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Phase 1-2 MAHCT w/ TCell Depleted Graft w/ Simultaneous Infusion Conventional and Regulatory T Cell
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Summary Quick facts Who can join Endpoints Results Adverse events Publications Related trials Sources
Completed
Phase 1, PHASE2
Results posted
Last updated 19 August 2025
What this trial tests
Phase 1, PHASE2 trial testing CD34+ Hematopoietic Progenitor Cells (HSPC) in Myeloid Leukemia, Chronic in 68 participants. Completed in 20 December 2023.
Timeline
9 February 2012
Primary endpoint
20 December 2023
Quick facts
Lead sponsor Stanford University Phase Phase 1, PHASE2 Status Completed Study type INTERVENTIONAL Allocation non randomized Design sequential Masking none Primary purpose treatment Enrollment 68 Start date 9 February 2012 Primary completion 20 December 2023 Estimated completion 20 December 2023 Sites 1 location across United States
Drugs / interventions tested
Conditions studied
Sponsor
Stanford University
Who can join
Adults 13 to 73, any sex, with Myeloid Leukemia, Chronic or Acute Myelogenous Leukemia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
GvHD Free Relapse Free Survival (GRFS)
Primary
· 12 months
GvHD-free is defined as no GvHD symptoms, and relapse free survival is defined as survival at 12 months without relapse.
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 9 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 7 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 26
Number of Participants With Dose-Limiting Toxicity (DLT) Within 28 Days
Secondary
· 28 days
Dose-limiting Toxicity (DLT) was assessed as:
* Absolute neutrophil count \<500/µL, to 28 day
* Cytokine release syndrome/acute infusion reactions as CTCAE Grade 3 to 5
* Grade 3 to 4 acute GvHD. GvHD was staged as follows:
* 1: Skin: rash \<25%. Liver: bilirubin (BIL) 2-3mg/dL. Gut: diarrhea (DIA) 500-1000 mL/day
* 2: Skin: rash 25-50%. Liver: BIL 3-6mg/dL. Gut: DIA 1001-1500 mL/day
* 3: Skin: rash \> 50%. Liver: BIL 6-15mg/dL. Gut: DIA \>1501-2000 mL/day
* 4: Skin: generalized erythroderma. Liver: BIL \>15mg/dL. Gut: DIA \>2001 mL/day GvHD was graded as follows.
* 1: Skin Stage 1
Total DLT Events
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 0 Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A) 0
Grade 3 Acute GvHD
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 2 Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A) 1
Grade 4 Acute GvHD
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 0 Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A) 2
Grade 3-5 CRS
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 0 Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A) 0
ANC <500
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 0 Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A) 0
Number of Participants With Overall Survival (OS) at 1 Year
Secondary
· 1 year
Overall Survival (OS) at 1 year was assessed as the number of participants per treatment level that received the hematopoietic cell transplant (HCT), and remained alive 12 months later.
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 2 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 6 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 11 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 10 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 28
Number of Participants With Severity of Chronic Graft-vs-Host Disease (cGvHD) at 24 Months
Secondary
· 2 years
Incidence and severity of chronic GvHD was assessed in participants who received the hematopoietic cell transplant (HCT) at 24 months.
Milld cGvHD
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 1 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 1 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 1 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 6
Moderate cGvHD
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 3 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 7
Severe cGvHD
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 0 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 1 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 0
Number of Participants With Incidence of Serious Infections
Secondary
· 24 months
The outcome is reported as the number of serious infections per treatment level, in participants who received the hematopoietic cell transplant (HCT).
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 6 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 3 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 0 Phase 2, Phase 2 Treg + Tcon With Immunosuppression (Cohort 2A) 2
Number of Participants Receiving Concomitant Single-Agent Immunosuppression
Secondary
· 2 years
During Phase 2, stage 1, concomitant single-agent immunosuppression was assessed as in participants receiving fresh Treg cells.
