Amyotrophic Lateral SclerosisPhase 2NCT00635960
Growth Hormone in Amyotrophic Lateral Sclerosis
A Phase 2 study testing Growth Hormone (Somatropin) in people with Amyotrophic Lateral Sclerosis, run by Federico II University at 2 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 40 to 85 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Growth Hormone (Somatropin)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 2 sitesItaly
- Study dates
- Mar 2007to May 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Definite/probable ALS according to El Escorial criteria
- Aged > 40, < 85 years
- Progression from onset
- Disease duration ≤3 years
- Treatment with Riluzole
Who cannot join
- 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
Show all 10Show fewer
- Signs of active neoplasia
- Complicated Diabetes
- Severe hypertension
- Unable to undergo MRI exams
Trial locations and contacts
2 sites in 1 country.
Italy2 sites
Diparimento di Scienze Neurologiche
Naples, 80131
Istituto Biostrutture e Bioimmagini, Consiglio Nazionale delle Ricerche
Naples, 80131
About this study
Official title: 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.
Read the 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
- Being tested
- Growth Hormone (Somatropin), Placebo
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, Growth Hormone, IGF-I
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Growth Hormone (Somatropin)Drug
Also called Saizen 8mg
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.
PlaceboDrug
Also called Saizen 8mg placebo
Same as for Growth hormone group
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Patients randomly assigned to treatment Receives: Drug: Growth Hormone (Somatropin) |
| 2 | Placebo comparator | Patients randomly assigned to placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary endpoint is the N-acetylaspartate/Creatine ratio in the motor cortex assessed with magnetic resonance spectroscopy.
Measured: 0, 6 and 12 months after treatment start
Secondary outcomes (4)
Difference in mortality between groups
Measured: 12 months
Difference in the ALS-FRS score (motor function scale)
Measured: 0, 6, and 12 months after treatment start
Difference in the SF-36 score (quality of life )
Measured: 0, 6, and 12 monthst after treatmetn start
Safety and tolerability
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2008 (estimated)
- Study startMar 2007
- Primary completionJul 2009
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Federico II UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federico II University
- Sponsor type
- University, hospital or other
- Collaborators
- Istituto Biostrutture e Immagini, CNR Naples, Agenzia Italiana del Farmaco
- Investigators
- Alessandro Filla, MD, Principal investigator, University "Federico II", Naples
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00635960
- Sponsor's protocol ID
- SLA_GH_1
- Status last verified
- May 2010
- First submitted
- 3 Mar 2008
- Last updated
- 27 May 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy, Safety and Tolerability of Growth Hormone in Patients With Amyotrophic Lateral Sclerosis as add-on Therapy to Riluzole
- ClinicalTrials.gov ID
- NCT00635960
- Acronym / sponsor ID
- SLA_GH_1
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, Growth Hormone, IGF-I
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Federico II UniversityUniversity, hospital or other
- Collaborators
- Istituto Biostrutture e Immagini, CNR Naples, Agenzia Italiana del Farmaco
- Study start
- Mar 2007
- Primary completion
- Jul 2009 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 14 Mar 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Growth Hormone (Somatropin)Patients randomly assigned to treatment |
| 2 | Placebo comparator | Drug: PlaceboPatients randomly assigned to placebo |
Who may qualify
- Ages
- 40 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Definite/probable ALS according to El Escorial criteria
- Aged > 40, < 85 years
- Progression from onset
- Disease duration ≤3 years
- Treatment with Riluzole
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Growth Hormone (Somatropin)Also: Saizen 8mgThe 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. | Drug | 1 |
| PlaceboAlso: Saizen 8mg placeboSame as for Growth hormone group | Drug | 2 |
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
4 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2010.
- University "Federico II", NaplesLink provided by the sponsor.
- Policlinico "Federico II", NaplesLink provided by the sponsor.
- AIFALink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alessandro Filla, MD principal investigator
University "Federico II", Naples
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Growth Hormone (Somatropin) in people with Amyotrophic Lateral Sclerosis, run by Federico II University at 2 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 May 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2010.