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New chemo cocktail aims to boost stem cell transplant success in blood cancers

NCT ID NCT02250937

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 2 trial tests whether adding venetoclax to a specific chemotherapy regimen before a donor stem cell transplant can improve outcomes for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study enrolls 116 adults aged 18-70 who have a matched donor. The goal is to see if this approach helps destroy cancer cells more effectively and leads to longer survival without the disease returning.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
chemotherapy (venetoclax, busulfan, cladribine, fludarabine) followed by donor stem cell transplant
What this could lead to
If successful, this approach could improve long-term survival and reduce relapse for people with AML or MDS undergoing a donor stem cell transplant.
What could go wrong
This is a mid-stage trial with only 116 participants, so results may not apply to everyone. The intense chemo regimen carries risks like infection, organ damage, and graft-versus-host disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 116 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2014

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients with biopsy-proven acute myeloid leukemia or myelodysplastic syndrome with persistent disease or in remission * Human leukocyte antigen (HLA)-identical sibling or 8/8 matched unrelated donor transplant * Patients age 18 to 70 years old; eligibility for pediatric patients will be determined in conjunction with an MD Anderson Cancer Center (MDACC) pediatrician; patients age 2-17 years may be enrolled after at least 10 adults (ages 18-70 years old) have been assessed for safety at day 30 * Direct bilirubin \< 1 mg/dl * Alanine aminotransferase (ALT) \< 3 times upper limit of normal * Creatinine clearance \> 50 ml/min (calculated creatinine clearance is permitted) * Forced expiratory volume in 1 second (FEV1) \>= 50% of expected corrected for hemoglobin and/or volume * Forced vital capacity (FVC) \>= 50% of expected corrected for hemoglobin and/or volume * Diffusing capacity of the lungs for carbon monoxide (DLCO) \>= 50% of expected corrected for hemoglobin and/or volume * Children unable to perform pulmonary function tests (e.g., less than 7 years old) pulse oximetry of \>= 92% on room air * Left ventricular ejection fraction (LVEF) \>= 40% * Patient, legally authorized representative (LAR), or parent able to sign informed consent; able to give assent for patients age 7-17 * Negative beta human chorionic gonadotropin (HCG) test in a woman with child bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization; women of child bearing potential must be willing to use an effective contraceptive measure while on study * Performance score of \>= 70 by Karnofsky/Lansky or performance status (PS) 0 or 1 (Eastern Cooperative Oncology Group \[ECOG\] =\< 1) Exclusion Criteria: * Prior allogeneic or autologous transplantation * Uncontrolled infections * Human immunodeficiency virus (HIV) seropositivity * Hematopoietic cell transplantation (HCT) co-morbidity index score \> 3; the principal investigator is the final arbiter of eligibility for comorbidity score \> 3 * Patients with prior coronary artery disease

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • M D Anderson Cancer Center

    Houston, Texas, 77030, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.