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Acute-graft-versus-host DiseasePhase 1/2NCT05123040

Ruxolitinib, Human Chorionic Gonadotropin (uhCG/EGF), and Dose De-escalated Corticosteroids

A Phase 1/2 study testing Ruxolitinib 10 MG Oral Tablet, hCG and Corticosteroids in people with Acute-graft-versus-host Disease, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This multi-center center phase I/II study to establish the lowest possible recommended phase 2 dose (RP2D) of corticosteroids in conjunction with ruxolitinib and uhCG/EGF (a novel combination) for high-risk aGVHD. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Acute-graft-versus-host Disease
What is being tested?
Ruxolitinib 10 MG Oral Tablet, hCG and Corticosteroids
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
5 Jun 2023to 27 Feb 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • HCT recipients over 12 years of age within the first 7 days of initial treatment of high-risk aGVHD, defined as:
  • Newly diagnosed Minnesota high-risk aGVHD -OR-
  • Newly diagnosed Minnesota standard risk aGVHD with plasma amphiregulin ≥ 33 pg/ml tested at the UMN Cytokine Reference Lab. For amphiregulin lab ordering information, see Fairview Lab Guide: http://labguide.fairview.org/showtest.asp?testid=6766\&format=long -OR-
  • Newly diagnosed Minnesota standard risk aGVHD Ann Arbor 3 biomarkers tested by Viracor. For ordering information, see: https://www.viracor-eurofins.com/test-menu/403572p-agvhd-symptomatic- onset-algorithm/
  • Renal: Serum 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).

Who cannot join

  • Progressive malignancy
  • Uncontrolled bacterial, fungal, parasitic, or viral infection at initiation of protocol treatment
  • Unwilling or unable to stop supplemental sex hormone therapy (estrogen, progesterone, and/or testosterone preparations)
  • Unwilling or unable to stop GnRH antagonists, aromatase inhibitors, or anti-androgens
  • History of a hormone responsive malignancy
  • Current thromboembolic disease requiring full-dose anticoagulation - patients receiving pharmacologic prophylaxis for thromboembolic disease will be eligible
Show all 9Show fewer
  • Active or recent (within prior 3 months) thrombus, irrespective of anticoagulation status
  • Pregnancy
  • Women or men of childbearing potential unwilling to take adequate precautions to avoid unintended pregnancy from the start of protocol treatment through 30 days after the last treatment

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Masonic Cancer Center at University of Minnesota

    Minneapolis, Minnesota, 55455

Other trials in United States

About this study

Official title: Ruxolitinib, Human Chorionic Gonadotropin (uhCG/EGF), and Dose De-escalated Corticosteroids for Treatment of Minnesota High-Risk Acute GVHD (aGVHD): A Phase I/II Study

This multi-center center phase I/II study to establish the lowest possible recommended phase 2 dose (RP2D) of corticosteroids in conjunction with ruxolitinib and uhCG/EGF (a novel combination) for high-risk aGVHD.

This is a single arm study designed to determine the lowest dose of corticosteroids required (toxicity endpoint) without impairing GVHD complete response or partial response (CR/PR) at day 28 when given in conjunction with uhCG/EGF and ruxolitinib.

After completion of the corticosteroid dose finding, the final dose will be carried forward into a two-stage phase II extension trial to confirm safety and make a preliminary determination of efficacy of this novel drug combination for high-risk aGVHD.

Being tested
Ruxolitinib 10 MG Oral Tablet, hCG, Corticosteroids
Keywords
aGVHD

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ruxolitinib 10 MG Oral TabletDrug

