Recruiting
Early Phase 1

HSC with Venetoclax

Sponsor:

Massachusetts General Hospital

Code:

NCT07358195

Conditions

Kidney Failure Chronic

Stem Cell Transplant

Stem Cell Transplant Complications

Tolerance

Hematologic Cancer

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

Interventions

Venetoclax

Hematopoietic Cell Transplantation Conditioning Regimen

Kidney Transplant

Hematopoietic Cell Transplantation

Study Details

Brief summary:

The primary objective is to assess the safety of the addition of venetoclax to reduced intensity conditioning for HLA-matched and haploidentical combined HSC and kidney transplantation as measured by stable full donor hematopoiesis and absence of CTCAE grade IV or V toxicity attributable to venetoclax.

Conditions

Kidney Failure Chronic

Stem Cell Transplant

Stem Cell Transplant Complications

Tolerance

Hematologic Cancer

Study ID

NCT07358195

Start date

Feb 4, 2026

Status verified date

Oct, 2026

Completion date

Dec, 2031

Anticipated

Primary completion date

Dec, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

Recipient Inclusion Criteria:

  • Patient ages 18-70
  • Underlying hematological malignancy which is deemed as being potentially curable with allogeneic bone marrow or PBSC transplantation by the BMT voting team.
  • Hematological malignancies include, but are not limited to: acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myelogenous leukemia (CML), chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), Hodgkin lymphoma, multiple myeloma (MM), myelodysplastic syndrome (MDS. Patient should be in a partial (PR) or complete remission (CR) at the time of the transplant.
  • Existence of an HLA-matched or haploidentical relative who passes standard donor evaluations for bone marrow and kidney donation
  • LVEF > 40% as measured by echocardiography or MUGA
  • FEV1, FVC, and DLCO > 50% of predicted as measured by standard PFTs
  • Total bilirubin < 2.0 (unless diagnosis of Gilbert's or hemolysis is made) and AST, ALT, alkaline phosphatase all < 5x institutions upper limit of normal
  • ABO compatibility in the host vs. graft direction
  • Men and women of reproductive potential must agree to use a reliable method of birth control during the treatment, and women should do so for a period of 1 year following the transplant.
  • Participants should be on dialysis or have a CrCl ≤ 35 ml/min
  • Life expectancy greater than 6 months
  • Recipient ability to understand and provide informed consent

Donor Inclusion Criteria:

  • HLA matched or haploidentical relative as defined by 3/6, 4/6, or 5/6 HLA-matched at HLA -A, -B, or -DRB1 who is 18-70 years of age
  • ECOG performance status 0 or 1
  • Excellent health per conventional pre-donor history (medical and psychosocial evaluation)

• Acceptable laboratory parameters (hematology in normal or near-normal range; liver function < 3 times the upper limit of normal and normal creatinine)
  • Compatible ABO blood group
  • Negative donor lymphocyte cross match
  • No positive testing for active viral infection (Hepatitis B, Hepatitis C, HIV)
  • Donor ability to understand and provide informed consent
  • Meets standard institutional criteria for both bone marrow or peripheral blood stem cell (PBSC) and kidney donation

Exclusion Criteria:

  • Active serious infection
  • Participation in other investigational drug use at the time of enrollment
  • Positivity for active infection with HIV, HCV, or HBV
  • ABO blood group incompatibility in the host-vs-graft direction

Study Design

Enrollment

3 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Recipient

Stem Cell and Kidney Transplant

Interventions

Venetoclax

Venetoclax 400mg orally once daily on days -8 through -1 with daily monitoring for tumor lysis syndrome. Venetoclax tablets will be swallowed (not crushed). Venetoclax dosing will not be repeated in the event of vomiting.

Hematopoietic Cell Transplantation Conditioning Regimen

Cytoxan Day -6 \& -5 Fludarabine Day -4, -3, -2 TBI Day -1

Kidney Transplant

Living donor kidney transplant

Hematopoietic Cell Transplantation

Donor bone marrow (target of 4 x 108 nucleated cells/kg of recipient ideal body weight)

PBSC A goal dose of 5 x 106 CD34+ cells / kg of recipient actual body weight will be infused (minimum acceptable is 2 x 106 CD34+ cells / kg).

Primary outcome measure

  • Incidence of full donor chimerism [ Time Frame: 24 months ]
  • Incidence of Grade IV Toxicity due to Venetoclax [ Time Frame: Days -8 to Days -1 ]
  • Incidence of Grade V Toxicity due to Venetoclax [ Time Frame: Days -8 to Days -1 ]

Central Contacts and Locations

Central contacts

Thomas Spitzer, MD

617-724-1124tspitzer@mgh.harvard.edu

Locations

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Thomas Spitzer, MD

617-724-1124tspitzer@mgh.harvard.edu

More Information

Sponsor

Massachusetts General Hospital

Last update posted

Oct 14, 2026

Last verified

Oct, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-15. This information was provided to ClinicalTrials.gov by Massachusetts General Hospital on 2026-10-14. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.