Amyotrophic Lateral SclerosisNCT00718393
Compassionate Use of Ceftriaxone in Patients With Amyotrophic Lateral Sclerosis (ALS)
An observational study of Amyotrophic Lateral Sclerosis and Cerebrospinal Fluid, run by Drexel University College of Medicine.
Trial summary
Amyotrophic lateral sclerosis is a uniformly progressive and fatal neurodegenerative disorder for which there is no known cure. In a novel attempt to widen the search for potential therapeutic agents, a NINDS- led cooperative group performed an in-vitro screening program of 1040 FDA approved drugs in over 28 assays… Read more about this study
- Who is it for?
- Ages 20 to 85 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid, Neurodegenerative Disease
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- Jun 2007to Oct 2015
- Phase
- Observational
- 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 20 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: ALS clinic patients at MDA/ALS Center of Hope.
Who may be able to join
- Diagnosis of definite or probable ALS by the El Escorial Criteria
- Participants must be medically able to undergo the study procedures
- Subjects must have a registered nurse who can administer the drug daily and also inspect the IV site
- Not Pregnant
- Willing to pay for the cost of drug, administration, and safety testing
- Able to give informed consent
Who cannot join
- Patients who are not diagnosed with ALS by a physician
- Patients unable to give informed consent
- Patients who have a history of sensitivity to cephalosporin or penicillin antibiotics (such as Ancef, Keflex, Ceclor, Ceftin, Lorabid, Suprax, or Fortaz).
About this study
Official title: Compassionate Use of Ceftriaxone in Patients With ALS
Amyotrophic lateral sclerosis is a uniformly progressive and fatal neurodegenerative disorder for which there is no known cure. In a novel attempt to widen the search for potential therapeutic agents, a NINDS- led cooperative group performed an in-vitro screening program of 1040 FDA approved drugs in over 28 assays relevant to various neurodegenerative disorders. Several cephalosporins showed hits in ALS relevant assays. Efficacy was noted in models suggesting increased expression of the astrocytic glutamate transporter, EAAT2, as well as models of superoxide dismutase mediated toxicity. Ceftriaxone is a third generation cephalosporin with good CNS penetration, a long half-life, and was effective in both types of ALS assays. Ceftriaxone has calcium binding activity, antioxidant properties, and rescues motor neurons in culture from chronic glutamate toxicity. Since completion of the original NINDS screen, Ceftriaxone has been shown to increase by three fold EAAT2 activity in rodent brains, due to ceftriaxone's ability to increase EAAT2 promotor activation This program is for the use of ceftriaxone in ALS for compassionate care. Currently ceftriaxone is approved by the U.S. Food and Drug Administration (FDA) for treating bacterial infections but not for treating ALS. However, there is an ongoing phase I study -by NEALS Consortium and the National Institute of Health- with three cohorts -a placebo group and two groups receiving either 2 or 4 grams of ceftriaxone daily-. Unfortunately there are only a limited number of patients being enrolled and the next phase of the project will not be undertaken until next year. At this point there are ALS patients unable to participate in this Phase I trial and unlikely to be alive when the next phase of study begins. Some of these patients want to receive the drug and are willing to pay for the drug and nursing care. We are therefore requesting a compassionate use protocol for these patients who request the medication and are willing to pay for the drug and nursing care to administer it. Dr. Terry Heiman-Patterson will supervise the administration and safety monitoring including labs for renal and hepatic function as well as IV site inspection.
- Conditions
- Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid, Neurodegenerative Disease, Motor Neuron Disease
- Keywords
- Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid, Neurodegenerative Disease, Motor Neuron Disease, Autonomic Nervous System, Neurodegenerative Diseases, Movement Disorders
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Neurodegenerative Diseases, Motor Neuron Disease, Movement Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| ALS | Subjects having either definite or probable ALS by El Escorial Criteria. |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2008 (estimated)
- Study startJun 2007
- Primary completionOct 2015
- Study completionOct 2015
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.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Drexel University College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- MDA/ALS Center of Hope
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00718393
- Sponsor's protocol ID
- Internal-16964
- Status last verified
- Mar 2017
- First submitted
- 14 Jul 2008
- Last updated
- 9 Mar 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of Ceftriaxone in Patients With ALS
- ClinicalTrials.gov ID
- NCT00718393
- Acronym / sponsor ID
- Internal-16964
- Conditions
- Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid, Neurodegenerative Disease, Motor Neuron Disease
- Keywords
- Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid, Neurodegenerative Disease, Motor Neuron Disease, Autonomic Nervous System, Neurodegenerative Diseases, Movement Disorders
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Drexel University College of MedicineUniversity, hospital or other
- Collaborators
- MDA/ALS Center of Hope
- Study start
- Jun 2007
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 18 Jul 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.
Amyotrophic lateral sclerosis is a uniformly progressive and fatal neurodegenerative disorder for which there is no known cure. In a novel attempt to widen the search for potential therapeutic agents, a NINDS- led cooperative group performed an in-vitro screening program of 1040 FDA approved drugs in over 28 assays relevant to various neurodegenerative disorders. Several cephalosporins showed hits in ALS relevant assays. Efficacy was noted in models suggesting increased expression of the astrocytic glutamate transporter, EAAT2, as well as models of superoxide dismutase mediated toxicity. Ceftriaxone is a third generation cephalosporin with good CNS penetration, a long half-life, and was effective in both types of ALS assays. Ceftriaxone has calcium binding activity, antioxidant properties, and rescues motor neurons in culture from chronic glutamate toxicity. Since completion of the original NINDS screen, Ceftriaxone has been shown to increase by three fold EAAT2 activity in rodent brains, due to ceftriaxone's ability to increase EAAT2 promotor activation This program is for the use of ceftriaxone in ALS for compassionate care. Currently ceftriaxone is approved by the U.S. Food and Drug Administration (FDA) for treating bacterial infections but not for treating ALS. However, there is an ongoing phase I study -by NEALS Consortium and the National Institute of Health- with three cohorts -a placebo group and two groups receiving either 2 or 4 grams of ceftriaxone daily-. Unfortunately there are only a limited number of patients being enrolled and the next phase of the project will not be undertaken until next year. At this point there are ALS patients unable to participate in this Phase I trial and unlikely to be alive when the next phase of study begins. Some of these patients want to receive the drug and are willing to pay for the drug and nursing care. We are therefore requesting a compassionate use protocol for these patients who request the medication and are willing to pay for the drug and nursing care to administer it. Dr. Terry Heiman-Patterson will supervise the administration and safety monitoring including labs for renal and hepatic function as well as IV site inspection.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALS | – | –Subjects having either definite or probable ALS by El Escorial Criteria. |
Who may qualify
- Ages
- 20 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- ALS clinic patients at MDA/ALS Center of Hope.
Key inclusion criteria (6 listed)
- Diagnosis of definite or probable ALS by the El Escorial Criteria
- Participants must be medically able to undergo the study procedures
- Subjects must have a registered nurse who can administer the drug daily and also inspect the IV site
- Not Pregnant
- Willing to pay for the cost of drug, administration, and safety testing
Key exclusion criteria (3 listed)
- Patients who are not diagnosed with ALS by a physician
- Patients unable to give informed consent
- Patients who have a history of sensitivity to cephalosporin or penicillin antibiotics (such as Ancef, Keflex, Ceclor, Ceftin, Lorabid, Suprax, or Fortaz).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
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 9 Mar 2017.
- Individual participant dataSharing planned: No
Related trials for Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Cerebrospinal Fluid, run by Drexel University College of Medicine.
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 6 inclusion and 3 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 9 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
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.
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 9 Mar 2017.