A Phase 2, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of KD025 in Subjects with Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy (The ROCKstar Study)

Protocol # :
Chronic Graft-versus-host-disease
Disease Sites
Other Hematopoietic
Non-Hodgkin's Lymphoma
Hodgkin's Lymphoma
Multiple Myeloma
Lymphoid Leukemia
Myeloid and Monocytic Leukemia
Leukemia, other
Principal Investigator
DeFilipp, Zachariah
Site Investigator
Cutler, Corey, S.
Site Research Nurses
Beshere, Gianna
Graham, Kristen, A.
Halloran, Elizabeth
More, Avnee, P.

Trial Description

This is a Phase 2, randomized, multicenter study to evaluate the efficacy and safety of KD025
in subjects with Chronic Graft Versus Host Disease (cGVHD) after at least 2 prior lines of
systemic therapy

Eligibility Requirements

Inclusion Criteria:

1. Male and female subjects at least 12 years of age who have had allogenic hematopoietic
cell transplant (HCT).

2. Previously received at least 2 and not more than 5 lines of systemic therapy for cGVHD

3. Receiving glucocorticoid therapy with a stable dose over the 2 weeks prior to

4. Have persistent cGVHD manifestations and systemic therapy is indicated

5. Karnofsky Performance Score of ≥ 60 (if aged 16 years or older); Lansky Performance
Score of ≥ 60 (if aged < 16 years)

6. Weight ≥ 40kg

Exclusion Criteria:

1. Subjects has not been on a stable dose / regimen of systemic cGVHD treatments for at
least 2 weeks prior to screening. (Note: Concomitant corticosteroids, calcineurin
inhibitors, sirolimus, MMF, methotrexate, rituximab, and extracorporeal photophoresis
(ECP) are acceptable. Systemic investigational GVHD treatments are not permitted).

2. Histological relapse of the underlying cancer or post-transplant lymphoproliferative
disease at the time of screening.

3. Current treatment with ibrutinib. Prior treatment with ibrutinib is allowed with a
washout of at least 28 days prior to randomization.