CompletedNCT02525029Updated Dec 14, 2023Results posted

Study of Pregnyl as Adjunct Therapy for High-Risk or Refractory Acute GVHD

A Phase 1/2 interventional study of Pregnyl® in Graft vs Host Disease, sponsored by Masonic Cancer Center, University of Minnesota. Completed at 1 site in United States. Open to participants aged Up to 76 Years. Per ClinicalTrials.gov, last updated 2023-12-14.

Sponsored by Masonic Cancer Center, University of Minnesota · Phase 1/2, Interventional, and Supportive care

Phase: Phase 1/2
Study type: Interventional
Enrollment: 53
Allocation: Non-randomized
Ages: Up to 76 Years
Sex: All

Study 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.

Conditions studied

Graft vs Host Disease

Browse trials for

Graft vs Host Disease 806

Keywords

GVHD

In context

Graft vs Host Disease

806 studies on the registry are indexed under Graft vs Host Disease; 138 are open to participants now.

This study's enrollment of 53 is above the median of 35 across 637 interventional studies indexed under Graft vs Host Disease.

Browse Graft vs Host Disease studies →

Lead sponsor

Masonic Cancer Center, University of Minnesota is the lead sponsor of 284 studies on the registry; 34 are open to participants now.

Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: Up to 76 Years
Sexes eligible: All
Accepts healthy volunteers: No

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

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)

Study design

Phase: Phase 1 / Phase 2
Primary purpose: Supportive care
Allocation: Non-randomized
Intervention model: Sequential assignment
Masking: None (open label)
Enrollment: 53 participants (actual)

Study arms

Experimental
Arm 1: Phase 1 Dose Level -2: 125 USP hCG/875 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 5 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 1: Phase I Dose Level -1: 250 USP hCG/1,750 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 5 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 5 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort have reached day 21 in order to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 5 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort have reached day 21 in order to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 5 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort have reached day 21 in order to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2A: Phase 1 Dose Level -2: 125 USP hCG/875 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2A: Phase I Dose Level -1: 250 USP hCG/1,750 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2A: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase 1 Dose Level -2: 125 USP hCG/875 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase I Dose Level -1: 250 USP hCG/1,750 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Standard of care immunosuppression, plus Pregnyl® (hCG supplementation) at assigned dose subcutaneously every other day for up to 7 doses of Pregnyl®; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. The 1st 2 patients will be enrolled in dose level 1. The next cohort of 2 patients will not begin treatment until all patients in the current cohort has reached day 21 to assess for dose limiting toxicity (DLT). Individual patient dose reductions: If a patient has a toxicity, the patient can drop down one dose level for the next injection and continue treatment.
Drug: Pregnyl®
Experimental
Arm 1: Phase 2 MTD
After completion of the dose finding trial for each arm, the final doses will be carried forward into a two-stage phase II extension trial to confirm safety and make a preliminary determination of the activity level for Arm 1 and Arm 2. If the phase I trial enrolls fewer than 13 patients at the MTD, we will employ Simon's Minmax two-stage design with the possibility to discontinue after the 1st stage if the response rate is low
Drug: Pregnyl®
Experimental
Arm 2A: MTD
After completion of the dose finding trial for each arm, the final doses will be carried forward into a two-stage phase II extension trial to confirm safety and make a preliminary determination of the activity level for Arm 1 and Arm 2. If the phase I trial enrolls fewer than 13 patients at the MTD, we will employ Simon's Minmax two-stage design with the possibility to discontinue after the 1st stage if the response rate is low
Drug: Pregnyl®
Experimental
Arm 2B: MTD
After completion of the dose finding trial for each arm, the final doses will be carried forward into a two-stage phase II extension trial to confirm safety and make a preliminary determination of the activity level for Arm 1 and Arm 2. If the phase I trial enrolls fewer than 13 patients at the MTD, we will employ Simon's Minmax two-stage design with the possibility to discontinue after the 1st stage if the response rate is low
Drug: Pregnyl®

