Early Detection and Management of Bronchiolitis Obliterans Syndrome Following Pediatric Hematopoietic Stem Cell Transplantation

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT03072849
• Date of registration: 02/03/2017
• Prospective Registration: No
• Primary sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Public title: Early Detection and Management of Bronchiolitis Obliterans Syndrome Following Pediatric Hematopoietic Stem Cell Transplantation
• Scientific title: Early Detection and Management of Bronchiolitis Obliterans Syndrome Following Pediatric Hematopoietic Stem Cell Transplantation
• Date of first enrolment: April 2015
• Target sample size: 40
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03072849
• Study type: Observational
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Jennifer Schneiderman, MD, MS

• Name: Jennifer Schneiderman, MD, MS

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Diagnosis: Patients undergoing myeloablative allogenic hematopoietic stem cell
transplant for any indication (both malignant and non-malignant) are eligible.

2. Graft: Eligible patients will have one or more of the following donor stem cell
sources:

- Bone marrow

- Placental blood (umbilical cord blood)

- Cytokine mobilized peripheral blood

3. Eligible patients will have one of the following sources of donor stem cells:

- HLA matched family member

- Partially matched family member (mismatched for a single HLA locus at A, B, C or
DR)

- Fully HLA matched or partially mismatched unrelated marrow or peripheral blood
stem cells (per institutional donor selection standards)

- HLA matched or partially mismatched (at least 4/6 match at A, B, DR) cord blood.

4. Conditioning Regimen: Patients expecting to receive any type of myeloablative HSCT
conditioning regimen are eligible.

5. Prior therapies: Patients undergoing stem cell transplant of any kind.

6. Required laboratory parameters: Patients able to adequately perform pulmonary function
testing per ATS/ERS guidelines, as determined by the enrolling investigator and
trained respiratory therapists.

7. The patient and/or the patient's legally authorized guardian must acknowledge in
writing that consent to become a study subject has been obtained in accordance with
the institutional policies approved by the U.S. Department of Health and Human
Services. Informed consent must be signed prior to registration on study.

Exclusion Criteria:

1. Subjects with a previous solid organ transplant.

2. Recurrent or progressive malignancy requiring anti-cancer therapy.

3. Subjects with evidence of underlying obstructive pulmonary disease prior to transplant
(clinical history of asthma or baseline FEV1 <80% predicted with FEV1/FVC <80%).

4. Known history of allergy or intolerance to Montelukast, Zafirleukast, Azithromycin,
Erythromycin, Clarithromycin, Prednisone, or Sirolimus.

5. Chronic supplemental oxygen requirement or hypoxemia <92% SpO2.

6. Clinical asthma (variable and recurrent symptoms of airflow obstruction and airway
hyper-responsiveness).

7. Pregnancy or nursing: All females of childbearing age must have a negative serum or
urine pregnancy test <7 days before study drug administration.

8. Chronic treatment with any inhaled steroid for >1 month in past three months.

9. Treatment with montelukast or zafirukast for >1 month in past three months.

10. Treatment with systemic steroids for >1 month in past three months.

11. Treatment with any FDA non-approved study medication within the past four weeks. Off
label treatment with FDA approved medication is allowed.

12. Evidence of any viral, bacterial, or fungal infection involving the lung and not
responding to appropriate treatment.

13. Inability to perform pulmonary function testing (PFT), as determined by the enrolling
investigator or PFT lab.

14. Any condition that, in the opinion of the enrolling investigator, would interfere with
the subject's ability to comply with the study requirements.



Age minimum: 6 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Bronchiolitis Obliterans Syndrome

Intervention(s)

Procedure: Pulmonary function testing

Drug: FAM Therapy

Primary Outcome(s)

Lung function [Time Frame: 2 years]

Survival [Time Frame: 2-4 years]

Secondary Outcome(s)

Risk factor assessment [Time Frame: 2 years]

Secondary ID(s)

SCT 1214 BOS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors