Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||18 Years - 70 Years|
- - Patients aged between 18 and 70 years.
- - Primary myelofibrosis or myelofibrosis secondary to essential thrombocythemia or polycythemia Vera proven by marrow biopsy.
- - The myelofibrosis should combine at least 2 of the following criteria: - constitutional symptoms: weight loss > 10% in one year, fever (without infection), recurrent muscle, bone or join pains, extreme fatigue.
- - anemia with hemoglobin < 10 gr/dL or red blood cell transfusion requirement.
- - thrombocytopenia < 100 G/L.
- - peripheral blast count > 1% at least found 2 times.
- - white blood cell count > 25 G/L (before a cytoreductive treatment) - Karyotype: +8, -7/7q-, i(17q), -5, 5q-, 12p-, inv(3), 11q23.
- - Performance status according to ECOG at 0, 1 or 2.
- - With health insurance coverage.
- - Having signed a written informed consent.
- - Women agreed to take nomegestrol acetate as contraception during and up to 6 months after treatment by treosulfan.
- - Men agreed not to conceive child during and up to 6 months after treatment by treosulfan.
- - Myelofibrosis transformed into acute leukemia.
- - Poor performance status with ECOG 3 or more.
- - Cardiac failure with EF < or = 50% currently or in the past (even if corrected after treatment) - Renal failure with creatininemia > 130 µmol/L or clearance < 50ml/min.
- - Respiratory function altered with vital capacity < 70% or forced expired volume < 70% - Biological significant liver abnormalities; ASAT or ALAT> 2 x normal range, bilirubin > 1,5 x normal range.
- - HLA matched donor available.
- - Tutorship or curatorship.
- - Unwilling or unable to comply with the protocol.
- - Pregnant woman or breastfeeding.
- - Contraindications to treosulfan.
- - Hypersensitivity to the active substance.
- - Active non-controlled infectious disease.
- - Fanconi anaemia and other DNA breakage repair disorders.
- - Administration of live vaccine.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|Assistance Publique - Hôpitaux de Paris|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Principal Investigator Affiliation||N/A|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
|Overall Status||Not yet recruiting|
The disease, disorder, syndrome, illness, or injury that is being studied.
Experimental: Allogenic transplantation using treosulfan in conditioning regimen
Haplo-identical transplantation using treosulfan in conditioning regimen Treosuflan, in the conditioning regimen will be administrated as followed 10 gr/m2 per day -4, -3 and -2 IV route In combination with: Thiotepa 5 mg/kg on day -6 Fludarabine 30 mg/m2 per day from day -5 to day -1
Other: - Allogenic transplantation transplantation
Haplo-identical transplantation with the use of Treosulfan, Thiotepa and Fludarabine in conditioning regimen.
This trial has no sites locations listed at this time. If you are interested in learning more, you can contact the trial's primary contact:
Marie Robin, Dr
For additional contact information, you can also visit the trial on clinicaltrials.gov.