AHSCT With Fludarabine and Cyclophosphamide Based Conditioning Regimes in Patients With Multiple Sclerosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Fludarabine Phosphate for Injection.
- Who it may be relevant to
- Registry conditions: Multiple Sclerosis. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Russia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Trial of the Efficacy and Safety of High-dose Immunosuppressive Therapy Based on Fludarabine and Cyclophosphamide-containing Conditioning Regimen Followed by Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple Sclerosis.
Overview
One of the possible options for the treatment of MS at present is a high-dose immunosuppressive therapy followed by autologous hematopoietic stem cell transplantation (HIST-AHSCT), which is a highly effective treatment for patients with relapsing-remitting MS. This method of MS treatment was introduced in 1997. Significant complications and mortality associated with HIST-ATHSC is an obstacle to broad use of this method. The risk is even greater in patients with advanced disease, long duration of previous treatment and aggressive forms of MS. Despite toxicity certain progressive cases of MS are still an indication for HIST-autoHSCT. Most commonly used conditioning regimens for multiple sclerosis include high-dose cyclophosphamide. One of the options to reduce cyclophosphamide-related toxicity and dose is addition of fludarabine. Fludarabine is a cytostatic drug, an antimetabolite from the group of purine antagonists. It has a pronounced immunosuppressive activity and no overlapping toxicity with cyclophosphamide. The study will evaluate the safety and efficacy of this combination.
Interventions
- Drug Fludarabine Phosphate for Injection
Intravenous injection of fludarabine phosphate at a dose of 30 mg/m2 from day -5 to day -2 of immunoablative conditioning regimen.
Primary outcome measures
- Multiple sclerosis progression free survival [Time frame: 365 days]
Secondary outcome measures (9)
- Cumulative incidence of mortality [Time frame: 365 days]
- To evaluate adverse effects after FluCy200 conditioning regimen [Time frame: 365 days]
- Quality of life status 1 [Time frame: 365 days]
- Quality of life status 2 [Time frame: 365 days]
- Quality of life status 3 [Time frame: 365 days]
- Quality of life status 4 [Time frame: 365 days]
- Neurological status 1 [Time frame: 365 days]
- Immune reconstitution [Time frame: 365 days]
- Magnetic resonance imaging response [Time frame: 365 days]
Eligibility criteria
Inclusion criteria
- Age 18-65;
- 1.0-6.5 points on the EDSS scale (for MS);
- Length of illness - any;
- Disease progression during the last 6 months while taking drugs of 1st and 2nd lines;
- An established and confirmed diagnosis of an autoimmune disease in the previous stages of treatment;
- Ineffectiveness, inaccessibility or intolerance of Disease-Modifying Therapies;
- Relapse after AHSCT.
- Absence of severe concomitant somatic pathology;
- Left ventricular injection fraction > 50%;
- Karnofsky Performance Score (KPS) > 30%;
- The ability to take oral medications;
- Life expectancy is more than 1 month;
- Signed informed consent of the patient or legal representatives.
Exclusion criteria
- Moderate or severe cardiac dysfunction, left ventricular ejection fraction <50%
- Moderate or severe decrease in pulmonary function, FEV1 <70% or DLCO<70% of predicted
- Respiratory distress >grade I
- Severe organ dysfunction: AST or ALT >5 upper normal limits, bilirubin >1.5 upper normal limits, creatinine >2 upper normal limits
- Creatinine clearance < 60 mL/min
- Uncontrolled bacterial or fungal infection at the time of enrollment
- Requirement for vasopressor support at the time of enrollment
- Karnofsky performans status <30%
- Pregnancy
- Somatic or psychiatric disorder making the patient unable to sign informed consent
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Russia · 1 center
- First Pavlov State Medical University of St. Petersburg — Saint Petersburg
Identifiers
NCT: NCT05832515 · FluCy_MS