iPSC Neurons From Adult Survivors of Childhood Cancer Who Have Persistent Vincristine-Induced Neu… (NCT02564484) | Clinical Trial Compass
CompletedNot Applicable
iPSC Neurons From Adult Survivors of Childhood Cancer Who Have Persistent Vincristine-Induced Neuropathy
United States137 participantsStarted 2016-02-08
Plain-language summary
This observational study is designed to establish induced pluripotent stem cells (iPSCs) from childhood cancer survivors who did or did not develop persistent treatment-induced peripheral neuropathy, from which to make human neurons for comparing their sensitivity to vincristine and other potentially neurotoxic drugs.
Investigators will assess the effects of inherited genome variations on treatment-induced peripheral neuropathy that persists in adults who were cured of childhood cancer. Cells from childhood cancer survivors who did or did not develop drug-induced neuropathy will be isolated and induced to become neurons. Cell sensitivity to anticancer agents will be tested in both groups and compared to determine if the survivors have genetic variants that correspond to those identified in companion genomic studies. This will assist in determining if gene variants increase the risk of treatment-induced neurotoxicity.
The investigators are interested in detecting changes of phenotype pre-post treatment in each group (cases, controls) respectively, as well in comparing the pre-post treatment phenotypic changes between the two groups (cases vs. controls).
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria - Neuropathy Patients:
* Adult survivor of childhood ALL
* Presence of any neuromotor, neurocortical, or neurocerebellar toxicity after vincristine treatment (either acute and/or persistent neuropathy)
* At least 50% of the persistent vincristine neuropathy cases will have the CEP72 promoter variant (rs924607) or a coding variant in CEP72 that is predicted to be damaging (CADD score \>9). The balance of neuropathy cases will either have variants in other genes that are associated with vincristine neuropathy identified in the ongoing genome-wide association study (GWAS) of 1250 St. Jude Life ALL survivors, or have neuropathy in the absence of known genetic variants altering the risk of neuropathy.
* Patient understands the nature of the study and provides informed consent
Inclusion Criteria - Controls:
* Adult survivor of childhood diagnosis of ALL
* Absence of persistent neurotoxicity (grade 0) after completion of a standard vincristine-containing chemotherapy regimen (may or may not have experience acute neuropathy during treatment.
* Matched to a specified subject with neurotoxicity based on age (within 5 years), tumor type, chemotherapy regimen or total vincristine dosage, race and ethnicity.
* At least 50% of the controls will have the CEP72 promoter variant T/T genotype (at rs924607) or a coding variant in CEP72 that is predicted to be damaging (CADD score \>9). The balance of controls will either have variants in other genes that are associated wit…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants for whom iPSCs were created
Timeframe: Up to 6 months after participant enrollment