    By mouth twice daily through day 56, then tapered

  • hCGDrug

    Also called Pregnyl

    2,000 units/m2 SQ every other day x 3 doses, followed by twice weekly x 14 doses

  • CorticosteroidsDrug

    Also called Prednisone, IV methylprednisolone

    Dose level 1 (starting dose) = 1 mg/kg

    • Dose level 2 = 0.5 mg/kg
    • Dose level 3 = 0.25 mg/kg
    • Dose level 4 = 0.1 mg/kg
    • Dose level 5 = 0 mg/kg
    • If dose level 1 is determined to be below the Recommended Phase 2 Dose (RP2D), the dose will be escalated:
    • Dose level -1 = 1.5 mg/kg
    • Dose level -2 = 2 mg/kg
Study groups in NCT05123040
Study groupRoleWhat they receive
Ruxolitinib;hCG (Pregnyl®) ;CorticosteroidsExperimental* Ruxolitinib 10 mg by mouth twice daily (with dose adjustments as indicated) through day 56, followed by taper * hCG (Pregnyl®) 2,000 units/m2 SQ every other day x 3 doses, followed by twice weekly x 14 doses (total 17 doses through day 56) * Corticosteroids (Prednisone, or IV methylprednisolone equivalent) * Dose level 1 (starting dose) = 1 mg/kg * Dose level 2 = 0.5 mg/kg * Dose level 3 = 0.25 mg/kg * Dose level 4 = 0.1 mg/kg * Dose level 5 = 0 mg/kg
Receives: Drug: Ruxolitinib 10 MG Oral Tablet; Drug: hCG; Drug: Corticosteroids

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Recommend the lowest possible dose for Phase II of corticosteroids when given in combination with ruxolitinib and uhCG/EGF in pediatric based on DLT frequency

    Plan report patients proportions and their 95% confidence intervals of paitents who experience dose limiting toxicity. Determine best dose based on DLT criteria by CTCAE v5.0

    • Thrombosis requiring anticoagulation
    • Ascites (grade 3-5)
    • Ovarian hyperstimulation syndrome

    Measured: 28 days after therapy

  2. Best response of treatment in adult and children

    proportions of complete, partial, mixed, and no response among surviving patients at days 28 after initiation of protocol therapy in pediatric and adult patients with Minnesota high-risk aGVHD

    Measured: 28 days after therapy

Secondary outcomes (9)

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0

    Report any type of adverse event caused by a drug cause by dose of steroids in combination of ruxolitinib and uhCG/EGF.

    Due to the complex medical condition of the GVHD patient, monitoring for adverse events will focus on the following events beginning with the date consent is signed and continuing for 30 days after the subject has completed or discontinued from the study or has taken last dose of the study drug.

    • Rehospitalization
    • Death
    • Hematologic (grade 3-5 cytopenia)
    • Infections (grade 3-5)
    • Hyperglycemia (grade 3-5)
    • Steroid myopathy (grade 3-5)

    Measured: 30 days after treatment

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

    Find proportion of incidence of acute GVHD

    Measured: 28 days after treatment

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

    Find proportion of incidence of acute GVHD

    Measured: 56 days after treatment

  4. Compare the rate of treatment failure for acute GVHD after initiation of protocol therapy to historical controls

    Compare count of treatment failure to other number of failures in other historical protocols

    Measured: 28 days after treatment

  5. Compare the rate of treatment failure for acute GVHD after initiation of protocol therapy to historical controls

    Compare count of treatment failure to other number of failures in other historical protocols

    Measured: 56 days after treatment

  6. To assess patient quality of life on study

    Have participants take an overall survival survey

    Measured: 6 month after treatment

  7. Determine 1-year overall survival

    Provide proportions and their 95% confidence intervals of patients still alive at one year post-treatment

    Measured: 1 year post treatment

  8. Non-relapse mortality (death without recurrent or progressive disease after allo-HSCT)

    Provide proportions and their 95% confidence intervals of patients who expedience a non-relapse mortality.

    Measured: 1 year post treatment

  9. Collect blood samples and rectosigmoid biopsies for future correlative studies

    Give a count of the number of patients who had blood and rectosigmoid biopsies

    Measured: 1 year after treament

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Nov 2021
  2. Study start5 Jun 2023
  3. Primary completion27 Feb 2026
  4. Study completion27 Feb 2026

Why it stopped: Sponsor terminated financial support

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05123040
Sponsor's protocol ID
2020LS098
Other IDs
MT2020-28
Status last verified
Mar 2026
First submitted
3 Nov 2021
Last updated
24 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Ruxolitinib 10 MG Oral Tablet, hCG and Corticosteroids in people with Acute-graft-versus-host Disease, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 24 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Sponsor terminated financial support). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Recommend the lowest possible dose for Phase II of corticosteroids when given in combination with ruxolitinib and uhCG/EGF in pediatric based on DLT frequency (time frame: 28 days after therapy). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05123040.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2026.

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