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NCT05012111 · ClinicalTrials.gov registry record
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
A clinical trial of Severe Aplastic Anemia and Telomere Biology Disorders, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Recruiting
- Registry status
- 1,000
- Enrollment target
- 1
- Study location
NCT05012111: Recruiting study of Severe Aplastic Anemia and Telomere Biology Disorders, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT05012111 is a study of Severe Aplastic Anemia and Telomere Biology Disorders that is actively recruiting participants, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 1,000 participants, above the 64-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (1463% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05012111, a study of Severe Aplastic Anemia and Telomere Biology Disorders, is actively recruiting participants, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- RECRUITING
- Registry status
- 1,000 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking. Researchers want to better understand long-term outcomes in people with these diseases. Objective: To follow people diagnosed with acquired or inherited bone marrow failure disease and study the long-term effects of the disease and its treatments on organ function. Eligibility: People aged 2 years and older who have been diagnosed with acquired or inherited bone marrow failure or Telomere Biology Disorder. First degree family members may also be able to take part in the study. Design: Participants will be screened with a medical history, physical exam, and blood tests. They may have a bone marrow biopsy and aspiration. For this, a large needle will be inserted in the hip through a small cut. Marrow will be drawn from the bone. A small piece of bone may be removed. Participants may also be screened with some of the following: Cheek swab or hair follicle sample Skin biopsy Urine or saliva sample Evaluation by disease specialists (e.g., lung, liver, heart) Imaging scan of the chest Liver ultrasounds Six-Minute Walk Test Lung function test Participants will be put into groups based on their disease. They will have visits every 1 to 3 years. At visits, they may repeat some screening tests. They may fill out yearly surveys about their medicines, transfusions, pregnancy, bleeding, and so on. They may have other specialized procedures, such as imaging scans and ultrasounds. Participation will last for up to 20 years.
Primary Outcome
Rate of progression to transfusion dependent marrow failure, marrow failure requiring therapeutic intervention (such as medical therapy or HSCT) or the progression to hematological malignancy.
Conditions Studied
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1,000 participants |
| Start Date | 2021-10-25 |
| Est. Completion | 2041-02-04 |
What NCT05012111 shows while recruiting
NCT05012111 is an observational study that tracks outcomes without assigning an intervention. Its 1,000 participants enrollment target places it among the larger protocols in the corpus, above the 64-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (1463% higher).
The record links to 3 conditions, with Severe Aplastic Anemia appearing as the primary indexed condition, and to 0 interventions.
NCT05012111 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT05012111 about?
NCT05012111 is a clinical study titled "Natural History of Acquired and Inherited Bone Marrow Failure Syndromes". Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking. Researchers want to better understand long-term outcomes in people with these diseases. Objective: To foll...
What is the current status of trial NCT05012111?
This trial is currently recruiting. The enrollment target is 1,000 participants. The study started on 2021-10-25. Estimated completion is 2041-02-04.
What conditions does trial NCT05012111 study?
This clinical trial studies the following conditions: Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes.
Who is sponsoring clinical trial NCT05012111?
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 NCT05012111 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
Similar trials for Severe Aplastic Anemia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05012111's enrollment target sits among peer trials
1,000 1st of 16 higher than 16 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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