NCT04436380

Brief Summary

Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for most people with SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is immunosuppressive treatment (IST). For people who are treated with IST, relapse can occur. If this happens, they can have HSCT or be re-treated with IST. The two most common IST regimes used for relapsed SAA are rabbit ATG (rATG) and alemtuzumab. Both rATG and alemtuzumab have similar response rates and survival rates. There is not much long-term data on people who need repeat IST treatment due to relapse. Researchers want to look at data from past studies to learn more. Objective: To compare the data of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST treatment. Eligibility: Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034, 05-H-0242, 03-H-0249, 03-H-0193, 00-H-0032, or 90-H-0146 Design: This study uses data from past studies. The participants in those studies have allowed their data to be used in future research. Researchers will review participants medical records. They will collect clinical data, such as notes, test results, and imaging scans. They will also collect the research data gathered as part of the original study. Researchers will enter the data into an in-house database. It is password protected. All data will be kept in secure network drives or in secure sites. Other studies may be added in the future....

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
173

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jun 2020

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 15, 2020

Completed
2 days until next milestone

First Submitted

Initial submission to the registry

June 17, 2020

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 18, 2020

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 15, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 15, 2023

Completed
Last Updated

March 16, 2023

Status Verified

March 1, 2023

Enrollment Period

2.7 years

First QC Date

June 17, 2020

Last Update Submit

March 15, 2023

Conditions

Severe Aplastic Anemia

Keywords

AlemtuzumabrATGNatural History

Outcome Measures

Primary Outcomes (1)

  • Assessment of long-term outcomes of relapsed patients who received repeat IST

    Characteristics and outcomes of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST

    Between the Period of 1990 to 2020

Secondary Outcomes (2)

  • Clinical predictors for the development of relapse

    Between the Period of 1990 to 2020

  • Rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required

    Between the Period of 1990 to 2020

Study Arms (1)

Relapsed SAA Patients

Patients with Severe Aplastic Anemia who Relapsed after Immunosuppressive Therapy

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Relapsed SAA Subjects who had a repeat IST

* This is a retrospective chart review. The investigators will review the medical records of adults and children with severe aplastic anemia (SAA) enrolled on immunosuppressive therapy (IST) treatment protocols in the NHLBI. Patients who opted out of future use of data on their prior studies will be excluded from this study.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

National Heart, Lung and Blood Institute (NHLBI)

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Officials

  • Emma M Groarke, M.D.

    National Heart, Lung, and Blood Institute (NHLBI)

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 17, 2020

First Posted

June 18, 2020

Study Start

June 15, 2020

Primary Completion

March 15, 2023

Study Completion

March 15, 2023

Last Updated

March 16, 2023

Record last verified: 2023-03

Locations

US(1)