Interventions

DrugPregnyl®
Standard of care immunosuppression, plus Pregnyl® at assigned dose subcutaneously every other day for up to 5 doses through day 7 Phase I: up to 7 dose levels of Pregnyl® will be tested; however dose levels -1 and -2 will be used only if dose level 1 proves too toxic. Study will use Continual Reassessment Method (CRM) to establish a maximum tolerated dose (MTD). Step -2: 125 USP hCG/875 pg EGF/m2 Step -1: 250 USP hCG/1,750 pg EGF/m2 Step 1 (start): 500 USP hCG/3,500 pg EGF/m2 Step 2: 1,000 USP hCG/7,000 pg EGF/m2 Step 3: 2,000 USP hCG/14,000 pg EGF/m2 Step 4 (Arm 2b only): 3,500 USP hCG/24,500 pg EGF/m2 Step 5 (Arm 2b only): 5,000 USP hCG/35,000 pg EGF/m Phase II: the dose of Pregnyl will be that identified as the MTD during phase I.
Also known as: Human chorionic gonadotropin (hCG)

What researchers measure

Primary outcomes

Phase I: MTD (USP hCG)
Maximum tolerated dose (MTD) of Pregnyl® when given with standard immunosuppressive therapy in pediatric and adult patients with high-risk or refractory acute graft-versus-host disease (aGVHD). Dosage measured as (USP hCG/pg EGF/mm\^2).
Time frame: Day 14 after initiation of protocol therapy
Phase I: MTD (pg EGF/m^2)
Maximum tolerated dose (MTD) of Pregnyl® when given with standard immunosuppressive therapy in pediatric and adult patients with high-risk or refractory acute graft-versus-host disease (aGVHD). Dosage measured as (USP hCG/pg EGF/m\^2).
Time frame: Day 14 after initiation of protocol therapy
Number of Patients With Complete Response
Percentage of complete response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. Complete response is defined as a Stage of 0 for all organs with no additional intervening therapy for their GVHD.
Time frame: Day 28 after initiation of protocol therapy
Number of Patients With Partial Response
Percentage of partial response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. Partial response is defined as improvement by at least 1 stage in all involved organs without progression in others with no additional intervening therapy for their GVHD.
Time frame: Day 28 after initiation of protocol therapy
Number of Patients With Mixed Response
Percentage of mixed response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. Mixed Response is defined as improvement in one organ with deterioration in another organ manifesting symptoms of GVHD or development of symptoms of GVHD in a new organ.
Time frame: Day 28 after initiation of protocol therapy
Number of Patients With No Response
Percentage of no response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. No response is defined as deterioration of any organ involved.
Time frame: Day 28 after initiation of protocol therapy

Secondary outcomes

Number of Participants With Adverse Events as a Measure of Safety and Feasibility of hCG Supplementation With Pregnyl®
Safety and feasibility of hCG supplementation with Pregnyl® in combination with standard immunosuppressive therapy in pediatric and adult patients with high-risk or refractory aGVHD.
Time frame: Day 70 after initiation of protocol therapy
Number of Participants With Incidence of aGVHD Flare
Incidence of acute GVHD flare after CR/PR requiring increase of steroids or other systemic treatment at day 28.
Time frame: Day 28 after initiation of protocol therapy
Number of Participants With Incidence of aGVHD Flare
Incidence of acute GVHD flare after CR/PR requiring increase of steroids or other systemic treatment at day 56.
Time frame: Day 56 after initiation of protocol therapy
Rate of Participants Who Fail Treatment at Day 28
Rate of treatment failure for acute GVHD at day 28 after initiation of protocol therapy to historical controls.
Time frame: Day 28 after initiation of protocol therapy
Rate of Participants Who Fail Treatment at Day 56
Rate of treatment failure for acute GVHD at day 56 after initiation of protocol therapy to historical controls.
Time frame: Day 56 after initiation of protocol therapy

Results

Posted Dec 14, 2023

Participant flow

Participant flow — Overall Study
Milestone	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m
Started	2	2	9	13	1	4	8	2	1	5	1	2	3
Completed	2	2	9	13	1	4	8	1	1	5	1	2	3
Not completed	0	0	0	0	0	0	0	1	0	0	0	0	0

Outcome measures

PrimaryPhase I: MTD (USP hCG)

Maximum tolerated dose (MTD) of Pregnyl® when given with standard immunosuppressive therapy in pediatric and adult patients with high-risk or refractory acute graft-versus-host disease (aGVHD). Dosage measured as (USP hCG/pg EGF/mm\^2).

