/A - ANDROTELO

ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)0 个研究点目标入组 40 人开始时间: 2018年6月6日最近更新: 2019年11月25日

适应症

试验速览

暂无简介。

•Patient (s):
•- having a telomeropathy defined by the existence of a constitutional deleterious mutation of a gene involved in the maintenance of telomeres (TERT, TERC, DKC1, TINF2, RTEL1, PARN, ACD, NHP2, NOP10, NAF1, WRAP53, CTC1, ERCC6L2, USB1, POT1, DNAJC21 or a newly identified gene responsible for telomeropathy),
•- 15 years of age or older,
•- with severe hematologic involvement (platelets <20 G / L or PNN <0.5 G / L and / or hemoglobin <8 g / dL and / or need for transfusions) and / or pulmonary fibrosis with superior parenchymal involvement at 10% on the scanner.
•- being able to give informed consent for patients 18 years and older,
•- being able to give his consent and have the consent of the holder (s) of parental authority for children over 15,
•- being a beneficiary of a social security scheme.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 40
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Patient (s):
•- with HIV infection or active infection with hepatitis B or hepatitis C,
•- with severe hepatic impairment: transaminases ASAT and / or ALAT> 5N, or conjugated bilirubinemia> 30? mol / L, TP <50% (except vitamin K deficiency), or splanchnic thrombosis
•- having a tumoral pathology that has been active or treated for less than 5 years, with the exception of basal cell carcinoma or carcinoma in situ of the cervix,
•- having a history of organ transplantation or hematopoietic stem cells or having an indication for hematopoietic stem cell transplantation or organ transplantation within 6 months of inclusion,
•- having an absolute contraindication to treatment with danazol: active thrombosis or history of thromboembolic disease, porphyria, severe renal or cardiac insufficiency (NYHA stage III or IV), androgen-dependent tumor, pathological genital haemorrhage of etiology indeterminate,
•- having already received danazol for the treatment of telomeropathy,
•- having received another androgen within a period of less than 6 months,
•- receiving another experimental treatment,
•- receiving another hormonal treatment,
•- receiving treatment with simvastatin
•- unexplored pathological genital bleeding
•Concomitant treatment with Simvastatin
•- having a pregnancy plan and not committing to effective contraception while taking the treatment,
•- breastfeeding,
•- under tutorship or curatorship.

发起方