EUCTR2018-001489-41-SE进行中（未招募）1 期
Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Patients 30 days to 65 years of age at the time of inclusion with a diagnosis of severe aplastic anemia or single lineage cytopenia, defined as follows:
- •- Bone marrow hypocellular for age on biopsy based on review at the enrolling institution within 90 days of protocol inclusion
- •- One or more of the following (in peripheral blood):
- •-neutrophils < 0.5 x 109/L
- •-platelets < 30 x 109/L, or platelet transfusion dependence*
- •-reticulocytes < 50 x 109/L in anemic patients, or red cell transfusion dependence*
- •*transfusion dependence will be defined as greater than ONE transfusion of platelets or red blood cells in the last year prior to protocol inclusion
- •2. Diagnosis with one of the following DC categories on any prior evaluation:
- •A. age-adjusted mean telomere length < 1%ile in peripheral blood lymphocytes by flow-FISH on any occasion, as reported by a CLIA-approved laboratory
- •B. pathogenic mutation(s) in DKC1,TERC, TERT, NOP10, NHP2, TCAB1, TINF2, CTC1, RTEL1, ACD, or PARN as reported by a CLIA-approved laboratory
- •C. Dyskeratosis congenita diagnosed by:
- •1. clinical triad: abnormalities of skin pigmentation, nail dystrophy, oral leukoplakia
- •2. one of clinical triad and presence of two or more associated features:
- •epiphora, developmental delay, mental retardation, pulmonary disease, short stature, dental caries/loss, esophageal stricture, grey hair, hyperhidrosis, malignancy, intrauterine growth retardation (IUGR), liver disease, enteropathy, ataxia/cerebellar hypoplasia, hypogonadism, microcephaly, urethral stricture, osteoporosis/AVN, scoliosis, deafness
- •D. Hoyeraal Hreidarsson syndrome: cerebellar hypoplasia, microcephaly, IUGR, T+B-NK- immunodeficiency
- •E. Revesz syndrome: exudative retinopathy, cerebral calcifications, IUGR, fine sparse hair, ataxia
- •3. Availability of a HLA-compatible related or unrelated donor, defined as a 7/8 or 8/8 match for HLA-A, B, C, and DRB1. Related donors must be ruled out for telomere disease by appropriate clinical and diagnostic measures (for example, clinical evaluation, flow-FISH telomere length testing, genetic testing, and/or bone marrow examination).
- •4. For patients under 18 both parents must give a written informed consent.
- •5. Matched unrelated donor must consent to provide a bone marrow allograft.
- •6. The diagnosis of Fanconi anemia must be excluded by mitomycin C or diepoxybutane (DEB) chromosomal breakage testing on peripheral blood at a CLIA-approved laboratory on any prior evaluation. This evaluation is not required for patients with a pathogenic mutation in a known DC-associated gene as defined in 5.1.1.2.B above.
- •7. Adequate renal function with glomerular filtration rate equal to or greater than 30 ml/min/1.73 m2 measured within 30 days of protocol inclusion.
- •8.Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, if they are using effective methods of contraception during the study. Acceptable birth control methods are those with a failure rate of less than 1% per year when used consistently and correctly. Such methods include (in Recommendations related to contraception and pregnancy testing in clinical trials”, supplied from www.hma.eu/):
- •a. Combined (estrogen and progestogen containing hormonal contraception associated with inhibition of ovulation.
- •-intravaginal
- •-transdermal
- •b. progestogen-only hormonal contracption associated with inhibition of ovulation
- •- injectable
排除标准
- •1. Clonal cytogenetic abnormalities associated with MDS or AML on bone marrow examination performed within 90 days of protocol inclusion.
- •2. Karnofsky performance status < 40% for patients 16 years old or older; or Lansky < 40% for patients < 16 years old.
- •3. Uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms). Patients with fever despite broad-spectrum antimicrobials but no clinical or hemodynamic evidence of sepsis will be allowed.
- •4. Positive for the human immunodeficiency virus (HIV) by nucleic acid testing.
- •5. Pregnancy (positive B-HCG) or breastfeeding.
- •6. Known severe or life-threatening allergy or intolerance to fludarabine, alemtuzumab, cyclosporine, or mycophenolate mofetil.
- •7. In HLA mismatched transplants: positive patient anti-donor HLA antibody, defined as clinically significant based on institutional standards.
- •8. Prior allogeneic marrow or stem cell transplantation.
- •9. Prior solid organ transplantation.
- •10. Non-hematological co-morbid condition(s) or other factors (for example, psychological or social factors) that preclude the patients candidacy for performing BMT under the protocol regimen, in the opinion of the treating physician
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