Time frame:: Day 14 after initiation of protocol therapy
Reported as:: Number · Pregnyl dosage (USP hCG)

Phase I: MTD (USP hCG)
Pregnyl dosage (USP hCG)	1: High-Risk aGVHD	2a: Steroid-Dependent aGVHD	2b: Steroid-Refractory aGVHD
Phase I: MTD (USP hCG)	2000	2000	5000

PrimaryPhase I: MTD (pg EGF/m^2)

Time frame:: Day 14 after initiation of protocol therapy
Reported as:: Number · Pregnyl dosage (pg EGF/m^2)

Phase I: MTD (pg EGF/m^2)
Pregnyl dosage (pg EGF/m^2)	1: High-Risk aGVHD	2a: Steroid-Dependent aGVHD	2b: Steroid-Refractory aGVHD
Phase I: MTD (pg EGF/m^2)	14000	14000	34000

PrimaryNumber of Patients With Complete Response

Percentage of complete response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. Complete response is defined as a Stage of 0 for all organs with no additional intervening therapy for their GVHD.

Time frame:: Day 28 after initiation of protocol therapy
Reported as:: Number · participants

Number of Patients With Complete Response
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Patients With Complete Response	2	2	4	7	1	3	5	1	0	2	0	0	1

PrimaryNumber of Patients With Partial Response

Percentage of partial response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. Partial response is defined as improvement by at least 1 stage in all involved organs without progression in others with no additional intervening therapy for their GVHD.

Time frame:: Day 28 after initiation of protocol therapy
Reported as:: Number · participants

Number of Patients With Partial Response
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Patients With Partial Response	0	0	0	1	0	0	1	0	1	1	0	0	1

PrimaryNumber of Patients With Mixed Response

Percentage of mixed response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. Mixed Response is defined as improvement in one organ with deterioration in another organ manifesting symptoms of GVHD or development of symptoms of GVHD in a new organ.

Time frame:: Day 28 after initiation of protocol therapy
Reported as:: Number · participants

Number of Patients With Mixed Response
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Patients With Mixed Response	0	0	1	2	0	0	2	0	0	1	1	0	0

PrimaryNumber of Patients With No Response

Percentage of no response among surviving patients at day 28 after initiation of protocol therapy in pediatric and adult patients with high-risk (Arm 1) or refractory (Arm 2) aGVHD. No response is defined as deterioration of any organ involved.

Time frame:: Day 28 after initiation of protocol therapy
Reported as:: Number · participants

Number of Patients With No Response
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Patients With No Response	0	0	4	3	0	1	0	0	0	1	0	1	1

SecondaryNumber of Participants With Adverse Events as a Measure of Safety and Feasibility of hCG Supplementation With Pregnyl®

Safety and feasibility of hCG supplementation with Pregnyl® in combination with standard immunosuppressive therapy in pediatric and adult patients with high-risk or refractory aGVHD.

Time frame:: Day 70 after initiation of protocol therapy
Reported as:: Count of participants · Participants

Number of Participants With Adverse Events as a Measure of Safety and Feasibility of hCG Supplementation With Pregnyl®
Participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Participants With Adverse Events as a Measure of Safety and Feasibility of hCG Supplementation With Pregnyl®	1	1	3	6	1	2	5	2	1	3	1	2	1

SecondaryNumber of Participants With Incidence of aGVHD Flare

Incidence of acute GVHD flare after CR/PR requiring increase of steroids or other systemic treatment at day 28.

Time frame:: Day 28 after initiation of protocol therapy
Reported as:: Number · participants

Number of Participants With Incidence of aGVHD Flare
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Participants With Incidence of aGVHD Flare	0	0	3	5	0	0	1	0	0	0	0	1	1

SecondaryNumber of Participants With Incidence of aGVHD Flare

Incidence of acute GVHD flare after CR/PR requiring increase of steroids or other systemic treatment at day 56.

Time frame:: Day 56 after initiation of protocol therapy
Reported as:: Number · participants

Number of Participants With Incidence of aGVHD Flare
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Number of Participants With Incidence of aGVHD Flare	0	0	2	8	0	1	4	0	0	0	0	1	1

SecondaryRate of Participants Who Fail Treatment at Day 28

Rate of treatment failure for acute GVHD at day 28 after initiation of protocol therapy to historical controls.

Time frame:: Day 28 after initiation of protocol therapy
Reported as:: Number · participants

Rate of Participants Who Fail Treatment at Day 28
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Rate of Participants Who Fail Treatment at Day 28	0	0	8	0	0	1	0	1	0	3	0	1	1

SecondaryRate of Participants Who Fail Treatment at Day 56

Rate of treatment failure for acute GVHD at day 56 after initiation of protocol therapy to historical controls.