Group Value 95% CI Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation) 0 Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation) 1 Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation) 6 Phase 2, Stage 1: Treg + Tcon With Immunosuppression (Cohort 2A) 12 Phase 2, Stage 1: Treg + Tcon Without Immunosuppression (Cohort 2A) 12 Phase 2, Stage 2: Treg + Tcon With Immunosuppression (Cohort 2A) 32
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 2 years.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Cohort 1, Phase I: Low Dose Treg + Tcon (Dose Escalation)
Serious: 1/1 (100%)
Deaths: 1/1
Cohort 1A, Phase I: Low Dose Treg + Tcon (Dose Escalation)
Serious: 2/5 (40%)
Deaths: 3/5
Cohort 2A, Phase I: Mid Dose Treg + Tcon (Dose Escalation)
Serious: 6/6 (100%)
Deaths: 2/6
Phase 2, Staage 1 Treg + Tcon With Immunosuppression (Cohort 2A)
Serious: 2/12 (17%)
Deaths: 3/12
Phase 2, Stage 1 Treg + Tcon Without Immunosuppression (Cohort 2A)
Serious: 6/12 (50%)
Deaths: 2/12
Phase 2, Stage 2 Treg + Tcon With Immunosuppression (Cohort 2A)
Serious: 10/32 (31%)
Deaths: 6/32
Serious adverse events (50 terms) Reaction System Cohort 1, Phase I: Low Dos… Cohort 1A, Phase I: Low Do… Cohort 2A, Phase I: Mid Do… Phase 2, Staage 1 Treg + T… Phase 2, Stage 1 Treg + Tc… Phase 2, Stage 2 Treg + Tc… Febrile neutropenia Blood and lymphatic system disorders — — — — — — Mucositis oral Gastrointestinal disorders — — — — — — Renal insufficiency Renal and urinary disorders — — — — — — Skin GVHD Skin and subcutaneous tissue disorders — — — — — — Neutrophil count decreased Blood and lymphatic system disorders — — — — — — Platelet count decreased Blood and lymphatic system disorders — — — — — — CML relapse Blood and lymphatic system disorders — — — — — — Graft failure Blood and lymphatic system disorders — — — — — — Pancytopenia Blood and lymphatic system disorders — — — — — — Abdominal pain Gastrointestinal disorders — — — — — — Ascites Gastrointestinal disorders — — — — — — Colitis Gastrointestinal disorders — — — — — — Diarrhea Gastrointestinal disorders — — — — — — Epigastric pain Gastrointestinal disorders — — — — — — Nausea Gastrointestinal disorders — — — — — — Vomiting Gastrointestinal disorders — — — — — — Acute GI and Liver GVHD Gastrointestinal disorders — — — — — — Fever General disorders — — — — — — Acute cholecystitis Hepatobiliary disorders — — — — — — Gallbladder obstruction Hepatobiliary disorders — — — — — — Sinusoidal obstruction syndrome Hepatobiliary disorders — — — — — — Liver GVHD Hepatobiliary disorders — — — — — — Bacteremia Infections and infestations — — — — — — Cytomegalovirus infection reactivation Infections and infestations — — — — — — Epstein-Barr Infection reactivation Infections and infestations — — — — — —
Other adverse events (100 terms — click to expand) Reaction System Cohort 1, Phase I: Low Dos… Cohort 1A, Phase I: Low Do… Cohort 2A, Phase I: Mid Do… Phase 2, Staage 1 Treg + T… Phase 2, Stage 1 Treg + Tc… Phase 2, Stage 2 Treg + Tc… Mucositis oral Gastrointestinal disorders — — — — — — Febrile neutropenia Blood and lymphatic system disorders — — — — — — Diarrhea Gastrointestinal disorders — — — — — — Abdominal pain Gastrointestinal disorders — — — — — — Fever General disorders — — — — — — Dysuria General disorders — — — — — — Rash Skin and subcutaneous tissue disorders — — — — — — Neutrophil count decreased Blood and lymphatic system disorders — — — — — — Sinus tachycardia Cardiac disorders — — — — — — Nausea Gastrointestinal disorders — — — — — — Vomiting (Emesis) Gastrointestinal disorders — — — — — — Rectal hemorrhoids Gastrointestinal disorders — — — — — — Anorexia Gastrointestinal disorders — — — — — — Fatigue General disorders — — — — — — Alanine aminotransferase increased Investigations — — — — — — Aspartate aminotransferase increased Investigations — — — — — — Back pain Musculoskeletal and connective tissue disorders — — — — — — Headache Nervous