A Phase 3 interventional study of PCA plus methadone (Experimental) and PCA Alone (SOC) in Leukemia, Aplastic Anemia and Bone Marrow Failure Syndromes, sponsored by University of Texas Southwestern Medical Center. Suspended at 1 site in United States. Open to participants aged 6 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-10-23.
Sponsored by University of Texas Southwestern Medical Center · Phase 3, Interventional, and Supportive care
Mucositis is a normal side effect of stem cell transplant which happens as a result of chemotherapy being given prior to a new donor cell infusion (bone marrow transplant). The chemotherapy will kill cancer cells, but good cells, such as those in the mouth, are killed too. The mouth cells going away causes the areas in the mouth to be blistered, irritated, sore, and extremely painful. Pain medication (usually morphine or hydromorphone if allergic to morphine) are given when oral blisters are seen or felt by patient in patient's mouth. However, one pain medication given through a vein in the patient may or may not be effective and providers are often challenged with providing good pain control while waiting for the new donor cells to grow, which will then heal the mouth. This is a period of waiting that is 6-8 weeks.
The investigators know that methadone, a second pain medication, may decrease pain in a different way than morphine. This is because methadone works in a different way in the brain than morphine. By giving these pain medicines together, the hope of the study is to show decreased pain while waiting for new cells to grow.
The goal of this clinical trial is to hope to learn whether adding methadone (second pain medication) to the current pain medication which is morphine alone (all patients will receive this pain medication) will help reduce the pain experience of participant. Current treatment of morphine alone is sometimes not entirely effective and so any improvement of pain while waiting for new cells to grow is one of the goals of this study. If methadone is effective in decreasing pain, then patients may benefit in the future from using these two medications up front when getting a transplant.
Participant in this study between 6-18 years of age and is needing a stem cell transplant for a disease that can potentially be cured by transplantation.
Participant in this study is receiving chemotherapy and/or radiation conditioning that can cause mucositis. Participants are being asked to participate in this study because participants meet criteria to receive methadone that may or may not reduce pain experience versus just being given morphine alone, which is what all patients are given when the participants have mucositis.
The main goal of the study is to see if less opioid (pain medication) when methadone is added in comparison to participant who uses PCA only. The investigators also want to learn if patient's overall function is improved if given methadone. Another goal would be to see the number of TPN days the participant received and if the participant who was given methadone began to eat sooner. Other smaller goals include learning about side effects of methadone, and if the hospital stay is less for those who receive the study medication.
This medication will be given at Children's Medical Center of Dallas while participant is admitted for the stem cell transplant. There is no sponsor that is funding the study and this drug will be given free of charge in exchange for participation in the study
Given high reports of pain while despite receiving PCA during transplant, our current management of mucositis induced pain is suboptimal. In a study of children receiving transplantation, pain was a main concern for this cohort during and after transplantation. The investigators propose to conduct this study to determine if methadone used as a primary analgesic in patients with mucositis can improve the current standard of care which will be demonstrated by improved pain scores, maintenance of functionality, and recovery during and following HSCT. This study will be a single blind randomized controlled design. Patients will be randomly assigned by permuted block randomization in groups of eight. Patient will be randomly assigned to receive either basal/PCA of one opioid (control) in the first arm OR PCA plus methadone where methadone will be used as basal long-acting medication (experimental).
Study subjects will be children who are undergoing an autologous or allogeneic stem cell transplant receiving a myeloablative regimen and meet all inclusion criteria. This unique population is being studied as it is a controlled setting where the PI can anticipate pain in >90% of the patients following ablation, the number of patients who experience mucositis after receiving ablative conditioning. The study will only be conducted in the inpatient stem cell transplant setting and no follow up should be necessary when these subjects are discharged from the hospital after transplant.
The following goals of the study are as follows:
2,381 studies on the registry are indexed under Leukemia; 253 are open to participants now.
This study's planned enrollment of 60 is above the median of 40 across 1,769 interventional studies indexed under Leukemia.
Browse Leukemia studies →University of Texas Southwestern Medical Center is the lead sponsor of 1,010 studies on the registry; 199 are open to participants now.
