Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study ID
NCT04304820
Phase
PHASE2
Status
Recruiting
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Conditions

  • Severe Aplastic Anemia

Eligibility Criteria

Sex
ALL
Age
3 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Eltrombopag — DRUG
    Between 12 and 17yo (adult dose of 150mg) Between 6 and 11yo (75 mg) Between 3 and 5 yo (2.5 mg/kg)
  • Cyclosporine — DRUG
    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
  • Horse-Anti-thymocyte-Globulin — DRUG
    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

Study Details

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....

Key Dates

Start date
May 7, 2020
Status verified
Feb 2026
Primary completion
Dec 18, 2024
Completion
Dec 18, 2033

Study Design

Enrollment
80 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Participants with Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag
    Treatment-naïve Severe Aplastic Anemia (SAA) participants start Cyclosporine (CsA) on Day 1 at 2 mg/kg/day. At initiation of Horse Anti-Thymocyte-Globulin (h-ATG), dosing increases to 3 mg/kg every 12 hours for those ≥12 years (6 mg/kg/day total) and 6 mg/kg every 12 hours for those \<12 years (12 mg/kg/day total). Actual body weight is used unless \>125% ideal body weight (IBW), where adjusted IBW applies. CsA is titrated to a trough of 200-400 mcg/L. At 6 months, responders reduce to 2 mg/kg/day through 24 months, with return to full dosing if relapse occurs. h-ATG is given at 40 mg/kg/day for 4 days IV, unless the patient already shows a complete response at the initial NIH visit after remote oral therapy. Eltrombopag (EPAG) starts on Day 1 at 150 mg/day for ages 12-17, 75 mg/day for ages 6-11, and 2.5 mg/kg/day for ages 3-5. For East and Southeast Asian patients, starting doses are reduced to 75 mg/day (ages 12-85), 37.5 mg/day (ages 6-11), and 1.25 mg/kg/day (ages 3-5).
  • Experimental: Extension Cohort
    Treatment-naïve Severe Aplastic Anemia (SAA) participants start Cyclosporine (CsA) on Day 1 at 2 mg/kg/day. At initiation of Horse Anti-Thymocyte-Globulin (h-ATG), dosing increases to 3 mg/kg every 12 hours for those ≥12 years (6 mg/kg/day total) and 6 mg/kg every 12 hours for those \<12 years (12 mg/kg/day total). Actual body weight is used unless \>125% ideal body weight (IBW), where adjusted IBW applies. CsA is titrated to a trough of 200-400 mcg/L. At 6 months, responders reduce to 2 mg/kg/day through 24 months, with return to full dosing if relapse occurs. h-ATG is given at 40 mg/kg/day for 4 days IV, unless the patient already shows a complete response at the initial NIH visit after remote oral therapy. Eltrombopag (EPAG) starts on Day 1 at 150 mg/day for ages 12-17, 75 mg/day for ages 6-11, and 2.5 mg/kg/day for ages 3-5. For East and Southeast Asian patients, starting doses are reduced to 75 mg/day (ages 12-85), 37.5 mg/day (ages 6-11), and 1.25 mg/kg/day (ages 3-5).

Primary Outcome Measure

Number of Participants Meeting Safety and Feasibility Criteria During Study Treatment Period With Oral Treatment (Low Dose Cyclosporine and Eltrombopag) Until the Start of Standard of Care Treatment (Horse Anti-thymocyte-globulin) [ Time Frame: Up to 12 Weeks from the initiation of oral treatment (Low Dose Cyclosporine and Eltrombopag) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)
[email protected]
800-411-1222

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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