Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A

MI-összefoglaló

A vizsgálat saját részvételi feltételeiből. Gép olvasta ki az oldalon álló regiszterszövegből, ezért tévedhet. Hogy ki vehet részt, azt a lenti regiszterszöveg és a vizsgálat csapata dönti el.

The trial is looking for adults with acquired aplastic anemia whose disease did not respond to cyclosporin A (CsA), or who could not tolerate it. They will get sirolimus alone or sirolimus with CsA.

Életkor

18 years and older

Nem

All sexes

A betegség stádiuma

Acquired aplastic anemia, not the inborn type. At least one low blood count: hemoglobin < 100 g/L, platelets < 50 × 10⁹/L, or neutrophils < 1.0 × 10⁹/L. Able to do daily activities (ECOG score 0-2). No active infections.

Korábbi kezelés

CsA did not work after at least 3 months (no partial response), or the disease came back, or CsA could not be given at the standard dose because of side effects or other conditions. People who have used sirolimus before cannot join.

Biomarker vagy leletérték

PNH clone must be below 50%.

A részvétel fő kizáró okai

• Low blood counts from another cause, or bone marrow cancers such as MDS or AML
• Past stem cell transplant
• Serious side effects from CsA in the past, or allergy to sirolimus
• Active HIV, HCV or HBV infection, liver cirrhosis, or other cancer in the past 5 years (except localized basal cell skin cancer)
• Past blood clots, heart attack or stroke, use of blood thinners, or being pregnant or breastfeeding

A regiszter feltételei, szó szerint

Mit akar kideríteni a vizsgálat

This is a single center, randomized, open-label, phase II study to compare the efficacy of sirolimus combined with cyclosporin A (CsA) to sirolimus alone in Chinese subjects with aplastic anemia refractory/intolerant to CsA. The safety would also be evaluated. Patients would be randomized to receive sirolimus alone or sirolimus combined with CsA at a 1:3 ratio. Treatment with sirolimus will be started at 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng/ml. CsA will be given at 25-150 mg orally every 12 hours, with the dose adjusted based on renal function and trough concentration. For patients with normal renal function, the target trough concentration is approximately 150 ng/ml. For patients with impaired renal function, the cyclosporine A dose is reduced to 25-50 mg every 12 hours, aiming for recovery or stabilization of renal function. The hematological response rate and safety will be recorded and compared at 3 and 6 months after starting the study treatment (Week 13 and 25).

A vizsgálat 2 csoportja

• Sirolimus + cyclosporin AA vizsgált kezelés

  Kezelés: Sirolimus (Rapamune®) és Cyclosporin A (CsA)

• SirolimusPlacebo, összehasonlításként

  Kezelés: Sirolimus (Rapamune®)