Growth Hormone in Amyotrophic Lateral Sclerosis

This study has been completed.
Sponsor:
Collaborators:
Istituto Biostrutture e Immagini, CNR Naples
Agenzia Italiana del Farmaco
Information provided by:
Federico II University
ClinicalTrials.gov Identifier:
NCT00635960
First received: March 3, 2008
Last updated: May 25, 2010
Last verified: May 2010

March 3, 2008
May 25, 2010
March 2007
July 2009   (final data collection date for primary outcome measure)
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 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00635960 on ClinicalTrials.gov Archive Site
  • Difference in mortality between groups [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Difference in the ALS-FRS score (motor function scale) [ Time Frame: 0, 6, and 12 months after treatment start ] [ Designated as safety issue: No ]
  • Difference in the SF-36 score (quality of life ) [ Time Frame: 0, 6, and 12 monthst after treatmetn start ] [ Designated as safety issue: Yes ]
  • Safety and tolerability [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
Growth Hormone in Amyotrophic Lateral Sclerosis
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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis
  • 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 Name: Saizen 8mg
  • Drug: Placebo
    Same as for Growth hormone group
    Other Name: Saizen 8mg placebo
  • Experimental: 1
    Patients randomly assigned to treatment
    Intervention: Drug: Growth Hormone (Somatropin)
  • Placebo Comparator: 2
    Patients randomly assigned to placebo
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
40
May 2010
July 2009   (final data collection date for primary outcome measure)

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
Both
40 Years to 85 Years
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
NCT00635960
SLA_GH_1
Yes
Alessandro Filla, Prof., Dipartimento di Scienze Neurologiche, "Federico II" University, Naples
Federico II University
  • Istituto Biostrutture e Immagini, CNR Naples
  • Agenzia Italiana del Farmaco
Principal Investigator: Alessandro Filla, MD University "Federico II", Naples
Federico II University
May 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP