TuberculosisPhase 2/3NCT04620772
Cyclophosphamide in the Treatment of Refractory Proliferative Arachnoiditis in CNS Tuberculosis
A Phase 2/3 study testing Cyclophosphamide injection in people with Tuberculosis and Tubercular Meningitis, run by All India Institute of Medical Sciences.
Trial summary
Tubercular meningitis occurs in around 10% of those with extrapulmonary tuberculosis and is a major cause of mortality and morbidity. Inspite of effective Anti-tubercular drugs, still around 30% of patients develop complications due to arachnoiditis such as spinal tubercular radiculomyelitis, optico-chiasmatic… Read more about this study
- Who is it for?
- Ages 14 to 60 years
- Conditions the study is for
- Tuberculosis, Tubercular Meningitis, Arachnoiditis; Tuberculous (Etiology)
- What is being tested?
- Cyclophosphamide injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2021to 31 Dec 2024
- Phase
- Phase 2/3
- 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 14 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients attending Neurology/Pulmonary Medicine/Medicine/Geriatric Medicine OPD/admitted in respective wards with proliferative tubercular arachnoiditis refractory to corticosteroids and standard Anti-tubercular drugs for CNS tuberculosis
- Atleast 14 years of age of all sexes
- Not more than 60 years of age at time of enrolment
- Patient was started on ATT for tubercular meningitis and had clearcut clinical improvement with resolution of fever/constitutional symptoms AND improvement in headache, vomiting and sensorium for atleast 10 days following which there is deterioration again due to arachnoiditis
- Developed paraparesis/quadriparesis/sphincter dysfunction due to spinal radiculomyelitis or vision loss due to due to optico-chiasmatic arachnoiditis with imaging evidence of arachnoiditis
- Has received standard ATT for atleast 3 months with adequate dose and compliance
Show all 14Show fewer
- Received corticosteroids for treatment of arachnoiditis and deemed to be refractory to corticosteroids by the primary physician treating the patient
- MRI brain and spine are suggestive of Arachnoiditis
- CSF GeneXpert/Line Probe assay/cultures are not suggestive of drug resistant tuberculosis
- Reasonable clinical certainty OR allied investigations such as CECT chest/abdomen/PET CT ruling out drug resistant tuberculosis
- Other relevant investigations like CSF analysis not suggestive of alternative diagnosis such as cysticercal/ cryptococcal/other fungal infections/other causes of chronic meningitis such as brucella/ nocardia/ syphilis/recurrent viral meningitis/ carcinomatous/ lymphomatous meningitis or non infective causes such as sarcoidoisis/sub-arachnoid hemorrhage etc.
- Willing to undergo periodic assessment clinically and with MRI.
- Ready to provide consent for cyclophosphamide therapy
- Willing to adhere to protocol and comply with follow up visits
Who cannot join
- Not willing to provide consent
- Not willing to adhere to protocol
- Developed significant drug induced liver dysfunction so that patient is not being given Rifampicin, INH or pyrazinamide and is on modified ATT including quinolones, ethambutol and aminoglycosides or second line drugs only in the absence of Rifampicin and INH
- Drug resistant tubeculosis
- Men and Women of childbearing potential who are not using adequate contraception or women who are pregnant and lactating
- Patients who are on immunosuppressants such as cyclophosphamide/ azathioprine/ methotrexate/MMF/ calcineurin inhibitors for autoimmune conditions/post transplantation or chemotherapy for any systemic malignancy
Show all 16Show fewer
- HBsAg, HIV serology and anti HCV positive
- Having life threatening infections such as pneumonia/urosepsis
- Patients who have developed large artery strokes with significant brain parenchymal damage
- Patients with expected life expectancy less than 1 year due to primary disease or comorbidity based on clinical prediction scores for specific disease
- Patients with systemic malignancy within the last 5 years
- Known allergy to cyclophosphamide or its preservatives/excipients
- Receiving cyclophosphamide for any indication in the last 12 weeks
- Gross hematuria prior to enrolment to the study/USG features of hemorrhagic cystitis
- Cytopenias Hct <25%, TLC<4000/mm3 or Platelet count <1,20,000/mm3 at the time of enrolment
- Alanine amino transferase (ALT) > 3 upper limit of normal at time of enrolment
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Efficacy and Safety of Cyclophosphamide in the Treatment of Refractory Proliferative Arachnoiditis in Central Nervous System Tuberculosis- A Randomized Double Blinded Placebo Controlled Trial
Tubercular meningitis occurs in around 10% of those with extrapulmonary tuberculosis and is a major cause of mortality and morbidity. Inspite of effective Anti-tubercular drugs, still around 30% of patients develop complications due to arachnoiditis such as spinal tubercular radiculomyelitis, optico-chiasmatic arachnoiditis, development of new tuberculomas after starting therapy etc. which are probably immune mediated inflammatory responses due to paradoxical reaction to ATT.
The management of arachnoiditis is far from satisfactory. High dose methylprednisolone, intrathecal hyaluronic acid, thalidomide have been tried in small case series and case reports. However, the results have not been satisfactory.
There are two published reports of cyclophosphamide usage in TBM related vasculitis and stroke The investigators tried cyclophosphamide in four patients after consent, and found remarkable improvement in all of them. (Under peer review) In order to test this hypothesis, a randomized controlled trial is needed.
Read the detailed description
Tubercular arachnoiditis occurring as a late complication of TBM is due to immune mediated reactions and is usually treated with high doses of corticosteroids. In the investigators' experience patients have received corticosteroids upto a duration of 20-28 months. In such cases, if the patient is refractory to corticosteroids, it is logical to step up to cyclophosphamide rather than continuing to give high dose steroids only similar to other immune mediated conditions such as CIDP, systemic and CNS vasulitis, Lupus nephritis etc.
Cyclophosphamide has been used most widely in the management of Lupus nephritis and maximum experience with the drug has been in this indication. So the dosage and duration of administration has been adapted from the induction phase dosing schedule of the ACR guidelines for the management of Lupus nephritis. The guidelines recommend 500 to 1000 mg/m2 BSA of cyclophosphamide. So the investigators decided to use 750 mg/m2 of the drug rounded off to the higher 50 mg.
Pathogenesis of tubercular arachnoiditis and Possible mechanism of cyclophosphamide Tubercular arachnoidiitis is a delayed complication in most cases due to inflammation in the optochiasmatic, spinal and other cranial nerves leading to neurologic deficits. In such situations, it is seen that there is an initial improvement in clinical symptoms followed by deterioration due to enlargement of lesions or appearance of new lesions, especially in patients with extrapulmonary tuberculosis. These new lesions are probably due to immune mediated mechanisms-it has been seen that in more than 95% of cases are drug sensitive and corticosteroids are the cornerstone of management of such patients. The complete pathogenesis of immune medicated tissue injury in tuberculosis has not been elucidated. Various studies have shown that there is an excess of pro-inflammatory cytokines and chemokines including TNF-alpha and interleukins that lead to recruitment of inflammatory cells and an exhuberant immune response in patients who develop these complications as compared to those who don't. There have been studies which have shown genetic polymorphisms in the genes encoding pro-inflammatory and anti-inflammatory cytokines-leading to difference in response to corticosteroids like LTA4 (Leukotriene- A4 hydrolase) gene. It is also well known that various immune mediated diseses such as SLE, multiple sclerosis etc are triggered by viruses and other infections. It is routine practice to treat acute bacterial meninigitis and tubercular meningitis with dexamethasone, which is again a steroid and immunosuppressant along with specific antibiotics or anti-tubercular therapy respectively based on evidence from systematic reviews and meta-analysis. Neurocysticercosis is treated with corticosteroids and other immunosuppressant medications such as methotrexate as recommended in guidelines, adalimumab and eternacept used as steroid sparing agents. Various other immune mediated complications of acute and chronic infections such as dengue myocarditis, ADEM etc are also treated with immunosuppression including corticosteroids. Lepra reactions in Leprosy are also treated using steroids, thalidomide, methotrexate, azathioprine, cyclophosphamide, cyclosporine etc.
In recent times, the corticosteroid- Dexamethasone has been shown to have mortality benefit in patients with moderate to severe COVID, proven in a randomized controlled trial. Simliarly, even though there are no RCT data, Tocilizumab, siltuximab and other immunosuppressant medications have been approved for emergency use based on their efficacy shown in large case series in COVID-19.
Justification for the use of cyclophosphamide this study:
Cyclophosphamide is a potent immunosuppressant agent. It is used in Lupus and other immune mediated conditions and has been shown to be effective in randomized controlled trials in Lupus nephritis, granulomatous polyangiiitis etc. Though it is widely used off label in various inflammatory immune mediated neurologic conditions such as CNS vasculitis, CIDP, multiple sclerosis, autoimmune encephalitis, refractory myasthenia gravis etc, the evidence for this is basically derived from the rheumatology and nephrology literature. It has to be emphasized that all the data for immunosuppression with azathioprine, cyclophosphamide, mycophenolate mofetil, methotrexate, calcineurin inhibitors in various immune mediated conditions have been derived from renal transplant/solid organ transplant cohorts, lupus nephritis cohorts, systemic vasculitis cohorts and inflammatory bowel disease cohorts. The various guidelines issued by the professional bodies for these conditions form the principles of treatment of rare conditions such as neuroimmunologic diseases. The major adverse effects of concern in patients on Cyclophosphamide is the occurrence of life threatening infections such as tuberculosis. It has been seen that patients on high dose corticosteroids and cyclophosphamide are at higher risk of infection than those on steroids alone or on neither drug.Though the risk of TB increases with administration of immunosuppressants, the mainstay of management is early diagnosis and starting appropriate therapy and not cessation of immunosuppression. In the context of SLE the occurrence of infection is also dependent on various other factors such as disease activity, previous exposure to tuberculosis etc. The Americal college of rheumatology guidelines do not say anything on the course of action in case patients develop infections. There are no professional guidelines for the management of infections in patients on cyclophosphamide. There are no RCTs on the most appropriate form of ATT regimens or the modification of immunosuppression in these conditions. There are various case series and case reports who have been systematically studied infections and their management including the large prospective EuroLupus cohort. None of these authors advice complete cessation of immunosuppression in patients who develop infections including tuberculosis. The guidelines for management of tuberculosis such as British thoracic society guidelines for the management of TB in CKD,do not suggest cessation of immunosuppression in post renal transplant patients. The guidelines clearly mention that TB in patients with CKD have to be treated in same lines of immunocompetent patients, and the anti-tubercular drugs may need to be given in renal modified doses. In case of tuberculosis risk, it has been shown by various long term follow up studies that a considerable proportion of patients with latent TB infections progress to develop active TB post transplantation or after treatment with immunosuppressants or biologic agents such as anti TNF therapy. In such circumstances, the guidelines suggest not dropping immunosuppression, but treatment of latent TB or active TB. The guidelines for the management of active TB in solid organ transplantation suggests early diagnosis and treatment of tuberculosis along with increasing the dose of immunosuppressants such as corticosteroids, cyclosporine or tacrolimus as the ATT regimen containing rifamycins tend to induce the metabolism of these immunosuppression and trough levels of calcineurin inhibitors have to be maintained by increasing the dose of cyclosporine or tacrolimus by upto 4-5 times.
These recommendations are mainly based on a large multicentre cohort of more than 2000 patients derived from 187 publications who had received solid organ transplantation and developed tuberculosis, where the dose of immunosuppressant drugs had been increased to maintain adequate blood levels to avoid rejection of the organ graft. Similar recommendations have been made by the TBNET consensus statement for management of Tuberculosis in transplant recipients.
The management of immunosuppression in these cases are mainly based on expert consensus in other conditions such as rheumatoid arthritis, spondyloarthropathy etc. The TBNET consensus statement also says that in case the organ is not vital or the infection is life threatening, it may be considered to reduce the level of immunosuppression. The guidelines for the use of anti TNF-α therapy is clearer in this aspect. While the occurrence of active TB during anti TNF-α therapy warrants withholding (NOT cessation) of immunosuppression, it may be re started 2 months after ATT the if patients demonstrate a favorable response to anti-TB therapy and require early resumption of anti TNF therapy.The BTS guidelines suggest that if active TB was diagnosed prior to starting of "Anti-TNF-α therapy, treatment should not be commenced for at least 2 months after antituberculosis treatment with full compliance has begun, supervised by a thoracic physician orinfectious disease specialist, and until the drug susceptibility profile of the organism in those with positive cultures is known, as a minimum." However, if the patient develops TB while on anti TNF therapy the anti-TNF-α treatment can be continued if clinically indicated because the patient would otherwise be prevented from receiving the continued clinical benefit to their underlying disease and may have a flare up or major clinical deterioration. The rationale provided by these guidelines is that HIV positive individuals with reduced CD4 counts and clinical TB, who are even more immunosuppressed than those on anti-TNF-a treatment, respond just as well to TB treatment as those who are HIV negative. Taking that logic forward, it may be reasonable to start therapy with cyclophosphamide three months after starting standard Anti tubercular therapy in patients with tubercular meningitis who have shown clearcut improvement followed by deterioration due to development of arachnoiditis and paradoxical immune responses.
- Being tested
- Cyclophosphamide injection
- Keywords
- tubercular optico-chiasmatic arachnoiditis, tubercular radiculo-myelitis
- Medical subject headings
- Tuberculosis, Tuberculosis, Meningeal, Arachnoiditis, Latent Tuberculosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclophosphamide injectionDrug
Cyclophosphamide vs placebo
| Study group | Role | What they receive |
|---|---|---|
| Cyclophosphamide arm | Experimental | Participants randomized to the cyclophosphamide arm will be administered 750 mg/m2 body weight (rounded off to the nearest 50 mg above the calculated value) of cyclophosphamide diluted in normal saline every month (Total 6 months) along with equal dose of mesna 50% administered prior to infusion and 50% after the infusion of cyclophosphamide Receives: Drug: Cyclophosphamide injection |
| Placebo arm | Other | Participants randomized to the placebo group will be given similar quantity of normal saline and mesna as described above Receives: Drug: Cyclophosphamide injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional independece at 6 months
To compare the proportion of patients who attain functional independence (mRS-modified Rankin scale 0-2) 6 months after cyclophosphamide therapy for proliferative arachnoiditis refractory to corticosteroids and standard Anti-tubercular therapy in CNS tuberculosis to those who receive placebo.
Measured: 6 months
Secondary outcomes (15)
Independent ambulation
To compare the proportion of patients who attain independent ambulation 6 months after cyclophosphamide therapy for proliferative arachnoiditis refractory to corticosteroids and standard Anti-tubercular therapy in CNS tuberculosis to those who receive placebo.
Measured: 6 months
Improvement in modified Rankin scale
To compare the proportion of patients improving from mRS ≥3 to mRS ≤2 six months post cyclophosphamide therapy
Measured: 6 months
Improvement in visual acuity (1)
To compare the proportion of patients who attain atleast 2 points improvement on Snellen's chart in visual acuity 6 months after cyclophosphamide therapy for proliferative arachnoiditis refractory to corticosteroids and standard Anti-tubercular therapy in CNS tuberculosis to those who receive placebo.
Measured: 6 months
Improvement in visual acuity (2)
To compare proportion of patients who attain atleast two point improvement on a semiquantitative visual acuity measurement in those who have visual acuity less than 1/60 on snellen's chart (finger counting at 1 m, hand movements at 1 m, perception of light, no perception of light considered as discrete points below 1/60 vision on standard Snellen's chart) 6 months after cyclophosphamide therapy
Measured: 6 months
Improvement in visual acuity (3)
To compare proportion of patients improving from visual acuity of <3/60 in the better eye to 3/60 or more 6 months post cyclophosphamide therapy
Measured: 6 months
Improvement in sphincter function
To compare the proportion of patients who attain improvement in bladder/bowel function 6 months after cyclophosphamide therapy for proliferative arachnoiditis refractory to corticosteroids and standard Anti-tubercular therapy in CNS tuberculosis to those who receive placebo..
Measured: 6 months
Change in mRS
Shift analysis pre-and 6 months post therapy in terms of change in mRS
Measured: 6 months
Patient well being
Comparing Global patient well being as assessed by SF-36 pre and 6 months post cyclophosphamide therapy
Measured: 6 months
Life threatening infections
Occurrence of life threatening infections necessitating cessation of therapy upto 3 months post cyclophosphamide therapy
Measured: 3 months
Infections needing hospitalization
Occurrence of infections needing hospitalization or intravenous antibiotic/antiviral/anti-fungal therapy upto 3 months post cyclophosphamide therapy
Measured: 3 months
Flare up of TB
Flare up of underlying tuberculosis upto 3 months post cyclophosphamide therapy
Measured: 3 months
Cytopenias
Occurrence of Grade III cytopenias defined as per common terminology criteria for adverse events v 5.0 upto 6 weeks post cyclophosphamide therapy
Measured: 6 weeks
Transaminitis
Grade III transaminitis as per CTCAE v 5.0 upto 6 weeks post cyclophosphamide therapy
Measured: 6 weeks
Hemorrhagic cystitis
Occurrence of hemorrhagic cystitis upto 2 weeks post cyclophosphamide therapy
Measured: 2 weeks
Adverse effects
Any other significant adverse effect
Measured: 3 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 2020
- Study start1 Jan 2021 (estimated)
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2024 (estimated)
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 TuberculosisStudies 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 2/3
- 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
- All India Institute of Medical Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Arunmozhimaran Elavarasi, All India Institute of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04620772
- Sponsor's protocol ID
- IEC-975/03.10.2020
- Status last verified
- Dec 2020
- First submitted
- 27 Oct 2020
- Last updated
- 30 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Based on Institutional protocol
Publications
Goyal V, Elavarasi A, Abhishek, Shukla G, Behari M. Practice Trends in Treating Central Nervous System Tuberculosis and Outcomes at a Tertiary Care Hospital: A Cohort Study of 244 Cases. Ann Indian Acad Neurol. 2019 Jan-Mar;22(1):37-46. doi: 10.4103/aian.AIAN_70_18.
PubMed 30692758Gonzalez-Duarte A, Higuera-Calleja J, Flores F, Davila-Maldonado L, Cantu-Brito C. Cyclophosphamide treatment for unrelenting CNS vasculitis secondary to tuberculous meningitis. Neurology. 2012 Apr 17;78(16):1277-8. doi: 10.1212/WNL.0b013e318250d84a. Epub 2012 Apr 4. No abstract available.
PubMed 22491870Celotti A, Vianello F, Sattin A, Malipiero G, Faggin R, Cattelan A. Cyclophosphamide immunomodulation of TB-associated cerebral vasculitis. Infect Dis (Lond). 2018 Oct;50(10):779-782. doi: 10.1080/23744235.2018.1467038. Epub 2018 Apr 28. No abstract available.
PubMed 29708006
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Cyclophosphamide in the Treatment of Refractory Proliferative Arachnoiditis in Central Nervous System Tuberculosis- A Randomized Double Blinded Placebo Controlled Trial
- ClinicalTrials.gov ID
- NCT04620772
- Acronym / sponsor ID
- IEC-975/03.10.2020
- Keywords
- tubercular optico-chiasmatic arachnoiditis, tubercular radiculo-myelitis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- All India Institute of Medical SciencesUniversity, hospital or other
- Study start
- 1 Jan 2021 (estimated)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 9 Nov 2020 (actual)
- 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.
Tubercular meningitis occurs in around 10% of those with extrapulmonary tuberculosis and is a major cause of mortality and morbidity. Inspite of effective Anti-tubercular drugs, still around 30% of patients develop complications due to arachnoiditis such as spinal tubercular radiculomyelitis, optico-chiasmatic arachnoiditis, development of new tuberculomas after starting therapy etc. which are probably immune mediated inflammatory responses due to paradoxical reaction to ATT.
The management of arachnoiditis is far from satisfactory. High dose methylprednisolone, intrathecal hyaluronic acid, thalidomide have been tried in small case series and case reports. However, the results have not been satisfactory.
There are two published reports of cyclophosphamide usage in TBM related vasculitis and stroke The investigators tried cyclophosphamide in four patients after consent, and found remarkable improvement in all of them. (Under peer review) In order to test this hypothesis, a randomized controlled trial is needed.
Detailed description
Tubercular arachnoiditis occurring as a late complication of TBM is due to immune mediated reactions and is usually treated with high doses of corticosteroids. In the investigators' experience patients have received corticosteroids upto a duration of 20-28 months. In such cases, if the patient is refractory to corticosteroids, it is logical to step up to cyclophosphamide rather than continuing to give high dose steroids only similar to other immune mediated conditions such as CIDP, systemic and CNS vasulitis, Lupus nephritis etc.
Cyclophosphamide has been used most widely in the management of Lupus nephritis and maximum experience with the drug has been in this indication. So the dosage and duration of administration has been adapted from the induction phase dosing schedule of the ACR guidelines for the management of Lupus nephritis. The guidelines recommend 500 to 1000 mg/m2 BSA of cyclophosphamide. So the investigators decided to use 750 mg/m2 of the drug rounded off to the higher 50 mg.
Pathogenesis of tubercular arachnoiditis and Possible mechanism of cyclophosphamide Tubercular arachnoidiitis is a delayed complication in most cases due to inflammation in the optochiasmatic, spinal and other cranial nerves leading to neurologic deficits. In such situations, it is seen that there is an initial improvement in clinical symptoms followed by deterioration due to enlargement of lesions or appearance of new lesions, especially in patients with extrapulmonary tuberculosis. These new lesions are probably due to immune mediated mechanisms-it has been seen that in more than 95% of cases are drug sensitive and corticosteroids are the cornerstone of management of such patients. The complete pathogenesis of immune medicated tissue injury in tuberculosis has not been elucidated. Various studies have shown that there is an excess of pro-inflammatory cytokines and chemokines including TNF-alpha and interleukins that lead to recruitment of inflammatory cells and an exhuberant immune response in patients who develop these complications as compared to those who don't. There have been studies which have shown genetic polymorphisms in the genes encoding pro-inflammatory and anti-inflammatory cytokines-leading to difference in response to corticosteroids like LTA4 (Leukotriene- A4 hydrolase) gene. It is also well known that various immune mediated diseses such as SLE, multiple sclerosis etc are triggered by viruses and other infections. It is routine practice to treat acute bacterial meninigitis and tubercular meningitis with dexamethasone, which is again a steroid and immunosuppressant along with specific antibiotics or anti-tubercular therapy respectively based on evidence from systematic reviews and meta-analysis. Neurocysticercosis is treated with corticosteroids and other immunosuppressant medications such as methotrexate as recommended in guidelines, adalimumab and eternacept used as steroid sparing agents. Various other immune mediated complications of acute and chronic infections such as dengue myocarditis, ADEM etc are also treated with immunosuppression including corticosteroids. Lepra reactions in Leprosy are also treated using steroids, thalidomide, methotrexate, azathioprine, cyclophosphamide, cyclosporine etc.
In recent times, the corticosteroid- Dexamethasone has been shown to have mortality benefit in patients with moderate to severe COVID, proven in a randomized controlled trial. Simliarly, even though there are no RCT data, Tocilizumab, siltuximab and other immunosuppressant medications have been approved for emergency use based on their efficacy shown in large case series in COVID-19.
Justification for the use of cyclophosphamide this study:
Cyclophosphamide is a potent immunosuppressant agent. It is used in Lupus and other immune mediated conditions and has been shown to be effective in randomized controlled trials in Lupus nephritis, granulomatous polyangiiitis etc. Though it is widely used off label in various inflammatory immune mediated neurologic conditions such as CNS vasculitis, CIDP, multiple sclerosis, autoimmune encephalitis, refractory myasthenia gravis etc, the evidence for this is basically derived from the rheumatology and nephrology literature. It has to be emphasized that all the data for immunosuppression with azathioprine, cyclophosphamide, mycophenolate mofetil, methotrexate, calcineurin inhibitors in various immune mediated conditions have been derived from renal transplant/solid organ transplant cohorts, lupus nephritis cohorts, systemic vasculitis cohorts and inflammatory bowel disease cohorts. The various guidelines issued by the professional bodies for these conditions form the principles of treatment of rare conditions such as neuroimmunologic diseases. The major adverse effects of concern in patients on Cyclophosphamide is the occurrence of life threatening infections such as tuberculosis. It has been seen that patients on high dose corticosteroids and cyclophosphamide are at higher risk of infection than those on steroids alone or on neither drug.Though the risk of TB increases with administration of immunosuppressants, the mainstay of management is early diagnosis and starting appropriate therapy and not cessation of immunosuppression. In the context of SLE the occurrence of infection is also dependent on various other factors such as disease activity, previous exposure to tuberculosis etc. The Americal college of rheumatology guidelines do not say anything on the course of action in case patients develop infections. There are no professional guidelines for the management of infections in patients on cyclophosphamide. There are no RCTs on the most appropriate form of ATT regimens or the modification of immunosuppression in these conditions. There are various case series and case reports who have been systematically studied infections and their management including the large prospective EuroLupus cohort. None of these authors advice complete cessation of immunosuppression in patients who develop infections including tuberculosis. The guidelines for management of tuberculosis such as British thoracic society guidelines for the management of TB in CKD,do not suggest cessation of immunosuppression in post renal transplant patients. The guidelines clearly mention that TB in patients with CKD have to be treated in same lines of immunocompetent patients, and the anti-tubercular drugs may need to be given in renal modified doses. In case of tuberculosis risk, it has been shown by various long term follow up studies that a considerable proportion of patients with latent TB infections progress to develop active TB post transplantation or after treatment with immunosuppressants or biologic agents such as anti TNF therapy. In such circumstances, the guidelines suggest not dropping immunosuppression, but treatment of latent TB or active TB. The guidelines for the management of active TB in solid organ transplantation suggests early diagnosis and treatment of tuberculosis along with increasing the dose of immunosuppressants such as corticosteroids, cyclosporine or tacrolimus as the ATT regimen containing rifamycins tend to induce the metabolism of these immunosuppression and trough levels of calcineurin inhibitors have to be maintained by increasing the dose of cyclosporine or tacrolimus by upto 4-5 times.
These recommendations are mainly based on a large multicentre cohort of more than 2000 patients derived from 187 publications who had received solid organ transplantation and developed tuberculosis, where the dose of immunosuppressant drugs had been increased to maintain adequate blood levels to avoid rejection of the organ graft. Similar recommendations have been made by the TBNET consensus statement for management of Tuberculosis in transplant recipients.
The management of immunosuppression in these cases are mainly based on expert consensus in other conditions such as rheumatoid arthritis, spondyloarthropathy etc. The TBNET consensus statement also says that in case the organ is not vital or the infection is life threatening, it may be considered to reduce the level of immunosuppression. The guidelines for the use of anti TNF-α therapy is clearer in this aspect. While the occurrence of active TB during anti TNF-α therapy warrants withholding (NOT cessation) of immunosuppression, it may be re started 2 months after ATT the if patients demonstrate a favorable response to anti-TB therapy and require early resumption of anti TNF therapy.The BTS guidelines suggest that if active TB was diagnosed prior to starting of "Anti-TNF-α therapy, treatment should not be commenced for at least 2 months after antituberculosis treatment with full compliance has begun, supervised by a thoracic physician orinfectious disease specialist, and until the drug susceptibility profile of the organism in those with positive cultures is known, as a minimum." However, if the patient develops TB while on anti TNF therapy the anti-TNF-α treatment can be continued if clinically indicated because the patient would otherwise be prevented from receiving the continued clinical benefit to their underlying disease and may have a flare up or major clinical deterioration. The rationale provided by these guidelines is that HIV positive individuals with reduced CD4 counts and clinical TB, who are even more immunosuppressed than those on anti-TNF-a treatment, respond just as well to TB treatment as those who are HIV negative. Taking that logic forward, it may be reasonable to start therapy with cyclophosphamide three months after starting standard Anti tubercular therapy in patients with tubercular meningitis who have shown clearcut improvement followed by deterioration due to development of arachnoiditis and paradoxical immune responses.
