Chronic Inflammatory Demyelinating PolyneuropathyPhase 2NCT01236456
High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy
A Phase 2 study testing Cyclophosphamide in people with Chronic Inflammatory Demyelinating Polyneuropathy, run by Stony Brook University.
Trial summary
The primary endpoint of this study is to determine what percentage of patients receiving high-dose Cyclophosphamide may experience a halt in the worsening of their disease or experience improvement of their disease and for how long the benefit may last. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Chronic Inflammatory Demyelinating Polyneuropathy
- What is being tested?
- Cyclophosphamide
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2003to Nov 2006
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of CIDP according to the American Academy of Neurology clinical and electrophysiologic criteria
- Age >18 but < 75 years
- Modified Rankin Scale score of >3 after two standard treatment regimens
- Patient must have a left ventricular ejection fraction of >45%
- Serum Creatinine <3mg/dL
- Willingness to participate in a clinical trial
Who cannot join
- Patients who are preterminal or moribund
- Patients with active malignancies
- Patients with chromosomal abnormalities or peripheral blood counts suggestive of myelodysplastic syndrome
- Patients with active bacterial or fungal infections requiring oral or intravenous antimicrobials are not eligible until resolution of the infection
- Pregnant women and breast-feeding women
About this study
Official title: A Phase II Trial of High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy
The primary endpoint of this study is to determine what percentage of patients receiving high-dose Cyclophosphamide may experience a halt in the worsening of their disease or experience improvement of their disease and for how long the benefit may last.
Read the detailed description
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a common and under-recognized peripheral neuropathy that is thought to be immune-mediated. Randomized, placebo controlled clinical trials in CIDP demonstrate benefit from treatment with corticosteroids, plasmapheresis, and IV Ig. However, not all patients respond to these therapies. IV cyclophosphamide, cyclosporine, interferons, total lymphoid irradiation, and mycophenolate mofetil have been proposed as appropriate therapies for refractory patients.
Patients with CIDP often respond to immune-modulating treatment. However, the high rate of relapse and treatment-related side effects result in poor long-term outcomes for many patients. CIDP is assumed to be an autoimmune disease, but the pathogenesis is poorly understood. T cell infiltrates are predominantly CD8, suggesting a T cell mediated process. There is not, however, restricted T cell receptor Vbeta utilization seen in sural nerve biopsies. Immunoglobulin and complement deposits noted on the myelin sheaths support an antibody-mediated process. Antibodies to the P0 myelin protein are seen in a minority of patients. High-dose cyclophosphamide is believed to eradicate both B and T lymphocytes. This therapy does not damage hematopoietic stem cells, which allows for rapid white cell recovery without stem cell rescue.
- Being tested
- Cyclophosphamide
- Keywords
- chronic inflammatory demyelinating polyneuropathy, cyclophosphamide, autoimmune
- Medical subject headings
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint of this study is to evaluate the response rate of CIDP patients as determined by functional score, change in Summated compound motor action potential and strength, after high-dose cyclophosphamide therapy.
Secondary outcomes (1)
The secondary endpoint of this study is to determine remission duration.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2010 (estimated)
- Study startOct 2003
- Study completionNov 2006 (estimated)
Why it stopped: I am relocating to Johns Hopkins Medical Center
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Chronic Inflammatory Demyelinating PolyneuropathyStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stony Brook University
- Sponsor type
- University, hospital or other
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01236456
- Sponsor's protocol ID
- 65865
- Status last verified
- Nov 2021
- First submitted
- 5 Nov 2010
- Last updated
- 9 Nov 2021
- Results posted
- No
Publications
Gladstone DE, Prestrud AA, Brannagan TH 3rd. High-dose cyclophosphamide results in long-term disease remission with restoration of a normal quality of life in patients with severe refractory chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2005 Mar;10(1):11-6. doi: 10.1111/j.1085-9489.2005.10104.x.
PubMed 15703014Brannagan TH 3rd, Pradhan A, Heiman-Patterson T, Winkelman AC, Styler MJ, Topolsky DL, Crilley PA, Schwartzman RJ, Brodsky I, Gladstone DE. High-dose cyclophosphamide without stem-cell rescue for refractory CIDP. Neurology. 2002 Jun 25;58(12):1856-8. doi: 10.1212/wnl.58.12.1856.
PubMed 12084892
Study at a glance
From the registry record, last updated by the sponsor on 9 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy
- ClinicalTrials.gov ID
- NCT01236456
- Acronym / sponsor ID
- 65865
- Keywords
- chronic inflammatory demyelinating polyneuropathy, cyclophosphamide, autoimmune
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: I am relocating to Johns Hopkins Medical Center
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Stony Brook UniversityUniversity, hospital or other
- Study start
- Oct 2003
- Study completion
- Nov 2006 (estimated)
- First posted
- 8 Nov 2010 (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.
The primary endpoint of this study is to determine what percentage of patients receiving high-dose Cyclophosphamide may experience a halt in the worsening of their disease or experience improvement of their disease and for how long the benefit may last.
Detailed description
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a common and under-recognized peripheral neuropathy that is thought to be immune-mediated. Randomized, placebo controlled clinical trials in CIDP demonstrate benefit from treatment with corticosteroids, plasmapheresis, and IV Ig. However, not all patients respond to these therapies. IV cyclophosphamide, cyclosporine, interferons, total lymphoid irradiation, and mycophenolate mofetil have been proposed as appropriate therapies for refractory patients.
Patients with CIDP often respond to immune-modulating treatment. However, the high rate of relapse and treatment-related side effects result in poor long-term outcomes for many patients. CIDP is assumed to be an autoimmune disease, but the pathogenesis is poorly understood. T cell infiltrates are predominantly CD8, suggesting a T cell mediated process. There is not, however, restricted T cell receptor Vbeta utilization seen in sural nerve biopsies. Immunoglobulin and complement deposits noted on the myelin sheaths support an antibody-mediated process. Antibodies to the P0 myelin protein are seen in a minority of patients. High-dose cyclophosphamide is believed to eradicate both B and T lymphocytes. This therapy does not damage hematopoietic stem cells, which allows for rapid white cell recovery without stem cell rescue.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of CIDP according to the American Academy of Neurology clinical and electrophysiologic criteria
- Age >18 but < 75 years
- Modified Rankin Scale score of >3 after two standard treatment regimens
- Patient must have a left ventricular ejection fraction of >45%
- Serum Creatinine <3mg/dL
Key exclusion criteria (5 listed)
- Patients who are preterminal or moribund
- Patients with active malignancies
- Patients with chromosomal abnormalities or peripheral blood counts suggestive of myelodysplastic syndrome
- Patients with active bacterial or fungal infections requiring oral or intravenous antimicrobials are not eligible until resolution of the infection
- Pregnant women and breast-feeding 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 |
|---|---|---|
| Cyclophosphamide | Drug | – |
Primary outcome
The primary endpoint of this study is to evaluate the response rate of CIDP patients as determined by functional score, change in Summated compound motor action potential and strength, after high-dose cyclophosphamide therapy.
1 secondary outcome
The secondary endpoint of this study is to determine remission duration.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Nov 2021.
- Results publication: PubMed 15703014Gladstone DE, Prestrud AA, Brannagan TH 3rd. High-dose cyclophosphamide results in long-term disease remission with restoration of a normal quality of life in patients with severe refractory chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2005 Mar;10(1):11-6. doi: 10.1111/j.1085-9489.2005.10104.x.
- Results publication: PubMed 12084892Brannagan TH 3rd, Pradhan A, Heiman-Patterson T, Winkelman AC, Styler MJ, Topolsky DL, Crilley PA, Schwartzman RJ, Brodsky I, Gladstone DE. High-dose cyclophosphamide without stem-cell rescue for refractory CIDP. Neurology. 2002 Jun 25;58(12):1856-8. doi: 10.1212/wnl.58.12.1856.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide in people with Chronic Inflammatory Demyelinating Polyneuropathy, run by Stony Brook University.
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 5 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: withdrawn, as of the sponsor's last update on 9 Nov 2021. 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.
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 was withdrawn before enrolling participants, so there are no results.
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 primary endpoint of this study is to evaluate the response rate of CIDP patients as determined by functional score, change in Summated compound motor action potential and strength, after high-dose cyclophosphamide therapy.. 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 NCT01236456, which also lists 2 linked publications.
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 Nov 2021.