Imatinib Mesylate and Mycophenolate Mofetil for Steroid-Refractory Sclerotic/Fibrotic cGVHD in Children

NCT01898377 | Terminated Phase 2

• 1 other identifier: SNUCH-1301
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

In this study we will combine mycophenolate mofetil and imatinib mesylate to treat steroid-refractory sclerotic/fibrotic type chronic graft-versus-host disease (GVHD) to see the response rate and to find the safety of combination.

Conditions

Chronic Graft-versus-host Disease

Keywords

Children; Chronic graft-versus-host disease

Outcome Measures

Primary Outcomes (1)

• Overall (complete and partial) response rate

  Response evaluation will be performed every 3 months during the treatment by comprehensive response criteria based on NIH criteria. The complete and partial response categories apply only to organs that have measurable and reversible GVHD-related abnormalities at baseline. * Complete response (CR): Resolution of all signs and symptoms of chronic GVHD * Partial response (PR) : Improvement (at least 1 clinical score reduction, see Appendix 2) in 1 or more organs of involvement and no evidence of worsening in any organ * Objective response (OR): Either CR or PR

  1 year

Secondary Outcomes (4)

• Evaluate the safety profile of MMF plus imatinib mesylate

  1 year

• Evaluate the quality of life (QOL)

  1 year

• Discontinuation of steroid

  1 year

• Overall survival rate

  1 year

Study Arms (1)

Imatinib mesylate, Mycophenolate mofetil

EXPERIMENTAL

MMF 15-20mg/kg (Max 1 g) bid + Imatinib mesylate qd * Dose of imatinib : starting dose 260 mg/m2/d (Max. 400 mg) * Imatinib dose adjustment : Dose is adjusted according to the guidelines if there is serious adverse event, toxicity, or intolerance.

Drug: Imatinib mesylate, Mycophenolate mofetil

Interventions

Imatinib mesylate, Mycophenolate mofetil DRUG

Also known as: Glivec, Cellcept

Imatinib mesylate, Mycophenolate mofetil

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients must have a diagnosis of chronic GVHD with fibrotic/scleroderma-like features. This diagnosis can be made clinically or by histopathology.
• Patients must have active disease with at least one of the following manifestations: skin sclerosis, symptomatic bronchiolitis obliterans, extensive lung fibrosis, pathologically demonstrated visceral fibrotic involvement of the gut.
• Patients with corticosteroid refractory or dependant cGVHD are eligible. Steroid-refractory chronic GVHD is defined as chronic GVHD of sustained severity during the last full month during which the patients received the equivalent of prednisone 0.5 mg/kg or more per day or 1 mg/kg or more every other day.
• Age under 21 years old

You may not qualify if:

• Patients who have had chemotherapy, radiotherapy within 4 weeks prior to entering the study.
• Patients who have not recovered from adverse events.
• Prior treatment with imatinib mesylate or other tyrosine kinase inhibitor after the date of transplant.
• Patients on pregnancy or lactating

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Children's Hospital

Seoul, Chongno-gu, South Korea

Location

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

Imatinib Mesylate; Mycophenolic Acid

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Intervention Hierarchy (Ancestors)

Benzamides; Amides; Organic Chemicals; Benzoates; Acids, Carbocyclic; Carboxylic Acids; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pyrimidines; Caproates; Acids, Acyclic; Fatty Acids; Lipids

Study Officials

• Hyoung Jin Kang, MD, Ph.D

  Seoul National University Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2013
• First Posted: July 12, 2013
• Study Start: August 1, 2013
• Primary Completion: February 14, 2018
• Study Completion: February 14, 2018
• Last Updated: October 23, 2020

Record last verified: 2020-10

Locations

KR (1)