Amyotrophic Lateral SclerosisPhase 2NCT00397423
G-CSF Treatment for Amyotrophic Lateral Sclerosis: A RCT Study Assessing Clinical Response
A Phase 2 study testing Granulocyte Colony Stimulating Factor and NS in people with Amyotrophic Lateral Sclerosis, run by Peking University at 1 site in China.
Trial summary
This study will examine the effectiveness of G-CSF in treating patients with amyotrophic lateral sclerosis (ALS) - a fatal neurological degenerative disease that causes adult-onset, progressive motor neurons loss in the spinal cord, brain stem and motor cortex. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Granulocyte Colony Stimulating Factor and NS
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- Dec 2006to Aug 2007
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All subjects must have a verifiable diagnosis of ALS of 0.5 to 2 years' duration. The diagnosis must be supported by the Revised Criteria of the World Federation of Neurology. The grades of diagnosis must be clinically definite ALS or clinically probable ALS.
- All subjects must be over age 18 and below 65.
- The ALS is mildly to moderate based on ALS Health State Scale.
- Electrophysiological features show CMAP amplitude of motor nerve normal or mild declining.
- Serum creatine kinase is normal or mild upper, less than 500U/L.
Who cannot join
- If anyone of the above eligibility requirements is not met
- Use of any other investigational agent within 30 days beginning the treatment phase of this study
- Severe cardiac, pulmonary, hepatic or/and hematic disease
- HIV positivity or signs and symptoms consistent with HIV infection
- Pregnant or nursing women
- History of cancer with less than 5 years documentation of a disease-free state
Show all 10Show fewer
- History of anaphylactic reaction or hypersensitivity to G-CSF or proteins derived from E.coli
- Alcohol or drug abuse in recent 1 year
- Can't understand or obey the rules of treatment
- Blood donor in recent 30 days
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Granulocyte-Colony Stimulating Factor Treatment for Amyotrophic Lateral Sclerosis: A Randomized Control Trial Study Assessing Clinical Response
This study will examine the effectiveness of G-CSF in treating patients with amyotrophic lateral sclerosis (ALS) - a fatal neurological degenerative disease that causes adult-onset, progressive motor neurons loss in the spinal cord, brain stem and motor cortex. Patients develop progressive wasting and weakness of both upper and lower limbs, bulbar and respiratory muscles. Usually death from respiratory failure typically is within 3-5 years of diagnosis. Although there are various treatments for ALS, riluzole is the only approved treatment to delay the disease progression. G-CSF is an approved drug that is used to increase white blood cell counts. Moreover, G-CSF and its receptor are expressed by neurons. It acts anti-apoptosis by activating several protective pathways, stimulates neuronal differentiation of adult neural stem cells in the brain, and improves long-term recovery. G-CSF is a novel neurotrophic factor, and a highly attractive candidate for the treatment of neurodegenerative conditions such as ALS.
Patients 18 to 65 years of age who have had mild to moderately severe ALS for 0.5 to 2 years of duration may be eligible for this study. Candidates will be screened with a medical history and possible review of medical records, physical examination, blood test, urine and stool analyses, electrocardiogram, electrophysiological examination, neurological imaging and, for women, a pregnancy test.
Participants will have drug therapy according to randomized number. One group receives G-CSF while other group receives placebo. All of the participants receive riluzole treatment. For the procedure, patients are given a medication to lessen anxiety and any discomfort. Patients receive drug injections every 3 months for 5 days. The G-CSF dosage is 5μg/kg/day. Physical examination and interview, Appel ALS scale and ALS-Functional Rating Scale will be done in 14, 28 days and 3, 6, 9, 12 months. Electrophysiological examination will be tested per 3 months. Blood samples will be collected on treat 5, 14, 28 days and 3, 6, 9, 12 months.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Granulocyte Colony Stimulating Factor, NS
- Keywords
- Amyotrophic Lateral Sclerosis, Granulocyte Colony Stimulating Factor, Randomized Control Trial Study
- 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.
Granulocyte Colony Stimulating FactorDrug
Granulocyte Colony Stimulating Factor,5ug/kg/day iH Qd*5 days,3 months interval for 4 times
NSDrug
NS 1ml iH Qd*5 days, 3 months interval for 4 times
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | 1,G-CSF,intervention Receives: Drug: Granulocyte Colony Stimulating Factor |
| 2 | Placebo comparator | 2,NS,intervention Receives: Drug: NS |
What is being measured
The primary outcome is the main question the study is designed to answer.
the mean rate of decline of ALS-FRS score
Measured: 12 months
Secondary outcomes (1)
the mean rate decline of the AARS score
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 posted9 Nov 2006 (estimated)
- Study startDec 2006
- Study completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- 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
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking University
- Sponsor type
- University, hospital or other
- Investigators
- Dongsheng Fan, MD, Study chair, Peking University Third Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00397423
- Sponsor's protocol ID
- R01-123456-1
- Status last verified
- Nov 2007
- First submitted
- 8 Nov 2006
- Last updated
- 28 Nov 2007
- Results posted
- No
Publications
Fan DS, Zhang J, Deng M, Kang DX, Zheng JY, Xu YS, Lu M, Fu Y, Shen Y, Zhang YS, Zhang HG. [Basic and clinical researches on amyotrophic lateral sclerosis/motor neuron disease]. Beijing Da Xue Xue Bao Yi Xue Ban. 2009 Jun 18;41(3):279-81. Chinese.
PubMed 19727208
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Granulocyte-Colony Stimulating Factor Treatment for Amyotrophic Lateral Sclerosis: A Randomized Control Trial Study Assessing Clinical Response
- ClinicalTrials.gov ID
- NCT00397423
- Acronym / sponsor ID
- R01-123456-1
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, Granulocyte Colony Stimulating Factor, Randomized Control Trial Study
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Peking UniversityUniversity, hospital or other
- Study start
- Dec 2006
- Study completion
- Aug 2007 (actual)
- First posted
- 9 Nov 2006 (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.
This study will examine the effectiveness of G-CSF in treating patients with amyotrophic lateral sclerosis (ALS) - a fatal neurological degenerative disease that causes adult-onset, progressive motor neurons loss in the spinal cord, brain stem and motor cortex. Patients develop progressive wasting and weakness of both upper and lower limbs, bulbar and respiratory muscles. Usually death from respiratory failure typically is within 3-5 years of diagnosis. Although there are various treatments for ALS, riluzole is the only approved treatment to delay the disease progression. G-CSF is an approved drug that is used to increase white blood cell counts. Moreover, G-CSF and its receptor are expressed by neurons. It acts anti-apoptosis by activating several protective pathways, stimulates neuronal differentiation of adult neural stem cells in the brain, and improves long-term recovery. G-CSF is a novel neurotrophic factor, and a highly attractive candidate for the treatment of neurodegenerative conditions such as ALS.
Patients 18 to 65 years of age who have had mild to moderately severe ALS for 0.5 to 2 years of duration may be eligible for this study. Candidates will be screened with a medical history and possible review of medical records, physical examination, blood test, urine and stool analyses, electrocardiogram, electrophysiological examination, neurological imaging and, for women, a pregnancy test.
Participants will have drug therapy according to randomized number. One group receives G-CSF while other group receives placebo. All of the participants receive riluzole treatment. For the procedure, patients are given a medication to lessen anxiety and any discomfort. Patients receive drug injections every 3 months for 5 days. The G-CSF dosage is 5μg/kg/day. Physical examination and interview, Appel ALS scale and ALS-Functional Rating Scale will be done in 14, 28 days and 3, 6, 9, 12 months. Electrophysiological examination will be tested per 3 months. Blood samples will be collected on treat 5, 14, 28 days and 3, 6, 9, 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Drug: Granulocyte Colony Stimulating Factor1,G-CSF,intervention |
| 2 | Placebo comparator | Drug: NS2,NS,intervention |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- All subjects must have a verifiable diagnosis of ALS of 0.5 to 2 years' duration. The diagnosis must be supported by the Revised Criteria of the World Federation of Neurology. The grades of diagnosis must be clinically definite ALS or clinically probable ALS.
- All subjects must be over age 18 and below 65.
- The ALS is mildly to moderate based on ALS Health State Scale.
- Electrophysiological features show CMAP amplitude of motor nerve normal or mild declining.
- Serum creatine kinase is normal or mild upper, less than 500U/L.
Key exclusion criteria (10 listed)
- If anyone of the above eligibility requirements is not met
- Use of any other investigational agent within 30 days beginning the treatment phase of this study
- Severe cardiac, pulmonary, hepatic or/and hematic disease
- HIV positivity or signs and symptoms consistent with HIV infection
- Pregnant or nursing women
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 |
|---|---|---|
| Granulocyte Colony Stimulating FactorGranulocyte Colony Stimulating Factor,5ug/kg/day iH Qd*5 days,3 months interval for 4 times | Drug | 1 |
| NSNS 1ml iH Qd*5 days, 3 months interval for 4 times | Drug | 2 |
Primary outcome
the mean rate of decline of ALS-FRS score
Time frame: 12 months
1 secondary outcome
the mean rate decline of the AARS score
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 28 Nov 2007.
- Derived from this study: PubMed 19727208Fan DS, Zhang J, Deng M, Kang DX, Zheng JY, Xu YS, Lu M, Fu Y, Shen Y, Zhang YS, Zhang HG. [Basic and clinical researches on amyotrophic lateral sclerosis/motor neuron disease]. Beijing Da Xue Xue Bao Yi Xue Ban. 2009 Jun 18;41(3):279-81. Chinese.
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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.
Dongsheng Fan, MD study chair
Peking University Third Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Granulocyte Colony Stimulating Factor and NS in people with Amyotrophic Lateral Sclerosis, run by Peking University at 1 site in China.
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 28 Nov 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
the mean rate of decline of ALS-FRS score (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00397423, which also lists 1 linked publication.
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 28 Nov 2007.