Phase 2
N=53
Study of Pregnyl as Adjunct Therapy for High-Risk or Refractory Acute GVHD
Graft vs Host Disease
Bottom Line
View on ClinicalTrials.gov: NCT02525029 ↗Enrolled (actual)
53
Serious AEs
47.2%
Results posted
Dec 2023
Primary outcome: Primary: Phase I: MTD (USP hCG) — 2000; 2000; 5000 Pregnyl dosage (USP hCG)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Pregnyl® (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- May 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Phase I: MTD (USP hCG) |
2000; 2000; 5000 | — |
| PRIMARY Phase I: MTD (pg EGF/m^2) |
14000; 14000; 34000 | — |
| PRIMARY Number of Patients With Complete Response |
2; 2; 4; 7; 1; 3 | — |
| PRIMARY Number of Patients With Partial Response |
0; 0; 0; 1; 0; 0 | — |
| PRIMARY Number of Patients With Mixed Response |
0; 0; 1; 2; 0; 0 | — |
| PRIMARY Number of Patients With No Response |
0; 0; 4; 3; 0; 1 | — |
| SECONDARY Number of Participants With Adverse Events as a Measure of Safety and Feasibility of hCG Supplementation With Pregnyl® |
1; 1; 3; 6; 1; 2 | — |
| SECONDARY Number of Participants With Incidence of aGVHD Flare |
0; 0; 2; 8; 0; 1 | — |
| SECONDARY Number of Participants With Incidence of aGVHD Flare |
0; 0; 2; 8; 0; 1 | — |
| SECONDARY Rate of Participants Who Fail Treatment at Day 28 |
0; 0; 8; 0; 0; 1 | — |
| SECONDARY Rate of Participants Who Fail Treatment at Day 56 |
0; 0; 2; 9; 0; 2 | — |
Summary
The purpose of this study is to determine the maximum tolerated dose (MTD) of Pregnyl® when given in combination with standard immunosuppressive therapy in pediatric and adult patients with high-risk (Arm 1) or refractory/dependent (Arm 2) aGVHD.
Eligibility Criteria
Inclusion Criteria
- Acute graft versus host disease (GVHD) fitting one of the following categories:
- High-Risk aGVHD (ARM 1): Pediatric or adult (ages 12-76 years) HCT recipients with high-risk acute GVHD, as determined by the refined MN acute GVHD risk score: http://z.umn.edu/MNAcuteGVHDRiskScore OR high risk on the basis of blood biomarkers (Ann Arbor Score 3 or amphiregulin ≥ 33 pg/ml) or
- Steroid-Refractory aGVHD (ARM 2): Pediatric or adult (ages 12-76 years) HCT recipient with grade II-IV steroid refractory or steroid-dependent acute GVHD, defined as any one of the following:
- No response of acute GVHD after at least 4 days of systemic corticosteroids of at least 2 mg/kg prednisone or equivalent
- Progression of acute GVHD within 3 days of systemic corticosteroids of at least 2 mg/kg prednisone or equivalent
- Failure to improve to at least grade II acute GVHD after 14 days of systemic corticosteroids, with initial doses of at least 2 mg/kg prednisone or equivalent
- Flare of acute GVHD of at least grade II/IV severity despite tapering dose of steroids being > 0.5 mg/kg/day.
- Adequate organ function at study enrollment defined as:
- Renal: 1.73m2Serum creatinine ≤2.5x upper limit of normal (ULN)
- Cardiac: Left ventricular ejection fraction (LVEF) ≥ 35%
- Voluntary written consent (adult or parent/guardian with minor assent for 12 through 17 year olds)
Exclusion Criteria
- Progressive malignancy
- Diagnosis of a hormone responsive malignancy
- Uncontrolled infection at initiation of protocol treatment
- Current thromboembolic disease requiring full-dose anticoagulation - patients receiving pharmacologic prophylaxis for thromboembolic disease will be eligible
- Active or recent (within prior 3 months) thrombus, irrespective of anticoagulation status
- Pregnancy as assessed on baseline blood hCG level
- Unwilling or unable to stop supplemental sex hormone therapy (estrogen, progesterone, and/or testosterone preparations)
- Women or men of childbearing potential unwilling to take adequate precautions to avoid pregnancy from the start of protocol treatment through 28 days after the last treatment
Screening Inclusion Criteria:
- Pediatric or adult (ages 0-76 years) HCT recipients
- Suspected high risk GVHD
- Voluntary written consent (adult or parent/guardian with minor assent for 12 through 17 year olds)
Data sourced from ClinicalTrials.gov (NCT02525029). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.