B Acute Lymphoblastic Leukemia Associated With Down SyndromeNCT05702645
A Study to Learn More About the Health of Persons With Down Syndrome After Treatment for Acute Leukemia
An observational study of B Acute Lymphoblastic Leukemia Associated With Down Syndrome and Down Syndrome, run by Children's Oncology Group at 73 sites in 2 countries.
Trial summary
This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. Read more about this study
- Who is it for?
- Ages 6 to 39 years
- Conditions the study is for
- B Acute Lymphoblastic Leukemia Associated With Down Syndrome, Down Syndrome, Myeloid Leukemia Associated With Down Syndrome
- What is being tested?
- Biospecimen Collection, Clinical Evaluation and Neurocognitive Assessment
- Type of treatment
- Surgery or procedure
- How many people
- 330 participants
- Where
- 73 sitesin 2 countries
- Study dates
- 30 Nov 2023to 30 Jun 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 6 to 39 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: DS-AL survivors from the Children's Oncology Group (COG) Registry and Epidemiology protocols or locally from participating institutions.
Who may be able to join
- Patients age >= 6 and < 40 years at the time of enrollment
- A diagnosis of Down syndrome is required, and may include any of the three recognized types: trisomy 21 resulting from chromosomal nondisjunction (most common), translocation (the patient has 46 chromosomes, but all or part of an additional copy of chromosome 21 is attached to another chromosome), or mosaicism (trisomy 21 that is present in only a fraction of cells)
- All patients must be DS-AL survivors (acute lymphoblastic leukemia [ALL] or acute myeloid leukemia [AML])
- Note 1: Myeloid leukemia of Down syndrome (ML-DS) is included in the AML category above. Per the World Health Organization (WHO) definition of ML-DS, this diagnosis encompasses both myelodysplastic syndrome (MDS) and overt AML. Also, note that survivors of relapsed disease are eligible, so long as the patient otherwise meets eligibility criteria, i.e., treatment for relapse was completed at least 36 calendar months prior to enrollment and did not include stem cell transplant
- Note 2: A diagnosis of transient abnormal myelopoiesis (TAM), also known as transient myeloproliferative disease (TMD), is not alone sufficient for inclusion in this study
- Patients must have been treated for ALL or AML
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- Note: History of COG therapeutic trial participation is not required. As a reminder ML-DS would be included under the AML category here above
- All cancer treatment (oral or intravenous) must have been completed at least 36 calendar months prior to enrollment
- Patients must have a life expectancy of > 1 year
- Patient and parent of subject must be either English or Spanish speaking. At least one parent or guardian must be able to read and write in English or Spanish
- Note: Parents or guardians are responsible for completing all questionnaires, even in the case of subjects that are >= 18 years old
- All patients and/or their parents or legal guardians must sign a written informed consent
- All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Who cannot join
- Patients with history of hematopoietic stem cell transplant (HSCT) are excluded
- Note: Patients with previous chimeric antigen receptor T-cell (CAR T-cell) therapy, and other cellular cancer therapies can participate, as long as all other eligibility criteria are satisfied
- Patients with a history of cancers prior to their ALL or AML diagnosis are excluded. Patients that developed a subsequent malignant neoplasm following their ALL or AML diagnosis are also excluded
- Note: Prior history of transient abnormal myelopoiesis is allowed, but is not sufficient for eligibility
- Patients whose parents or guardians are unable to complete the required forms are excluded
Trial locations and contacts
73 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
73 sites in 2 countries
United States72 sites, 72 recruiting
- Recruiting
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Site Public Contact, 330-543-3193
Erin Wright
- Recruiting
Albany Medical Center
Albany, New York, 12208
Site Public Contact, 518-262-5513
Lauren R. Weintraub
- Recruiting
C S Mott Children's Hospital
Ann Arbor, Michigan, 48109
Site Public Contact, 800-865-1125
Joshua W. Goldman
- Recruiting
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, 30329
Site Public Contact, 404-785-0232, Olivia.Floyd@choa.org
Karen E. Effinger
- Recruiting
Augusta University Medical Center
Augusta, Georgia, 30912
Site Public Contact, 706-721-2388, ga_cares@augusta.edu
Colleen H. McDonough
- Recruiting
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723
Site Public Contact, 512-628-1902, TXAUS-DL-SFCHemonc.research@ascension.org
Shannon M. Cohn
- Recruiting
