Clinical trial NCT04592640: Stem Cells for Uremic Calciphylaxis Patients
Official title: Effects of Human Amniotic Mesenchymal Stem Cells (hAMSCs) on Wound Healing in Uremic Calciphylaxis Patients: an Open Label Single Arm Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Chronic Kidney Diseases
Calciphylaxis
Calcific Uremic Arteriolopathy
Treatment
Keywords: Chronic Kidney Diseases
Calciphylaxis
Calcific Uremic Arteriolopathy
Human Amniotic Mesenchymal Stem Cells
Rare disease (ORPHA280062)
Lead sponsor: The First Affiliated Hospital with Nanjing Medical University
Sponsor class: OTHER
First posted: 2020-10-19
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Treatment for Uremic Calciphylaxis Patients with Human Amniotic Mesenchymal Stem Cells

Detailed description: Calciphylaxis is a rare, devastating disorder causing excruciatingly painful ischemic skin lesions. Sepsis due to the infection of ulcerated wounds is a common cause of death. To date, practical therapies typically include wound care, pain management, anti-infection and aggressive treatment of predisposing conditions. Drugs such as sodium thiosulfate, bisphosphonate, and cinacalcet are also suggested. However, it's still a lethal disease with 1-year mortality up to 80% for dialysis patients. Here the investigators plan to treat uremic calciphylaxis patients with human amniotic mesenchymal stem cells (hAMSCs).

Eligibility criteria: Inclusion Criteria:

1. 18-60 years old.
2. Patients with chronic kidney disease who did not or had regular dialysis (hemodialysis or peritoneal dialysis).
3. Clinical diagnosis of calciphylaxis.
4. All subjects signed informed consent.

Exclusion criteria:

1. Patients who refuse to sign informed consent.
2. Patients with malignant tumor or serious mental disease, cardiovascular disease, shock, abnormal liver function and secondary kidney disease.
3. Pregnant or lactating women of childbearing age.
4. Participation in another clinical trial with an experimental drug within 90 days prior the inclusion.

Primary outcomes:
measure: Wound Healing
timeFrame: Up to 1 year.
Secondary outcomes:
measure: Wound Pain
timeFrame: Up to 1 year.
measure: Measured Quality of Life
timeFrame: Up to 1 year.
measure: Systemic Infection
timeFrame: Up to 1 year.
measure: Survival State
timeFrame: From date of treatment until the date of die, assessed up to 1 year.
Other outcomes:
Locations:
city: Nanjing
country: China
facility: The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital
state: Jiangsu
Study officials:
affiliation: The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital
name: Ningning Wang, Professor
role: PRINCIPAL_INVESTIGATOR
affiliation: The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital
name: Jiayin Liu, Professor
role: STUDY_DIRECTOR
affiliation: The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital
name: Lianju Qin
role: PRINCIPAL_INVESTIGATOR
References:
citation: Nigwekar SU, Thadhani R, Brandenburg VM. Calciphylaxis. N Engl J Med. 2018 May 3;378(18):1704-1714. doi: 10.1056/NEJMra1505292. No abstract available.
citation: Seethapathy H, Brandenburg VM, Sinha S, El-Azhary RA, Nigwekar SU. Review: update on the management of calciphylaxis. QJM. 2019 Jan 1;112(1):29-34. doi: 10.1093/qjmed/hcy234.
citation: McCarthy JT, El-Azhary RA, Patzelt MT, Weaver AL, Albright RC, Bridges AD, Claus PL, Davis MD, Dillon JJ, El-Zoghby ZM, Hickson LJ, Kumar R, McBane RD, McCarthy-Fruin KA, McEvoy MT, Pittelkow MR, Wetter DA, Williams AW. Survival, Risk Factors, and Effect of Treatment in 101 Patients With Calciphylaxis. Mayo Clin Proc. 2016 Oct;91(10):1384-1394. doi: 10.1016/j.mayocp.2016.06.025.
citation: Baby D, Upadhyay M, Joseph MD, Asopa SJ, Choudhury BK, Rajguru JP, Gupta S. Calciphylaxis and its diagnosis: A review. J Family Med Prim Care. 2019 Sep 30;8(9):2763-2767. doi: 10.4103/jfmpc.jfmpc_588_19. eCollection 2019 Sep.
citation: Peng T, Zhuo L, Wang Y, Jun M, Li G, Wang L, Hong D. Systematic review of sodium thiosulfate in treating calciphylaxis in chronic kidney disease patients. Nephrology (Carlton). 2018 Jul;23(7):669-675. doi: 10.1111/nep.13081.
citation: Udomkarnjananun S, Kongnatthasate K, Praditpornsilpa K, Eiam-Ong S, Jaber BL, Susantitaphong P. Treatment of Calciphylaxis in CKD: A Systematic Review and Meta-analysis. Kidney Int Rep. 2018 Oct 9;4(2):231-244. doi: 10.1016/j.ekir.2018.10.002. eCollection 2019 Feb.
citation: Torregrosa JV, Sanchez-Escuredo A, Barros X, Blasco M, Campistol JM. Clinical management of calcific uremic arteriolopathy before and after therapeutic inclusion of bisphosphonates. Clin Nephrol. 2015 Apr;83(4):231-4. doi: 10.5414/CN107923.
citation: Naeem A, Gupta N, Naeem U, Khan MJ, Elrayess MA, Cui W, Albanese C. A comparison of isolation and culture protocols for human amniotic mesenchymal stem cells. Cell Cycle. 2022 Aug;21(15):1543-1556. doi: 10.1080/15384101.2022.2060641. Epub 2022 Apr 12.