Growth Hormone in Amyotrophic Lateral Sclerosis

NCT00635960 | Completed Phase 2 DMC

• 1 other identifier: SLA_GH_1
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis; ALS; Growth Hormone; IGF-I

Outcome Measures

Primary Outcomes (1)

• Primary endpoint is the N-acetylaspartate/Creatine ratio in the motor cortex assessed with magnetic resonance spectroscopy.

  0, 6 and 12 months after treatment start

Secondary Outcomes (4)

• Difference in mortality between groups

  12 months

• Difference in the ALS-FRS score (motor function scale)

  0, 6, and 12 months after treatment start

• Difference in the SF-36 score (quality of life )

  0, 6, and 12 monthst after treatmetn start

• Safety and tolerability

  12 months

Study Arms (2)

1

EXPERIMENTAL

Patients randomly assigned to treatment

Drug: Growth Hormone (Somatropin)

2

PLACEBO COMPARATOR

Patients randomly assigned to placebo

Drug: Placebo

Interventions

Growth Hormone (Somatropin) 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.

Also known as: Saizen 8mg

1

Placebo DRUG

Same as for Growth hormone group

Also known as: Saizen 8mg placebo

2

Eligibility Criteria

• Age: 40 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Federico II University: lead
• Istituto Biostrutture e Immagini, CNR Naples: collaborator
• Agenzia Italiana del Farmaco: collaborator

Study Sites (2)

Diparimento di Scienze Neurologiche

Naples, 80131, Italy

Location

Istituto Biostrutture e Bioimmagini, Consiglio Nazionale delle Ricerche

Naples, 80131, Italy

Location

Related Links

• Institute of Biostructure and Bioimaging, CNR Naples
• University "Federico II", Naples
• Policlinico "Federico II", Naples
• AIFA

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Growth Hormone; Human Growth Hormone

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Alessandro Filla, MD

  University "Federico II", Naples

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: March 3, 2008
• First Posted: March 14, 2008
• Study Start: March 1, 2007
• Primary Completion: July 1, 2009
• Study Completion: May 1, 2010
• Last Updated: May 27, 2010

Record last verified: 2010-05

Locations

IT (2)