Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Recruitment & Eligibility

Status: A

Sex: Not specified

Target Recruitment: 44

Inclusion Criteria

1. Part A: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic
therapy
2. Part B: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy, or subjects with new onset moderate or severe cGVHD and in need of systemic immunosuppression.
a. Subjects with new onset moderate or severe cGVHD must not have received previous systemic therapy for cGVHD with the exception of corticosteroids received within
72 hours prior to signing the informed consent form.
b. Subjects with newly diagnosed cGVHD may be receiving other immunosuppressants for the prophylaxis or treatment of acute GVHD, but if the subject is receiving prednisone for prophylaxis or treatment of acute GVHD it must be at or below 0.5 mg/kg/d at the time of enrollment.
3. History of allogeneic stem cell transplantation
4. Age
• Part A: =1 to <12 years of age at the time of enrollment
• Part B: =1 to <22 years of age at the time of enrollment
5. Written informed consent or parental or guardian permission and assent of children capable of understanding the nature of the study, per country specific or site-specific standards.
6. Ability of subject or, if a minor, parent/guardian to understand the purpose and risks of the study and to provide a signed and dated parental permission and authorization to use protected health information (in accordance with national and local subject privacy
regulations); willingness of child to provide an assent, if developmentally able to do so.
Are the trial subjects under 18? yes
Number of subjects for this age range: 44
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 11
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Presence of single organ genito-urinary involvement as the only manifestation of cGVHD.
Concurrent Conditions
2. Received an investigational agent within 28 days before enrollment.
3. Received donor lymphocyte infusion (DLI) within 56 days before enrollment.
4. Progressive underlying malignant disease or active post-transplant lymphoproliferative disease.
5. Ongoing anticoagulation treatment with warfarin or equivalent vitamin K antagonist.
6. History of other malignancy (not including the underlying malignancy that was the indication for transplant), with the following exceptions:
• Malignancy treated with curative intent and with no evidence of active disease present for more than 3 years prior to enrollment and felt to be at low risk for recurrence by treating physician
• Adequately treated non-melanomatous skin cancer or lentigo maligna melanoma without current evidence of disease
• Adequately treated cervical carcinoma in situ without current evidence of disease
7. History of major surgery within 28 days before enrollment or lack of full recovery from surgery.
8. Any life-threatening illness, medical condition, or organ system dysfunction that, in the investigator’s opinion, could compromise the subject’s safety or put the study outcomes at undue risk.
9. Female subject who is pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or within 3 months of the last dose of study drug. Male subject who plans to father a child while enrolled in this study or within 3 months after the last dose of study drug.
10. Unwilling or unable to participate in all required study evaluations and procedures.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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