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215
Site Public Contact, 410-601-9083
Jason M. Fixler
- Recruiting
Children's Hospital of Alabama
Birmingham, Alabama, 35233
Site Public Contact, 205-638-9285, oncologyresearch@peds.uab.edu
Smita Bhatia
- Recruiting
Roswell Park Cancer Institute
Buffalo, New York, 14263
Site Public Contact, 800-767-9355, askroswell@roswellpark.org
Denise A. Rokitka
- Recruiting
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599
Site Public Contact, 877-668-0683, cancerclinicaltrials@med.unc.edu
Thomas B. Alexander
- Recruiting
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611
Site Public Contact, 773-880-4562
Lindsay F. Schwartz
- Recruiting
University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637
Site Public Contact, 773-702-8222, cancerclinicaltrials@bsd.uchicago.edu
Gabrielle Lapping-Carr
- Recruiting
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, 44106
Site Public Contact, 216-844-5437
Duncan S. Stearns
- Recruiting
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Site Public Contact, 866-223-8100, TaussigResearch@ccf.org
Matteo M. Trucco
- Recruiting
Prisma Health Richland Hospital
Columbia, South Carolina, 29203
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Stuart L. Cramer
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
Site Public Contact, 614-722-6039, Melinda.Triplet@nationwidechildrens.org
Mark A. Ranalli
- Recruiting
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390
Site Public Contact, 214-648-7097, canceranswerline@UTSouthwestern.edu
Ksenya Shliakhtsitsava
- Recruiting
Dayton Children's Hospital
Dayton, Ohio, 45404
Site Public Contact, 800-228-4055
Jordan M. Wright
- Recruiting
Children's Hospital of Michigan
Detroit, Michigan, 48201
Site Public Contact, helpdesk@childrensoncologygroup.org
Alissa M. Martin
- Recruiting
Sanford Broadway Medical Center
Fargo, North Dakota, 58122
Site Public Contact, 701-323-5760, OncologyClinicalTrialsFargo@sanfordhealth.org
Samuel J. Milanovich
- Recruiting
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, 33908
Site Public Contact, 239-343-5333, molly.arnstrom@leehealth.org
Emad K. Salman
- Recruiting
Cook Children's Medical Center
Fort Worth, Texas, 76104
Site Public Contact, 682-885-2103, CookChildrensResearch@cookchildrens.org
Donald T. Beam
- Recruiting
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Site Public Contact, 616-391-1230, crcwm-regulatory@crcwm.org
Kathleen Y. Butler
- Recruiting
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, 29605
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Aniket Saha
- Recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Site Public Contact, 551-996-2897
Katharine R. Lange
- Recruiting
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
Site Public Contact, 954-265-1847, OHR@mhs.net
Iftikhar Hanif
- Recruiting
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96826
Site Public Contact, 808-983-6090
Wade T. Kyono
- Recruiting
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030
Site Public Contact, 713-798-1354, burton@bcm.edu
Omar Shakeel
- Recruiting
UT MD Anderson Cancer Center
Houston, Texas, 77030
Site Public Contact, 866-632-6789, askmdanderson@mdanderson.org
Najat C. Daw
- Recruiting
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Site Public Contact, 601-815-6700
Amanda Strobel
- Recruiting
Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, 32207
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Vibhuti Agarwal
- Recruiting
Bronson Methodist Hospital
Kalamazoo, Michigan, 49007
Site Public Contact, 616-391-1230, crcwm-regulatory@crcwm.org
Kathleen Y. Butler
- Recruiting
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Site Public Contact, 816-302-6808, COGResearchGroup@cmh.edu
Keith J. August
- Recruiting
East Tennessee Childrens Hospital
Knoxville, Tennessee, 37916
Site Public Contact, 865-541-8266
Susan E. Spiller
- Recruiting
University Medical Center of Southern Nevada
Las Vegas, Nevada, 89102
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Sunrise Hospital and Medical Center
Las Vegas, Nevada, 89109
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Summerlin Hospital Medical Center
Las Vegas, Nevada, 89144
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Valley Children's Hospital
Madera, California, 93636
Site Public Contact, 559-353-3000, Research@valleychildrens.org
Ruetima Titapiwatanakun
- Recruiting
University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792
Site Public Contact, 800-622-8922, clinicaltrials@cancer.wisc.edu
Cathy A. Lee-Miller
- Recruiting
Marshfield Medical Center-Marshfield
Marshfield, Wisconsin, 54449
Site Public Contact, 800-782-8581, oncology.clinical.trials@marshfieldresearch.org
Michelle A. Manalang
- Recruiting
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105
Site Public Contact, 888-226-4343, referralinfo@stjude.org
Lisa Jacola
