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NCT04304820 · ClinicalTrials.gov registry record · Phase 2
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
A Phase 2 study of Severe Aplastic Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Recruiting
- Registry status
- Phase 2
- Development phase
- 80
- Enrollment target
- 1
- Study location
NCT04304820: Recruiting Phase 2 study of Severe Aplastic Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT04304820 is a Phase 2 study of Severe Aplastic Anemia that is actively recruiting participants, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 80 participants, below the 122-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (34% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04304820, a Phase 2 study of Severe Aplastic Anemia, is actively recruiting participants, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 80 participants
- Enrollment target
- 1
- Study location
Study 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....
Primary Outcome
For the primary endpoint of this study, the treatment is defined to be "feasible" to a participant if he/she meets the safety and feasibility criteria. Safety and feasibility criteria is a composite measure defined as TRSAE, mis- and altered diagnosis, and non-compliance with the regimen or failure to establish care at the National Institutes of Health Clinical Center. Study treatment period is defined as the duration from the initiation of oral treatment (low dose Cyclosporine and Eltrombopag)
Conditions Studied
Interventions
- DRUG Cyclosporine
- DRUG Eltrombopag
- DRUG Horse-Anti-thymocyte-Globulin
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 80 participants |
| Start Date | 2020-05-07 |
| Est. Completion | 2033-12-18 |
| Phase | Phase 2 |
What NCT04304820 shows while recruiting
NCT04304820 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 80 participants, a relatively small participant target, below the 122-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (34% lower).
The record links to 1 condition, with Severe Aplastic Anemia appearing as the primary indexed condition, and to 3 interventions - of which Cyclosporine is the first listed.
NCT04304820 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT04304820 about?
NCT04304820 is a clinical study titled "Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)". 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...
What is the current status of trial NCT04304820?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 80 participants. The study started on 2020-05-07. Estimated completion is 2033-12-18.
What conditions does trial NCT04304820 study?
This clinical trial studies the following conditions: Severe Aplastic Anemia.
What interventions are being tested in trial NCT04304820?
The interventions under investigation include: Cyclosporine (DRUG), Eltrombopag (DRUG), Horse-Anti-thymocyte-Globulin (DRUG).
Who is sponsoring clinical trial NCT04304820?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04304820 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04304820's enrollment target sits among peer trials
80 6th of 16 higher than 11 of 16 other Severe Aplastic Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Severe Aplastic Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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