease duration > 3 years
  • Patient with exclusive bulbar or 2° motorneuron involvement
  • Hepatic/renal failure
  • Pregnant or breastfeeding
  • Signs of active neoplasia
  • Complicated Diabetes
  • Severe hypertension
  • Unable to undergo MRI exams

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
Patients randomly assigned to treatment
Drug: Growth Hormone (Somatropin)
The initial dose will be 2U s.c. every other day. The dose will be progressively increased to reach 1.5-2x the normal levels of IGF-I.
Other Names:
  • Saizen 8mg
Placebo Comparator: 2
Patients randomly assigned to placebo
Drug: Placebo
Same as for Growth hormone group
Other Names:
  • Saizen 8mg placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
0, 6 and 12 months after treatment start

Secondary Outcome Measures

Outcome Measure
Time Frame
Difference in mortality between groups
Time Frame: 12 months
12 months
Difference in the ALS-FRS score (motor function scale)
Time Frame: 0, 6, and 12 months after treatment start
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
0, 6, and 12 monthst after treatmetn start
Safety and tolerability
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Federico II University

Collaborators

Agenzia Italiana del Farmaco
Istituto Biostrutture e Immagini, CNR Naples

Investigators

  • Principal Investigator: Alessandro Filla, MD, University "Federico II", Naples

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2007

Primary Completion (Actual)

July 1, 2009

Study Completion (Actual)

May 1, 2010

Study Registration Dates

First Submitted

March 3, 2008

First Submitted That Met QC Criteria

March 13, 2008

First Posted (Estimate)

March 14, 2008

Study Record Updates

Last Update Posted (Estimate)

May 27, 2010

Last Update Submitted That Met QC Criteria

May 25, 2010

Last Verified

May 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SLA_GH_1

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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