Time frame:: Day 56 after initiation of protocol therapy
Reported as:: Number · participants

Rate of Participants Who Fail Treatment at Day 56
participants	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m^2
Rate of Participants Who Fail Treatment at Day 56	0	0	2	9	0	2	4	1	0	2	1	1	1

Adverse events

Collected over 70 Days after first treatment. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	0/2 (0%)	1/2 (50%)	1/2 (50%)
Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	1/2 (50%)	1/2 (50%)	1/2 (50%)
Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	0/9 (0%)	1/9 (11.1%)	3/9 (33.3%)
Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	0/13 (0%)	2/13 (15.4%)	6/13 (46.2%)
Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	0/1 (0%)	1/1 (100%)	1/1 (100%)
Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	1/4 (25%)	2/4 (50%)	2/4 (50%)
Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	0/8 (0%)	4/8 (50%)	5/8 (62.5%)
Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	0/2 (0%)	2/2 (100%)	2/2 (100%)
Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	1/1 (100%)	1/1 (100%)	1/1 (100%)
Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	0/5 (0%)	5/5 (100%)	3/5 (60%)
Arm 2B: Phase 2 Dose Level 3 2,000 USP	1/1 (100%)	0/1 (0%)	1/1 (100%)
Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	1/2 (50%)	2/2 (100%)	2/2 (100%)
Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m	1/3 (33.3%)	3/3 (100%)	1/3 (33.3%)

Most frequent serious events

Showing 10 of 30

Most frequent serious events
Event	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m
blood and lympathic systemBlood and lymphatic system disorders	0/2	1/2	0/9	0/13	0/1	0/4	1/8	0/2	1/1	0/5	0/1	0/2	0/3
Atrial FibrillationCardiac disorders	0/2	0/2	0/9	0/13	1/1	0/4	0/8	0/2	0/1	1/5	0/1	0/2	0/3
Colonic perforationGastrointestinal disorders	0/2	0/2	0/9	1/13	1/1	0/4	0/8	0/2	0/1	0/5	0/1	0/2	0/3
Enterocolitis infectiousInfections and infestations	0/2	0/2	0/9	0/13	0/1	0/4	0/8	0/2	1/1	0/5	0/1	0/2	0/3
Infections and infestations - Other,Infections and infestations	0/2	0/2	0/9	0/13	0/1	0/4	0/8	2/2	0/1	0/5	0/1	0/2	0/3
SepsisInfections and infestations	0/2	0/2	1/9	0/13	0/1	0/4	0/8	0/2	1/1	0/5	0/1	2/2	0/3
AnorexiaMetabolism and nutrition disorders	0/2	0/2	0/9	0/13	1/1	0/4	0/8	1/2	0/1	0/5	0/1	0/2	0/3
Respiratory failureRespiratory, thoracic and mediastinal disorders	0/2	0/2	0/9	0/13	0/1	0/4	0/8	0/2	1/1	0/5	0/1	0/2	0/3
Lung infectionInfections and infestations	0/2	0/2	0/9	0/13	0/1	0/4	0/8	0/2	0/1	1/5	0/1	0/2	2/3
Febrile neutropeniaBlood and lymphatic system disorders	0/2	0/2	0/9	0/13	0/1	0/4	0/8	0/2	0/1	0/5	0/1	1/2	0/3

Most frequent other events

Showing 10 of 125

Most frequent other events
Event	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m
Febrile neutropeniaBlood and lymphatic system disorders	0/2	0/2	0/9	0/13	0/1	0/4	0/8	1/2	1/1	0/5	0/1	0/2	0/3
Atrial fibrillationCardiac disorders	0/2	0/2	1/9	0/13	1/1	0/4	0/8	1/2	0/1	0/5	0/1	0/2	0/3
PalpitationsCardiac disorders	0/2	0/2	0/9	0/13	1/1	0/4	0/8	0/2	0/1	0/5	0/1	0/2	0/3
Supraventricular tachycardiaCardiac disorders	0/2	0/2	0/9	0/13	1/1	0/4	0/8	0/2	0/1	0/5	0/1	0/2	0/3
Abdominal distensionGastrointestinal disorders	0/2	0/2	1/9	2/13	1/1	0/4	0/8	0/2	0/1	0/5	0/1	0/2	0/3
ColitisGastrointestinal disorders	0/2	0/2	0/9	0/13	1/1	0/4	0/8	0/2	0/1	0/5	0/1	0/2	0/3
ConstipationGastrointestinal disorders	1/2	0/2	0/9	0/13	1/1	0/4	0/8	0/2	0/1	0/5	0/1	1/2	0/3
Hemorrhoidal hemorrhageGastrointestinal disorders	0/2	0/2	0/9	0/13	0/1	0/4	0/8	0/2	0/1	0/5	1/1	0/2	0/3
FatigueGeneral disorders	0/2	0/2	1/9	0/13	0/1	1/4	0/8	2/2	0/1	0/5	0/1	0/2	0/3
Infections and infestations - Other, specifyInfections and infestations	0/2	0/2	1/9	1/13	0/1	0/4	1/8	0/2	0/1	0/5	1/1	0/2	1/3