- Recruiting
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Site Public Contact, 414-955-4727, MACCCTO@mcw.edu
Rachel A. Phelan
- Recruiting
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, 55404
Site Public Contact, 612-813-5913, pauline.mitby@childrensmn.org
Michael K. Richards
- Recruiting
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455
Site Public Contact, 612-624-2620
Karim T. Sadak
- Recruiting
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232
Site Public Contact, 800-811-8480
Debra L. Friedman
- Recruiting
Yale University
New Haven, Connecticut, 06520
Site Public Contact, 203-785-5702, canceranswers@yale.edu
Rozalyn L. Rodwin
- Recruiting
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Site Public Contact, 510-428-3264, PedOncRschOAK@ucsf.edu
Karen R. Rabin
- Recruiting
Kaiser Permanente-Oakland
Oakland, California, 94611
Site Public Contact, 877-642-4691, Kpoct@kp.org
Aarati V. Rao
- Recruiting
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Site Public Contact, 405-271-8777, ou-clinical-trials@ouhsc.edu
Rene Y. McNall-Knapp
- Recruiting
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, 68114
Site Public Contact, 402-955-3949
Jill C. Beck
- Recruiting
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Site Public Contact, 402-559-6941, unmcrsa@unmc.edu
Jill C. Beck
- Recruiting
Nemours Children's Hospital
Orlando, Florida, 32827
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Vibhuti Agarwal
- Recruiting
Nemours Children's Clinic - Pensacola
Pensacola, Florida, 32504
Site Public Contact, helpdesk@childrensoncologygroup.org
Jeffrey H. Schwartz
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Site Public Contact, 267-425-5544, CancerTrials@email.chop.edu
Sogol Mostoufi-Moab
- Recruiting
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
Site Public Contact, 602-546-0920
Alexandra M. Walsh
- Recruiting
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Site Public Contact, 412-692-8570, jean.tersak@chp.edu
Jean M. Tersak
- Recruiting
Oregon Health and Science University
Portland, Oregon, 97239
Site Public Contact, 503-494-1080, trials@ohsu.edu
Susan J. Lindemulder
- Recruiting
Rhode Island Hospital
Providence, Rhode Island, 02903
Site Public Contact, 401-444-1488
Bradley DeNardo
- Recruiting
Renown Regional Medical Center
Reno, Nevada, 89502
Site Public Contact, 775-982-5050, Renown-CRD@renown.org
Alan K. Ikeda
- Recruiting
University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817
Site Public Contact, 916-734-3089
Marcio H. Malogolowkin
- Recruiting
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
Site Public Contact, 210-450-3800, phoresearchoffice@uthscsa.edu
Shafqat Shah
- Recruiting
Rady Children's Hospital - San Diego
San Diego, California, 92123
Site Public Contact, 858-966-5934
William D. Roberts
- Recruiting
UCSF Medical Center-Mission Bay
San Francisco, California, 94158
Site Public Contact, 877-827-3222, cancertrials@ucsf.edu
Karen R. Rabin
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Site Public Contact, 866-987-2000
Sarah E. Leary
- Recruiting
Sanford USD Medical Center - Sioux Falls
Sioux Falls, South Dakota, 57117-5134
Site Public Contact, 605-312-3320, OncologyClinicalTrialsSF@SanfordHealth.org
Jordan Fritch-Hanson
- Recruiting
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204
Site Public Contact, 800-228-6618, HopeBeginsHere@providence.org
Judy L. Felgenhauer
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Site Public Contact, 800-600-3606, info@siteman.wustl.edu
Robert J. Hayashi
- Recruiting
Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa, Florida, 33607
Site Public Contact, 813-357-0849, jennifer.manns@baycare.org
Don E. Eslin
- Recruiting
Montefiore Medical Center - Moses Campus
The Bronx, New York, 10467
Site Public Contact, 718-379-6866, eskwak@montefiore.org
Alice Lee
- Recruiting
Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Vibhuti Agarwal
- Recruiting
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
Site Public Contact, 336-713-6771
Sarah Supples
Canada1 site, 1 recruiting
- Recruiting
Children's Hospital
London, Ontario, N6A 5W9
Site Public Contact, 519-685-8306
Shayna M. Zelcer
About this study
Official title: Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)
This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. By learning more about the factors that may contribute to chronic health conditions and long-term effects after treatment for leukemia in persons with Down syndrome, clinical practice guidelines for survivorship care can be developed to help improve their quality-of-life.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine the prevalence, type, and severity of chronic health conditions (CHC) in survivors of Down syndrome-associated acute leukemia (DS-AL), and to compare CHC with frequency-matched DS individuals that have no cancer history.
SECONDARY OBJECTIVES:
- To characterize post-treatment clinical outcomes of DS-AL by prospective, in-person assessment.
II. To determine the prevalence and severity of parent-reported adverse neuropsychological (NP) outcomes in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.
III. To determine health-related quality of life (HRQOL) in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.
IV. To identify clinical risk determinants of CHC, NP, and clinical outcomes in survivors of DS-AL.
- To establish a well-annotated cohort of survivors of DS-AL and associated biobank as a resource for future investigations.
EXPLORATORY OBJECTIVES:
- For DS-acute lymphoblastic leukemia (DS-ALL), test if structural birth defects and genetic associations with etiology extend to CHC.
II. For DS-ALL, test if telomere length determined by polygenic risk score and telomere flow-fluorescence in situ hybridization (FISH) are associated with outcomes from in-person NP assessment.
OUTLINE:
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
- Conditions
- B Acute Lymphoblastic Leukemia Associated With Down Syndrome, Down Syndrome, Myeloid Leukemia Associated With Down Syndrome
- Being tested
- Biospecimen Collection, Clinical Evaluation, Neurocognitive Assessment, Questionnaire Administration, Survey Administration
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection
Patients undergo saliva/buccal and blood sample collection
Clinical EvaluationOther
Also called Clinical Assessment
Undergo a clinical assessment
Neurocognitive AssessmentOther
Undergo neurocognitive assessment
Questionnaire AdministrationOther
Ancillary studies
Survey AdministrationOther
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Observational (biospecimen collection, clinical evaluation) | Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study. Receives: Procedure: Biospecimen Collection; Other: Clinical Evaluation; Other: Neurocognitive Assessment; Other: Questionnaire Administration; Other: Survey Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence, type, and severity of chronic health conditions (CHC)
Summary statistics will be used to characterize the study populations on CHC outcomes. Quantitative data (number of comorbidities) will be summarized using descriptive statistics and correlational techniques. Will use pooled logistic regression to estimate overall response, 95% confidence interval (CI), and p-values for association of acute leukemia (AL) diagnosis with medical record-verified CHC, agnostic of time to CHC. Will use stratified Cox models to refine associations of AL diagnosis with CHC based on time to CHC incidence. Within each age interval, will estimate the hazard ratio, 95% CI, and p-values to report time-dependent effects of AL diagnosis on CHC.
Measured: Up to study completion
Secondary outcomes (5)
Post-treatment clinical outcomes
Summary statistics will be used to characterize the study population on clinical outcomes, by AL subtype and, for each test, the proportion of normal, abnormal, and missing tests.
Measured: Up to study completion
Prevalence and severity of parent-proxy neuropsychological (NP) outcomes
NP outcomes (measured by both parent proxy and direct assessment) will be reported by both raw and normalized scores, and quantitative data summarized using descriptive statistics and correlational techniques. The mean score for each test will be compared between the cohorts using a student t-test (2-sided significance level of 0.05 and equal variance).
