Demyelinating DiseasesPhase 2NCT00001287
Intravenous Immunoglobulin (IVIg) for the Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
A Phase 2 study testing intravenous immunoglobulin (IVIg) in people with Demyelinating Diseases and Paraproteinemias, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
Chronic Inflammatory Demylinating Polyneuropathy (CIDP) is an autoimmune condition affecting the nervous system. Researchers believe the immune system begins attacking the cells covering nerves called myelin. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Demyelinating Diseases, Paraproteinemias
- What is being tested?
- intravenous immunoglobulin (IVIg)
- Type of treatment
- Injection or infusion
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 1990to Jan 2001
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Selected patients should have CIDP with or without an associated monoclonal gammopathy.
Subjects should have clinical evidence of peripheral neuropathy with muscle weakness and sensory deficit.
Subjects should have evidence of clinical, histological or family history of another neuromuscular illness.
Subjects should have elevation of CSF protein during the course of the disease.
Subjects should have demyelination by nerve conduction study and/or nerve biopsy.
Suitable candidates for IVIg should be patients with active, bonefide CIDP who:
- have been treated with steroids but had: a) no response or incomplete response (as defined by continued muscle weakness) to high-dose therapy or b) a good response to steroids but inability to taper the dose without a flare of disease activity or c) unacceptable steroid side effects such as gastrointestinal hemorrhages, osteonecrosis, hyperglycemia, extreme weight gain etc. or
- have been additionally treated with one of the other immunosuppressive agents considered effective in some CIDP patients, such as azathioprine, chlorambucil, cyclophosphamide, cyclosporine or plasmapheresis but without benefit or with unacceptable side effects that had necessitated their discontinuation.
Subjects should not be pregnant or nursing.
Subjects should not be critically ill such as those requiring intravenous pressors for maintenance of cardiac output, patients with unstable respiratory insufficiency and patients with such severe muscle weakness requiring help for basic self care (Karnofsky performance scale less than 50).
No subjects below 18 years of age.
Patients should not have severe renal or hepatic disease and severe COPD or coronary artery disease.
Patients should not be allergic to IVIg or have a known IgA deficiency.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, 20892
About this study
Official title: The Efficacy of High-Dose Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Chronic Inflammatory Demylinating Polyneuropathy (CIDP) is an autoimmune condition affecting the nervous system. Researchers believe the immune system begins attacking the cells covering nerves called myelin. The destruction of myelin causes muscle weakness, loss of sensation, abnormal levels of protein in the fluid surrounding the brain (CSF), and slowing of the nervous system. The disease progresses slowly and disables patients suffering from it.
CIDP is treated with steroids, plasmapheresis, and immunosuppressive drugs. Many patients initially respond to these treatments, but develop resistance to the therapy or experience side effects causing the treatments to be stopped.
Read more
Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with CIDP a safer and more effective alternative to standard therapies for the disease. IVIg is a drug that has been used successfully to treat other immune-related diseases of the nervous system. However, because IVIg is so expensive, researchers believe it should first be proven effective on a small group of patients.
The study will take 60 patients with CIDP and divide them into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive injection of sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement (>25%) in muscle strength.
Read the detailed description
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a slowly progressive disabling neuropathy characterized by subacute onset of muscle weakness, distal sensory deficit, elevated spinal fluid protein, and slow nerve conduction velocity with or without conduction block. A monoclonal gammopathy is at times present in the serum of some patients. Because immune-mediated mechanisms against peripheral nerve myelin are thought to be primarily responsible for the clinical manifestations of CIDP, the treatment of choice is with corticosteroids, plasmapheresis or immunosuppressive drugs. Although many patients initially respond to these agents, a large number of them become resistant or develop unacceptable side effects that necessitate their discontinuation. The need for a more effective and safe immunotherapy in CIDP patients prompted the present study using high-dose intravenous immunoglobulin (IVIg). IVIg is an immunomodulating agent which has been recently shown to be effective and safe in the treatment of a number of patients with immune-related neuromuscular diseases.
This is a double blind, randomized, placebo controlled, trial involving 60 patients, half of which will receive IVIg and the other half placebo (D5/W). Because IVIg is prohibitively expensive, a controlled trial is needed to provide convincing evidence of efficacy, and ensure that the benefit is not due to spontaneous improvement or to observer bias. The dose of IVIg is 2 GM/Kg divided into two daily doses administered monthly for six months. The drug will be considered effective if patients experience an increase of more than 25% in their baseline muscle strength. Muscle strength will be assessed with a series of objective dynamometric measurements performed before and after each monthly infusion.
- Conditions
- Demyelinating Diseases, Paraproteinemias
- Being tested
- intravenous immunoglobulin (IVIg)
- Keywords
- Intravenous High-Dose Immunoglobulin, Monoclonal Gammopathies, Peripheral Nerve Demyelination, CIDP, Chronic Inflammatory Demyelinating Polyneuropathy
- Medical subject headings
- Demyelinating Diseases, Paraproteinemias, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
intravenous immunoglobulin (IVIg)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2002 (estimated)
- Study startDec 1990
- Study completionJan 2001
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Demyelinating DiseasesStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The 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
- Enrolment
- 60 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001287
- Sponsor's protocol ID
- 910039
- Other IDs
- 91-N-0039
- Status last verified
- Feb 2000
- First submitted
- 3 Nov 1999
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Dalakas MC, Engel WK. Chronic relapsing (dysimmune) polyneuropathy: pathogenesis and treatment. Ann Neurol. 1981;9 Suppl:134-45. doi: 10.1002/ana.410090719.