Of its 139 completed or terminated interventional studies of FDA-regulated products, 105 (76%) have results posted.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Pt will receive: PCA opioid will include morphine OR hydromorphone (if patient has a sensitivity or is unable to tolerate or allergic to morphine). This is the exclusive medication/opioid in this arm. The starting doses will be as follows: * Starting dose for basal infusion of morphine is 0.02 mg/kg/hour up to a maximum of 50 kg. * Starting PCA dose for morphine is 0.02 mg/kg every 15 minutes for a lockout of 0.1 mg/kg up to a maximum of 50 kg or ideal body weight (PCA starting dose should be no more than 1 mg/push and no more than 5 mg/hour lockout). * Starting dose for basal infusion of hydromorphone is 0.003 mg/kg/hour up to a maximum of 50 kg IBW. * Starting PCA dose for hydromorphone is 0.003 mg/kg every 15 minutes for an hourly maximum lockout of 0.012 mg/kg up to a maximum of 50 kg (PCA start * PCA medications may be titrated up or down by 20-50% each day for desired analgesic effect with improved pain score, continue to engraftment or when medicine is no longer needed.
Drug: PCA Alone (SOC)
The second arm will be methadone therapy that is scheduled to be given every 8 hours in addition to an on demand PCA. On demand PCA dosing will be the same as control arm but with NO basal/continuous rate. Intravenous methadone will be replacing basal infusion and serve as basal drug in place of continuous medication. * Starting dose for intravenous methadone is 0.1 mg/kg IV Q8hr up to a maximum of 50 kg. Singular starting methadone will be maximum of 5 mg. * Methadone may be increased by 20-50% every 48 hours due to half-life of medication. Reduction in analgesia will need to be monitored daily following a change. However, additional methadone increase should not occur until 48 hours have passed to allow for steady state of new dose to take effect. Reduction of methadone dosing by 20-50% can be made anytime per discretion of provider or if concerned about an adverse effect from the methadone. This will continue until engraftment or until medicine is no longer needed.
Drug: PCA plus methadone (Experimental)
Drug page: Methadone
This study is novel. There are no other interventions to distinguish in this or another study.
Also known as: Addition of methadone to standard of care (SOC)
PCA Alone (Control) arm. PCA opioid will include morphine OR hydromorphone (if patient has a sensitivity or is unable to tolerate or allergic to morphine).
Also known as: Morphine OR Hydromorphone
Reduction of pain score
The purpose of the study is to compare methadone versus standard of care at this center of morphine/hydromorphone PCA and see if methadone is superior to the standard of care as evidenced by reduction in pain scores during inpatient transplant. To determine whether concomitant administration of intravenous administered methadone with on demand opioid PCA will improve pain score numbers in the PCA/methadone arm by 25% in comparison to a continuous infusion PCA with demand opioid arm.
Time frame: Daily after completion of conditioning (day +7 to day +14) through discharge (approximately day +60)
REDUCTION OF OPIOID RECEIVED as evidenced by 25% reduction of MME in experimental arm in comparison to control arm
To determine if methadone (starting dose of 0.1 mg/kg IV Q8hr) with increased titration will result in a reduction of 25% in total morphine milligram equivalents (MME) per day in children who have mucositis following ablative conditioning and subsequent allogeneic or autologous HSCT
Time frame: Daily after completion of conditioning (day +7 to day +14) through discharge (approximately day +60)
IMPROVEMENT OF FUNCTIONALITY as evidenced by higher functionality scores in experimental arm in comparison to control
To determine how administration of methadone affects functionality during transplantation period
Time frame: Day 0 (stem cell infusion)
REDUCED NUMBER OF DAYS ON PARENTERAL NUTRITION as evidenced by reduced number of TPN days in experimental arm in comparison to control
To determine if the overall TPN days are reduced during hospital stay in experimental arm as compared to the standard PCA regimen
Time frame: From enrollment (initiation of TPN- typically on day +1) to engraftment day AND date when able to take solid food again (day just prior to discharge-approx. day+50 to day+60)
INCREASED RETURN TO ORAL LIQUID INTAKE as evidenced by more rapid return to tolerating clears in experimental group versus control
To determine if child resumes oral intake more rapidly compared to control arm
Time frame: From enrollment (initiation of TPN- typically on day +1) to engraftment day AND date when able to take solid food again (day just prior to discharge-approx. day+50 to day+60)
Adverse events
To describe adverse events and incidence associated with methadone when administered in combination with morphine or hydromorphone, specifically QTc prolongation that has been documented with study medication
Time frame: From first dose of methadone administered (day +1- day +7) until prior to discharge (approximately day +60)
Plan to share: No — There is no reason to share individualized participant data in this study. My plan is to deidentify all data and all data will be anonymous.
This study is suspended, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Methadone
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