Baseline characteristics

Age, Continuous
Age, Continuous(Years)	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m	Total
Median	46 (39 to 53)	64 (64 to 64)	60.5 (22 to 72)	60.5 (22 to 72)	57 (57 to 57)	61.5 (26 to 67)	61.5 (26 to 67)	56.5 (50 to 63)	63 (63 to 63)	64 (35 to 66)	64 (35 to 66)	61 (53 to 69)	50 (2 to 55)	61 (2 to 72)

Sex: Female, Male
Sex: Female, Male(Participants)	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m	Total
Female	0	1	3	3	0	1	3	1	0	1	1	0	0	14
Male	2	1	6	10	1	3	5	1	1	4	0	2	3	39

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m	Total
Hispanic or Latino	1	0	0	0	0	0	1	0	0	0	0	0	1	3
Not Hispanic or Latino	1	2	9	13	1	4	7	2	1	5	1	2	2	50
Unknown or Not Reported	0	0	0	0	0	0	0	0	0	0	0	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m	Total
American Indian or Alaska Native	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Asian	1	0	0	0	0	0	0	0	0	0	0	0	0	1
Native Hawaiian or Other Pacific Islander	0	0	0	1	0	0	0	0	0	0	0	0	0	1
Black or African American	0	0	1	0	0	1	0	0	0	0	0	0	0	2
White	1	1	8	12	1	3	7	2	1	5	0	2	3	46
More than one race	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Unknown or Not Reported	0	1	0	0	0	0	1	0	0	0	1	0	0	3

Region of Enrollment
Region of Enrollment(participants)	Arm 1: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 1: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 1: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 1: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2A: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2A: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 1 500 USP hCG/3,500 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 2 1,000 USP hCG/7,000 pg EGF/m^2	Arm 2B: Phase 1 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 2 Dose Level 3 2,000 USP hCG/14,000 pg EGF/m2	Arm 2B: Phase 1 Dose Level 4 3,500 USP hCG/24,500 pg EGF/m2	Arm 2B: Phase 1 Dose Level 5 5,000 USP hCG/35,000 pg EGF/m	Total
United States	2	2	9	13	1	4	8	2	1	5	1	2	3	53

Study locations

1 site

Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota 55455, United States

References and documents

Publications

Holtan SG, Hoeschen AL, Cao Q, Arora M, Bachanova V, Brunstein CG, Miller JS, Rashidi A, Slungaard A, Ustun C, Vercellotti GM, Warlick ED, Betts BC, El Jurdi N, He F, Chen C, Gandhi I, Wagner JE, Blazar BR, Jacobson PA, Shabaneh A, Wang J, Panoskaltsis-Mortari A, MacMillan ML, Weisdorf DJ. Facilitating resolution of life-threatening acute GVHD with human chorionic gonadotropin and epidermal growth factor. Blood Adv. 2020 Apr 14;4(7):1284-1295. doi: 10.1182/bloodadvances.2019001259. PubMed 32236525 ↗

Study documents

Protocol and statistical analysis plan · May 27, 2020
Informed consent form · Feb 20, 2020

Documents are hosted by the registry — open the source record to download them.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Dec 14, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT02525029
Lead sponsor: Masonic Cancer Center, University of Minnesota
Responsible party: Sponsor
First posted: Aug 17, 2015
Start date: Mar 2016
Primary completion: May 2021
Completion: Apr 2022
Results posted: Dec 14, 2023
Last update: Dec 14, 2023

Study contacts

Shernan Holtan, MD

principal investigator · University of Minnesota

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

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