PubMed 7224612Cook D, Dalakas M, Galdi A, Biondi D, Porter H. High-dose intravenous immunoglobulin in the treatment of demyelinating neuropathy associated with monoclonal gammopathy. Neurology. 1990 Feb;40(2):212-4. doi: 10.1212/wnl.40.2.212.
PubMed 2153942Basta M, Dalakas MC. High-dose intravenous immunoglobulin exerts its beneficial effect in patients with dermatomyositis by blocking endomysial deposition of activated complement fragments. J Clin Invest. 1994 Nov;94(5):1729-35. doi: 10.1172/JCI117520.
PubMed 7962520
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy of High-Dose Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- ClinicalTrials.gov ID
- NCT00001287
- Acronym / sponsor ID
- 910039
- Conditions
- Demyelinating Diseases, Paraproteinemias
- Keywords
- Intravenous High-Dose Immunoglobulin, Monoclonal Gammopathies, Peripheral Nerve Demyelination, CIDP, Chronic Inflammatory Demyelinating Polyneuropathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- Dec 1990
- Study completion
- Jan 2001
- First posted
- 10 Dec 2002 (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.
Chronic Inflammatory Demylinating Polyneuropathy (CIDP) is an autoimmune condition affecting the nervous system. Researchers believe the immune system begins attacking the cells covering nerves called myelin. The destruction of myelin causes muscle weakness, loss of sensation, abnormal levels of protein in the fluid surrounding the brain (CSF), and slowing of the nervous system. The disease progresses slowly and disables patients suffering from it.
CIDP is treated with steroids, plasmapheresis, and immunosuppressive drugs. Many patients initially respond to these treatments, but develop resistance to the therapy or experience side effects causing the treatments to be stopped.
Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with CIDP a safer and more effective alternative to standard therapies for the disease. IVIg is a drug that has been used successfully to treat other immune-related diseases of the nervous system. However, because IVIg is so expensive, researchers believe it should first be proven effective on a small group of patients.
The study will take 60 patients with CIDP and divide them into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive injection of sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement (>25%) in muscle strength.
Detailed description
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a slowly progressive disabling neuropathy characterized by subacute onset of muscle weakness, distal sensory deficit, elevated spinal fluid protein, and slow nerve conduction velocity with or without conduction block. A monoclonal gammopathy is at times present in the serum of some patients. Because immune-mediated mechanisms against peripheral nerve myelin are thought to be primarily responsible for the clinical manifestations of CIDP, the treatment of choice is with corticosteroids, plasmapheresis or immunosuppressive drugs. Although many patients initially respond to these agents, a large number of them become resistant or develop unacceptable side effects that necessitate their discontinuation. The need for a more effective and safe immunotherapy in CIDP patients prompted the present study using high-dose intravenous immunoglobulin (IVIg). IVIg is an immunomodulating agent which has been recently shown to be effective and safe in the treatment of a number of patients with immune-related neuromuscular diseases.
This is a double blind, randomized, placebo controlled, trial involving 60 patients, half of which will receive IVIg and the other half placebo (D5/W). Because IVIg is prohibitively expensive, a controlled trial is needed to provide convincing evidence of efficacy, and ensure that the benefit is not due to spontaneous improvement or to observer bias. The dose of IVIg is 2 GM/Kg divided into two daily doses administered monthly for six months. The drug will be considered effective if patients experience an increase of more than 25% in their baseline muscle strength. Muscle strength will be assessed with a series of objective dynamometric measurements performed before and after each monthly infusion.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| intravenous immunoglobulin (IVIg) | Drug | – |
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 4 Mar 2008.
- Background: PubMed 7224612Dalakas MC, Engel WK. Chronic relapsing (dysimmune) polyneuropathy: pathogenesis and treatment. Ann Neurol. 1981;9 Suppl:134-45. doi: 10.1002/ana.410090719.
- Background: PubMed 2153942Cook D, Dalakas M, Galdi A, Biondi D, Porter H. High-dose intravenous immunoglobulin in the treatment of demyelinating neuropathy associated with monoclonal gammopathy. Neurology. 1990 Feb;40(2):212-4. doi: 10.1212/wnl.40.2.212.
- Background: PubMed 7962520Basta M, Dalakas MC. High-dose intravenous immunoglobulin exerts its beneficial effect in patients with dermatomyositis by blocking endomysial deposition of activated complement fragments. J Clin Invest. 1994 Nov;94(5):1729-35. doi: 10.1172/JCI117520.
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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.
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 intravenous immunoglobulin (IVIg) in people with Demyelinating Diseases and Paraproteinemias, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2008. 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 United States. 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. 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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001287, which also lists 3 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 4 Mar 2008.