NCT04098445

Brief Summary

Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Early pulmonary injury includes diffuse alveolar hemorrhage (DAH), thrombotic microangiopathy (TMA) interstitial pneumonitis (IPS) and infection, while later, bronchiolitis obliterans is a complication of chronic GVHD associated with severe morbidity and mortality. Improved diagnosis and treatment of pulmonary complications are urgently needed as survival after HSCT improves, and as HSCT is increasingly used for non-malignant disorders such as sickle cell disease. Currently, there are large and important gaps in the investigator's knowledge regarding incidence, etiology and optimal treatment of pulmonary complications. Moreover, young children unable to perform spirometry are often diagnosed late, and strategies for monitoring therapeutic response are limited. This is a prospective multi-institutional cohort study in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (alloHSCT). Assembly of a large prospective uniformly screened cohort of children receiving HSCT, together with collection of biological samples, will be an effective strategy to identify mechanisms of lung injury, test novel diagnostic strategies for earlier diagnosis, and novel treatments to reduce morbidity and mortality from lung injury after transplant.

Trial Health

77
On Track

Trial Health Score

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

Enrollment
2,000

participants targeted

Target at P75+ for all trials

Timeline
125mo left

Started Sep 2021

Longer than P75 for all trials

Geographic Reach
1 country

9 active sites

Status
recruiting

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress31%
Sep 2021Sep 2036

First Submitted

Initial submission to the registry

September 19, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 23, 2019

Completed
2 years until next milestone

Study Start

First participant enrolled

September 8, 2021

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2026

Expected
10 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2036

Last Updated

July 15, 2025

Status Verified

July 1, 2025

Enrollment Period

5 years

First QC Date

September 19, 2019

Last Update Submit

July 13, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

  • Incidence of Lung Injury after HSCT

    Participants will be assessed for lung injury at 24 months after HSCT

    24 months after HSCT

Study Arms (1)

Pediatric and young adult HSCT recipients

Prospective multi-institutional cohort study in pediatric patients undergoing allogeneic (alloHSCT) or autologous hematopoietic stem cell transplantation (autoHSCT).

Eligibility Criteria

AgeUp to 24 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

The study participants will include eligible patients undergoing allogeneic or autologous HSCT at participating institutions.

You may qualify if:

  • Subjects ≤ 24 years of age undergoing allogeneic or autologous HSCT.

You may not qualify if:

  • Subjects over 24 years of age.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

University of California San Francisco

San Francisco, California, 94158, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Dana-Farber Cancer Institute/Boston Children's Hospital

Boston, Massachusetts, 02215, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Baylor College of Medicine/Texas Children'S Hospital

Houston, Texas, 77030, United States

RECRUITING

Seattle Children'S Hospital

Seattle, Washington, 98105, United States

RECRUITING

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

RECRUITING

Related Publications (1)

  • Koo J, Cooper R, Edwards SL, Lane A, Loveless SK, Strecker L, Lake KE, Myers KC, Towe C, Patti J, Walkup LL, Wikenheiser-Brokamp KA, MacMillan ML, Lacher P, Griffin T, Tekman M, Cisneros GS, Wu K, Zinter MS, Urrego FA, Baker KS, Abts MF, Ballard S, Freedman JL, Caraballo A, Young LR, Josephson MB, Allen JL, Camburn DM, Doherty EE, Azamian MS, Arredondo M, Silva-Carmona M, Lehmann LE, Wong W, Gaffin JM, McAlpine W, Li M, Goldfarb SB, Woods JC, Davies SM. High Prevalence of Abnormal Baseline Lung Function in Pediatric and Young Adult Hematopoietic Stem Cell Transplant Recipients: A Report from the TRANSPIRE Study. Pediatr Blood Cancer. 2025 Oct;72(10):e31916. doi: 10.1002/pbc.31916. Epub 2025 Jul 21.

MeSH Terms

Conditions

Thrombotic MicroangiopathiesLung Diseases, InterstitialBronchiolitis Obliterans

Condition Hierarchy (Ancestors)

ThrombocytopeniaBlood Platelet DisordersHematologic DiseasesHemic and Lymphatic DiseasesCytopeniaLung DiseasesRespiratory Tract DiseasesBronchiolitisBronchitisBronchial DiseasesLung Diseases, Obstructive

Study Officials

  • Stella Davies, MBBS, PhD, MRCP

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephanie Edwards, BSN, RN

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 19, 2019

First Posted

September 23, 2019

Study Start

September 8, 2021

Primary Completion (Estimated)

September 1, 2026

Study Completion (Estimated)

September 1, 2036

Last Updated

July 15, 2025

Record last verified: 2025-07

Locations