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Protocol Details

U01 Cooperative Assessment of Late Effects for SCD Curative Therapies (COALESCE)

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

000697-H

Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 18 Years
Max Age: 65 Years

Referral Letter Required

Yes

Population Exclusion(s)

None

Keywords

Chip;
Immunosuppression;
Malignant Neoplasms;
Posttransplant Cyclophosphamide;
Tp53 Mutations;
Hydroxyurea-Treated Sickle Cell Disease;
Natural History

Recruitment Keyword(s)

None

Condition(s)

Sickle Cell Disease;
Pulmonary Disease;
Renal Disease;
Heart Disease

Investigational Drug(s)

None

Investigational Device(s)

None

Intervention(s)

None

Supporting Site

National Heart, Lung, and Blood Institute

Background:

Sickle cell disease (SCD) is an inherited blood disorder. It can damage the heart, lungs, and kidneys and can cause serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD; but, over time, these cures may increase the risk of organ damage and cancers. More research is needed to understand the long-term effects of treatments that cure SCD.

Objective:

To observe how different treatments for SCD affect the health of the heart, lungs, and kidneys over the long term.

Eligibility:

People aged 4 to 65 years with SCD who have been treated with either blood stem cell transplant or with standard disease-modifying therapy.

Design:

Participants will be followed in this natural history study for 5 years. Their annual visits to their regular doctors will serve as research study visits.

Participant s own doctors will conduct tests as part of their standard care. Researchers will collect data from these tests. Researchers may also review participants prior medical records.

Data to be used in research may come from these tests:

Physical exam. This may include heart rate, breathing rate, height, and weight.

Breathing tests (spirometry). These are given before and after a participant takes a rescue medication to see how well the lungs are functioning.

Heart tests (echocardiogram). These are images of the heart captured through sound waves.

Pulse oximetry. A sensor is placed on the finger to measure the amount of oxygen in the blood.

Participants will complete questionnaires. Topics will include their personal and family history, social environment, employment history, and education. At screening, they will be asked about their willingness to participate in research.

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Eligibility

INCLUSION CRITERIA:

All Aims:

Confirmed laboratory diagnosis of SCD;

Ability to give informed consent;

Ability to provide pre- and post-curative therapy data and/or biospecimens to meet study aims

Treated with either one HSCT (data collected after second curative therapy will be excluded),or with standard disease-modifying therapy.

Aim 1a: Ages 4 to 17 years

Aim 1b, 2, 3 & 4: Ages 18 to 65 years

Aim 4: Ages 4 to 65 years

Study Population:

Curative therapy cohort: Ages 4 - 65 years; receipt of HSCT or gene therapy/editing 2003 - 2026 at one of the 5 study sites.

Retrospective: 2003 - 2021

Prospective: 2021 - 2026

Standard therapy control cohort: Ages 4 - 65 years; receipt of disease-modifying therapy only between 2003 - 2026; followed as part of the VUMC-based Tennessee Never Lost Cohort.

EXCLUSION CRITERIA:

Participants judged to be non-compliant based on previous experience in terms of clinic appointments and following advice.


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Citations:

Seminog OO, Ogunlaja OI, Yeates D, Goldacre MJ. Risk of individual malignant neoplasms in patients with sickle cell disease: English national record linkage study. J R Soc Med. 2016 Aug;109(8):303-9. doi: 10.1177/0141076816651037. Epub 2016 Jun 20. PMID: 27325377; PMCID: PMC4976723.

Brunson A, Keegan THM, Bang H, Mahajan A, Paulukonis S, Wun T. Increased risk of leukemia among sickle cell disease patients in California. Blood. 2017 Sep 28;130(13):1597-1599. doi: 10.1182/blood-2017-05-783233. Epub 2017 Aug 22. PMID: 28830890; PMCID: PMC5620417.

Gondek LP, Zheng G, Ghiaur G, DeZern AE, Matsui W, Yegnasubramanian S, Lin MT, Levis M, Eshleman JR, Varadhan R, Tucker N, Jones R, Gocke CD. Donor cell leukemia arising from clonal hematopoiesis after bone marrow transplantation. Leukemia. 2016 Sep;30(9):1916-1920. doi: 10.1038/leu.2016.63. Epub 2016 Mar 15. PMID: 26975880; PMCID: PMC5014666.

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Contacts:

Principal Investigator

Referral Contact

For more information:

Courtney F. Joseph, M.D.
National Heart, Lung and Blood Institute (NHLBI)
NIHBC 10 - CLINICAL CENTER BG RM 6N240A
10 CENTER DR
BETHESDA MD 20892
(301) 402-6496
courtney.fitzhugh@nih.gov

Julia M. Varga
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 6-5140
10 Center Drive
Bethesda, Maryland 20892
(301) 402-3595
julia.varga@nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1
ccopr@nih.gov

Clinical Trials Number:

NCT05153967

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