system disorders — — — — — — Cytomegalovirus (CMV) infection Infections and infestations — — — — — — GI GVHD Gastrointestinal disorders — — — — — — Urinary tract infection Infections and infestations — — — — — — Insomnia Nervous system disorders — — — — — — Anxiety Psychiatric disorders — — — — — — Acute kidney injury Renal and urinary disorders — — — — — — Pruritis Skin and subcutaneous tissue disorders — — — — — — White blood cell decreased Blood and lymphatic system disorders — — — — — — Hypertension Cardiac disorders — — — — — — chest pain Cardiac disorders — — — — — — Periorbital Edema (retinal hemorrhage) Eye disorders — — — — — — Abdominal distention Gastrointestinal disorders — — — — — — Ascites Gastrointestinal disorders — — — — — — Dyspepsia Gastrointestinal disorders — — — — — — Gastric hemorrhage Gastrointestinal disorders — — — — — — Rectal Pain Gastrointestinal disorders — — — — — — constipation Gastrointestinal disorders — — — — — — Chills General disorders — — — — — — Epistaxis General disorders — — — — — — Hypothermia General disorders — — — — — — Limb edema General disorders — — — — — — Oral candidiasis (Thrush) Infections and infestations — — — — — —
Most-reported serious reactions: Febrile neutropenia , Mucositis oral , Renal insufficiency , Skin GVHD , Neutrophil count decreased , Platelet count decreased , CML relapse , Graft failure .
Data from ClinicalTrials.gov NCT01660607 adverse events section .
Sponsor's own description
This study looks at giving specific types of immune cells, called regulatory T cells and conventional T cells, to patients with blood cancers who are receiving a stem cell transplant. These cells are added back to help the immune system recover and reduce complications after the transplant.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
Therapeutic induction of antigen-specific immune tolerance.
Kenison JE, Stevens NA, Quintana FJ. ·
· 2024
· cited 94×
· PMID 38086932
· DOI 10.1038/s41577-023-00970-x
Opportunities for Treg cell therapy for the treatment of human disease.
Bluestone JA, McKenzie BS, Beilke J, Ramsdell F. ·
· 2023
· cited 90×
· PMID 37153574
· DOI 10.3389/fimmu.2023.1166135
Transplantation of donor grafts with defined ratio of conventional and regulatory T cells in HLA-matched recipients.
Meyer EH, Laport G, Xie BJ, MacDonald K, et al ·
· 2019
· cited 65×
· PMID 31092732
· DOI 10.1172/jci.insight.127244
Cell-Based Therapies with T Regulatory Cells.
Gliwiński M, Iwaszkiewicz-Grześ D, Trzonkowski P. ·
· 2017
· cited 60×
· PMID 28540499
· DOI 10.1007/s40259-017-0228-3
Current approaches to prevent and treat GVHD after allogeneic stem cell transplantation.
Hamilton BK. ·
· 2018
· cited 52×
· PMID 30504315
· DOI 10.1182/asheducation-2018.1.228
Clinical Grade Regulatory CD4<sup>+</sup> T Cells (Tregs): Moving Toward Cellular-Based Immunomodulatory Therapies.
Duggleby R, Danby RD, Madrigal JA, Saudemont A. ·
· 2018
· cited 52×
· PMID 29487602
· DOI 10.3389/fimmu.2018.00252
Regulatory T Cells in GVHD Therapy.
Guo WW, Su XH, Wang MY, Han MZ, et al ·
· 2021
· cited 51×
· PMID 34220860
· DOI 10.3389/fimmu.2021.697854
Ways Forward for Tolerance-Inducing Cellular Therapies- an AFACTT Perspective.
Ten Brinke A, Martinez-Llordella M, Cools N, Hilkens CMU, et al ·
· 2019
· cited 38×
· PMID 30853957
· DOI 10.3389/fimmu.2019.00181
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Verify against primary sources
Data sources for this page
Trial protocol + status : ClinicalTrials.gov NCT01660607 (US National Library of Medicine, public domain)Publications : Europe PMC API search by NCT ID, retrieved 10 June 2026Drug + disease cross-links : matched in real time against Drug Landscape's normalised drug + company + condition tablesSponsor : as reported to ClinicalTrials.gov by Stanford UniversityLast refreshed : 19 August 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01660607.
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