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 |
|---|---|---|
| Cyclophosphamide arm | Experimental | Drug: Cyclophosphamide injectionParticipants randomized to the cyclophosphamide arm will be administered 750 mg/m2 body weight (rounded off to the nearest 50 mg above the calculated value) of cyclophosphamide diluted in normal saline every month (Total 6 months) along with equal dose of mesna 50% administered prior to infusion and 50% after the infusion of cyclophosphamide |
| Placebo arm | Other | Drug: Cyclophosphamide injectionParticipants randomized to the placebo group will be given similar quantity of normal saline and mesna as described above |
Who may qualify
- Ages
- 14 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- Patients attending Neurology/Pulmonary Medicine/Medicine/Geriatric Medicine OPD/admitted in respective wards with proliferative tubercular arachnoiditis refractory to corticosteroids and standard Anti-tubercular drugs for CNS tuberculosis
- Atleast 14 years of age of all sexes
- Not more than 60 years of age at time of enrolment
- Patient was started on ATT for tubercular meningitis and had clearcut clinical improvement with resolution of fever/constitutional symptoms AND improvement in headache, vomiting and sensorium for atleast 10 days following which there is deterioration again due to arachnoiditis
- Developed paraparesis/quadriparesis/sphincter dysfunction due to spinal radiculomyelitis or vision loss due to due to optico-chiasmatic arachnoiditis with imaging evidence of arachnoiditis
Key exclusion criteria (16 listed)
- Not willing to provide consent
- Not willing to adhere to protocol
- Developed significant drug induced liver dysfunction so that patient is not being given Rifampicin, INH or pyrazinamide and is on modified ATT including quinolones, ethambutol and aminoglycosides or second line drugs only in the absence of Rifampicin and INH
- Drug resistant tubeculosis
- Men and Women of childbearing potential who are not using adequate contraception or women who are pregnant and lactating
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 injectionCyclophosphamide vs placebo | Drug | Cyclophosphamide arm, Placebo arm |
Primary outcome
Functional independece at 6 months
Time frame: 6 months
To compare the proportion of patients who attain functional independence (mRS-modified Rankin scale 0-2) 6 months after cyclophosphamide therapy for proliferative arachnoiditis refractory to corticosteroids and standard Anti-tubercular therapy in CNS tuberculosis to those who receive placebo.
15 secondary outcomes
Independent ambulation
Time frame: 6 months
Improvement in modified Rankin scale
Time frame: 6 months
Improvement in visual acuity (1)
Time frame: 6 months
Improvement in visual acuity (2)
Time frame: 6 months
Improvement in visual acuity (3)
Time frame: 6 months
Improvement in sphincter function
Time frame: 6 months
Change in mRS
Time frame: 6 months
Patient well being
Time frame: 6 months
Life threatening infections
Time frame: 3 months
Infections needing hospitalization
Time frame: 3 months
Flare up of TB
Time frame: 3 months
Cytopenias
Time frame: 6 weeks
Transaminitis
Time frame: 6 weeks
Hemorrhagic cystitis
Time frame: 2 weeks
Adverse effects
Time frame: 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2020.
- Background: PubMed 30692758Goyal V, Elavarasi A, Abhishek, Shukla G, Behari M. Practice Trends in Treating Central Nervous System Tuberculosis and Outcomes at a Tertiary Care Hospital: A Cohort Study of 244 Cases. Ann Indian Acad Neurol. 2019 Jan-Mar;22(1):37-46. doi: 10.4103/aian.AIAN_70_18.
- Background: PubMed 22491870Gonzalez-Duarte A, Higuera-Calleja J, Flores F, Davila-Maldonado L, Cantu-Brito C. Cyclophosphamide treatment for unrelenting CNS vasculitis secondary to tuberculous meningitis. Neurology. 2012 Apr 17;78(16):1277-8. doi: 10.1212/WNL.0b013e318250d84a. Epub 2012 Apr 4. No abstract available.
- Background: PubMed 29708006Celotti A, Vianello F, Sattin A, Malipiero G, Faggin R, Cattelan A. Cyclophosphamide immunomodulation of TB-associated cerebral vasculitis. Infect Dis (Lond). 2018 Oct;50(10):779-782. doi: 10.1080/23744235.2018.1467038. Epub 2018 Apr 28. No abstract available.
- Individual participant dataSharing planned: Undecided. Based on Institutional protocol
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Cyclophosphamide injection in people with Tuberculosis and Tubercular Meningitis, run by All India Institute of Medical Sciences.
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 14 inclusion and 16 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: status not confirmed, as of the sponsor's last update on 30 Dec 2020. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Arunmozhimaran Elavarasi, MD DM (+919013844274, arun_ela@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Functional independece at 6 months (time frame: 6 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 NCT04620772, 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 30 Dec 2020.