Measured: Up to study completion
Health-related quality of life (HRQOL)
HRQOL will be assessed using the Pediatric Quality of Life Inventory. Parents will be asked to scale 23 items comprising four dimensions (Physical, Emotional, Social, and School functioning) on a 5 point unweighted Likert scale. Will use a t-test (or Wilcoxon rank sum test if appropriate) to compare the mean (or median) HRQOL score.
Measured: Up to study completion
Clinical risk determinants of CHC, NP, and clinical outcomes
Will use Cox regression to estimate the hazard ratio and 95% CI for (1) number, severity, and type of CHC, and (2) NP deficits dichotomized as clinically significant impairment yes/no (=< 1.5 standard deviation outside of the mean for the age-normative sample) according to age at diagnosis, years off therapy, sex, race/ethnicity, diagnosis, and treatment exposures.
Measured: Up to study completion
Well-annotated cohort of Down syndrome phenotyping acute leukemia study (DS-PALS) survivors and associated biobank
The number of patients who agree to be in the Biobanking part of the study and have leftover tumor tissue and some normal blood, bone marrow, or other tissue saved for future research.
Measured: Up to study completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2023
- Study start30 Nov 2023
- Primary completion30 Jun 2029 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for B Acute Lymphoblastic Leukemia Associated With Down SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 330 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Maria M Gramatges, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05702645
- Sponsor's protocol ID
- ALTE22C1
- Other IDs
- NCI-2022-09410, ALTE22C1, COG-ALTE22C1, ALTE22C1, R01CA263000, UG1CA189955
- Status last verified
- Apr 2026
- First submitted
- 9 Jan 2023
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Gramatges MM, Sanclemente LN, Hall L, Taylor OA, Nuno MM, Bhatia S, Chow EJ, Getz KD, Hitzler JK, Li AM, McCloskey K, Nathan PC, O'Brien MM, Patel S, Verma A, Yarbrough AR, Richard MA, Rosser TC, Jacola LM, Lupo PJ, Rabin KR. A multicenter observational cohort study in survivors of Down Syndrome-associated acute leukemia (ALTE22C1): a report from the Children's Oncology Group. BMC Cancer. 2025 Oct 20;25(1):1611. doi: 10.1186/s12885-025-14898-z.
PubMed 41116168
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)
- ClinicalTrials.gov ID
- NCT05702645
- Acronym / sponsor ID
- ALTE22C1
- Conditions
- B Acute Lymphoblastic Leukemia Associated With Down Syndrome, Down Syndrome, Myeloid Leukemia Associated With Down Syndrome
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 330 participants (estimated)
- Lead sponsor
- Children's Oncology GroupResearch network
- Study start
- 30 Nov 2023 (actual)
- Primary completion
- 30 Jun 2029 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 27 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. By learning more about the factors that may contribute to chronic health conditions and long-term effects after treatment for leukemia in persons with Down syndrome, clinical practice guidelines for survivorship care can be developed to help improve their quality-of-life.
Detailed description
PRIMARY OBJECTIVE:
- To determine the prevalence, type, and severity of chronic health conditions (CHC) in survivors of Down syndrome-associated acute leukemia (DS-AL), and to compare CHC with frequency-matched DS individuals that have no cancer history.
SECONDARY OBJECTIVES:
- To characterize post-treatment clinical outcomes of DS-AL by prospective, in-person assessment.
II. To determine the prevalence and severity of parent-reported adverse neuropsychological (NP) outcomes in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.
III. To determine health-related quality of life (HRQOL) in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.
IV. To identify clinical risk determinants of CHC, NP, and clinical outcomes in survivors of DS-AL.
- To establish a well-annotated cohort of survivors of DS-AL and associated biobank as a resource for future investigations.
EXPLORATORY OBJECTIVES:
- For DS-acute lymphoblastic leukemia (DS-ALL), test if structural birth defects and genetic associations with etiology extend to CHC.
II. For DS-ALL, test if telomere length determined by polygenic risk score and telomere flow-fluorescence in situ hybridization (FISH) are associated with outcomes from in-person NP assessment.
OUTLINE:
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
Study design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational (biospecimen collection, clinical evaluation) | – | Procedure: Biospecimen Collection, Other: Clinical Evaluation, Other: Neurocognitive Assessment, Other: Questionnaire Administration, Other: Survey AdministrationPatients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study. |
Who may qualify
- Ages
- 6 to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- DS-AL survivors from the Children's Oncology Group (COG) Registry and Epidemiology protocols or locally from participating institutions.
