At a glance
ClinicalIndex Comparison Record- ✓Age ≥3 years old
- ✓Weight >12 kg
- ✓Bone marrow cellularity <30% excluding lymphocytes
- ✓At least two cytopenias: ANC <500/μL, platelets <20,000/μL, or reticulocyte count <60,000/μL
- ✕Known Fanconi anemia or constitutional marrow failure syndrome
- ✕Clonal disorder on cytogenetics within 12 weeks involving chromosome 7 or complex karyotype
- ✕Prior immunosuppressive therapy (ATG, cyclosporine, alemtuzumab, high-dose cyclophosphamide) or eltrombopag
- ✕SGOT or SGPT >2.5× ULN or total bilirubin >1.5× ULN
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
NCT04304820Phase 2RecruitingUpdate OverdueUpdated 3mo ago · Completion was 18mo agoEarly Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
In Brief
A Phase 2 clinical trial evaluating Eltrombopag, Cyclosporine, and 1 other intervention for Severe Aplastic Anemia. Currently recruiting, targeting 80 participants across 1 site.
Signals
Detailed Summary
Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells. Standard treatment for SSA is a combination of 3 drugs (Cyclosporine \[CsA\], Eltrombopag \[EPAG\], and horse anti-thymocyte globulin \[h-ATG\]). Researchers want to see if starting people at a lower dose of CsA with EPAG before giving them h-ATG is helpful. Objective: To learn if early initiation of oral therapy with CsA and EPAG is safe and effective in people who have SAA and have not been treated with a course of immunosuppressive therapy and EPAG. Eligibility: People ages 3 and older with SAA Design: Participants will be screened with: * medical history * physical exam * electrocardiogram * blood tests * family history * bone marrow biopsy * current medicines. Participants may be screened remotely via telephone conference. Participants will take a lower oral dose of CsA and EPAG. They will take CsA twice a day for 6 months. They will take EPAG for 6 months. Those who cannot visit the NIH Clinical Center within 72 hours will start taking the drugs at home. They will have weekly telephone calls with NIH staff until they visit the Clinical Center. Participants may get h-ATG at the Clinical Center for 4 days. For this, they will have a central line placed. It is a plastic tube inserted into a neck, chest, or arm vein. Participants will repeat most screening tests throughout the study. Participants will have follow-up visits at the Clinical Center at 3 months, 6 months, and annually for 5 years after the start of the study....
Study Details
Timeline
Interventions
Between 12 and 17yo (adult dose of 150mg) Between 6 and 11yo (75 mg) Between 3 and 5 yo (2.5 mg/kg)
Day 1 to start of h-ATG: 2mg/kg/day by mouth (All subjects) Start of h-ATG to Month 6: 3 mg/kg/dose by mouth administered every 12 hours (12yo and above) 6 mg/kg/dose by mouth administered every 12 hours (less than 12yo) Month 6 to Month 24: Dosing to be adjusted based on response
h-ATG at a dose of 40 mg/kg/day for 4 days (intravenously for approximately 4 hours daily) Note: Omitted in patients who have achieved a complete response at the initial NIH visit after initiating oral treatment remotely