Pheripheral blood stem cells for Chronic Granulomatous Disease
Study Summary
This trial is testing a new drug treatment to see if it can improve the success rates of stem cell transplants in people with CGD.
- Chronic Granulomatous Disease
Treatment Effectiveness
Effectiveness Progress
Study Objectives
1 Primary · 5 Secondary · Reporting Duration: 1 years post-transplant.
Trial Safety
Safety Progress
Trial Design
2 Treatment Groups
Arm 1 / Group 1 Standard Risk
1 of 2
Arm 2 / Group 2 High Risk.
1 of 2
Active Control
Experimental Treatment
50 Total Participants · 2 Treatment Groups
Primary Treatment: Pheripheral blood stem cells · No Placebo Group · Phase 1 & 2
Trial Logistics
Trial Timeline
Who is running the clinical trial?
Eligibility Criteria
Age 4 - 65 · All Participants · 13 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:- Parta, Mark, Corin Kelly, Nana Kwatemaa, Narda Theobald, Diane Hilligoss, Jing Qin, Douglas B. Kuhns, et al.. 2017. “Allogeneic Reduced-intensity Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease: A Single-center Prospective Trial”. Journal of Clinical Immunology. Springer Science and Business Media LLC. doi:10.1007/s10875-017-0422-6.
- Chiesa, Robert, Junfeng Wang, Henric-Jan Blok, Sheree Hazelaar, Benedicte Neven, Despina Moshous, Ansgar Schulz, et al.. 2020. “Hematopoietic Cell Transplantation in Chronic Granulomatous Disease: A Study of 712 Children and Adults”. Blood. American Society of Hematology. doi:10.1182/blood.2020005590.
- Kadauke, Stephan, Regina M. Myers, Yimei Li, Richard Aplenc, Diane Baniewicz, David M. Barrett, Allison Barz Leahy, et al.. 2021. “Risk-adapted Preemptive Tocilizumab to Prevent Severe Cytokine Release Syndrome After CTL019 for Pediatric B-cell Acute Lymphoblastic Leukemia: A Prospective Clinical Trial”. Journal of Clinical Oncology. American Society of Clinical Oncology (ASCO). doi:10.1200/jco.20.02477.
- Chiesa R, Wang J, Blok HJ, Hazelaar S, Neven B, Moshous D, Schulz A, Hoenig M, Hauck F, Al Seraihy A, Gozdzik J, Ljungman P, Lindemans CA, Fernandes JF, Kalwak K, Strahm B, Schanz U, Sedlacek P, Sykora KW, Aksoylar S, Locatelli F, Stepensky P, Wynn R, Lum SH, Zecca M, Porta F, Taskinen M, Gibson B, Matthes S, Karakukcu M, Hauri-Hohl M, Veys P, Gennery AR, Lucchini G, Felber M, Albert MH, Balashov D, Lankester A, Gungor T, Slatter MA. Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults. Blood. 2020 Sep 3;136(10):1201-1211. doi: 10.1182/blood.2020005590.
- 2022. "Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05463133.
Frequently Asked Questions
What is the total sample size of this experiment?
"Affirmative, clinicaltrials.gov data indicates that this medical trial is actively recruiting its targeted 50 patients from a single site. It has been available since 8th July 2022 and was last modified on 23rd July 2022." - Anonymous Online Contributor
Who is able to partake in this clinical analysis?
"To be considered for this study, applicants must possess granuloma and have a minimum age of 4 years old up to 65. Approximately 50 people will join the trial." - Anonymous Online Contributor
Are there any additional opportunities for enrolment in this clinical research?
"Affirmative, the data published on clinicaltrials.gov affirms that this research project is actively searching for volunteers. It was initially posted on July 8th 2022 and recently amended on July 23rd 2022. The investigation seeks 50 participants from just one site." - Anonymous Online Contributor
Does this medical research accept participants who have reached the age of majority?
"This medical trial is seeking participants who are between 4 and 65 years old." - Anonymous Online Contributor