Growth Hormone in Amyotrophic Lateral Sclerosis

NCT ID: NCT00635960

Last Updated: 2010-05-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-03-31
• Study Completion Date: 2010-05-31

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 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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

1

Patients randomly assigned to treatment

Group Type: EXPERIMENTAL

Growth Hormone (Somatropin)

Intervention Type: DRUG

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.

2

Patients randomly assigned to placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Same as for Growth hormone group

Interventions

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.

Intervention Type: DRUG

Placebo

Same as for Growth hormone group

Intervention Type: DRUG

Other Intervention Names

Saizen 8mg; Saizen 8mg placebo

Eligibility Criteria

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

• Minimum Eligible Age: 40 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Istituto Biostrutture e Immagini, CNR Naples

UNKNOWN

Sponsor Role: collaborator

Agenzia Italiana del Farmaco

OTHER_GOV

Sponsor Role: collaborator

Federico II University

OTHER

Sponsor Role: lead

Responsible Party

Dipartimento di Scienze Neurologiche, "Federico II" University, Naples

Principal Investigators

Alessandro Filla, MD

Role: PRINCIPAL_INVESTIGATOR

University "Federico II", Naples

Locations

Diparimento di Scienze Neurologiche

Naples, , Italy

Istituto Biostrutture e Bioimmagini, Consiglio Nazionale delle Ricerche

Naples, , Italy

Countries

Italy

Related Links

http://www.ibb.cnr.it/

Institute of Biostructure and Bioimaging, CNR Naples

http://www.unina.it

University "Federico II", Naples

http://www.policlinico.unina.it

Policlinico "Federico II", Naples

http://www.agenziafarmaco.it

AIFA

Other Identifiers

SLA_GH_1

Identifier Type: -

Identifier Source: org_study_id