Key inclusion criteria (13 listed)
- Patients age >= 6 and < 40 years at the time of enrollment
- A diagnosis of Down syndrome is required, and may include any of the three recognized types: trisomy 21 resulting from chromosomal nondisjunction (most common), translocation (the patient has 46 chromosomes, but all or part of an additional copy of chromosome 21 is attached to another chromosome), or mosaicism (trisomy 21 that is present in only a fraction of cells)
- All patients must be DS-AL survivors (acute lymphoblastic leukemia [ALL] or acute myeloid leukemia [AML])
- Patients must have been treated for ALL or AML
- All cancer treatment (oral or intravenous) must have been completed at least 36 calendar months prior to enrollment
Key exclusion criteria (5 listed)
- Patients with history of hematopoietic stem cell transplant (HSCT) are excluded
- Patients with a history of cancers prior to their ALL or AML diagnosis are excluded. Patients that developed a subsequent malignant neoplasm following their ALL or AML diagnosis are also excluded
- Patients whose parents or guardians are unable to complete the required forms are excluded
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Specimen CollectionPatients undergo saliva/buccal and blood sample collection | Procedure or surgery | Observational (biospecimen collection, clinical evaluation) |
| Clinical EvaluationAlso: Clinical AssessmentUndergo a clinical assessment | Other | Observational (biospecimen collection, clinical evaluation) |
| Neurocognitive AssessmentUndergo neurocognitive assessment | Other | Observational (biospecimen collection, clinical evaluation) |
| Questionnaire AdministrationAncillary studies | Other | Observational (biospecimen collection, clinical evaluation) |
| Survey AdministrationAncillary studies | Other | Observational (biospecimen collection, clinical evaluation) |
Primary outcome
Prevalence, type, and severity of chronic health conditions (CHC)
Time frame: Up to study completion
Summary statistics will be used to characterize the study populations on CHC outcomes. Quantitative data (number of comorbidities) will be summarized using descriptive statistics and correlational techniques. Will use pooled logistic regression to estimate overall response, 95% confidence interval (CI), and p-values for association of acute leukemia (AL) diagnosis with medical record-verified CHC, agnostic of time to CHC. Will use stratified Cox models to refine associations of AL diagnosis with CHC based on time to CHC incidence. Within each age interval, will estimate the hazard ratio, 95% CI, and p-values to report time-dependent effects of AL diagnosis on CHC.
5 secondary outcomes
Post-treatment clinical outcomes
Time frame: Up to study completion
Prevalence and severity of parent-proxy neuropsychological (NP) outcomes
Time frame: Up to study completion
Health-related quality of life (HRQOL)
Time frame: Up to study completion
Clinical risk determinants of CHC, NP, and clinical outcomes
Time frame: Up to study completion
Well-annotated cohort of Down syndrome phenotyping acute leukemia study (DS-PALS) survivors and associated biobank
Time frame: Up to study completion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2026.
- Derived from this study: PubMed 41116168Gramatges MM, Sanclemente LN, Hall L, Taylor OA, Nuno MM, Bhatia S, Chow EJ, Getz KD, Hitzler JK, Li AM, McCloskey K, Nathan PC, O'Brien MM, Patel S, Verma A, Yarbrough AR, Richard MA, Rosser TC, Jacola LM, Lupo PJ, Rabin KR. A multicenter observational cohort study in survivors of Down Syndrome-associated acute leukemia (ALTE22C1): a report from the Children's Oncology Group. BMC Cancer. 2025 Oct 20;25(1):1611. doi: 10.1186/s12885-025-14898-z.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Maria M Gramatges principal investigator
Children's Oncology Group
73 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of B Acute Lymphoblastic Leukemia Associated With Down Syndrome and Down Syndrome, run by Children's Oncology Group at 73 sites in 2 countries.
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 13 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 73 sites in United States, Canada. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevalence, type, and severity of chronic health conditions (CHC) (time frame: Up to study completion). 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 NCT05702645, which also lists 1 linked publication.
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 25 Aug 2026.