Calcific Uremic Arteriolopathy, Calciphylaxis, Chronic Kidney Diseases, Efficacy, Safety, Treatment
Conditions
Keywords
Chronic Kidney Diseases, Calciphylaxis, Calcific Uremic Arteriolopathy, Human Amniotic-derived Mesenchymal Stem Cells, Rare disease (ORPHA280062), Safety, Efficacy
Brief summary
Treatment for Calciphylaxis Patients with Human Amniotic-derived 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, anticoagulant and aggressive treatment of predisposing conditions. Drugs such as sodium thiosulfate(STS), bisphosphonate, SNF472 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 calciphylaxis patients with human amniotic-derived mesenchymal stem cells (hAMSCs).
Interventions
BIOLOGICALHuman amniotic-derived mesenchymal stem cells
(1) Intravenous infusion: 1.0×10⁶ cells/kg administered three times consecutively at weeks 1, 2, and 4, followed by once every 4 weeks for a total of 8 doses (6 months). (2) Local injection: 2.0×10⁴ cells/cm² of wound area, administered concurrently. Treatment may be terminated earlier or extended based on clinical condition.
Sponsors
The First Affiliated Hospital with Nanjing Medical University
Westlake University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. 18-75 years old. 2. Clinical diagnosis of calciphylaxis, including patients with chronic kidney disease who did not or had regular dialysis (hemodialysis or peritoneal dialysis). 3. All subjects signed informed consent.
Exclusion criteria
1. Patients who refuse to sign informed consent. 2. Patients with malignant tumors or severe psychiatric disorders, or an expected survival time of less than 6 months. 3. Pregnant or lactating women of childbearing age. 4. Participation in another clinical trial with an experimental drug within 90 days prior the inclusion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Wound Healing | Up to 1 year. | The Bates-Jensen Wound Assessment (BWAT) is a standardized tool for quantitative assessment of wound healing that includes the 13 items listed below: (1)Size; (2)Depth; (3)Edges; (4)Undermining or pockets; (5)Necrotic tissue type; (6)Necrotic tissue amount; (7)Exudate type; (8)Exudate amount; (9)Surrounding skin color; (10)Peripheral tissue edema; (11)Peripheral tissue induration; (12)Granulation tissue; (13)Epithelialization. Each item was rated on a scale of 1 (best) to 5 (worst). The BWAT total score is the sum of the individual items with a possible range of 13 (best) to 65 (worst). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Wound Pain | Up to 1 year. | Assessed by pain visual analog scale (VAS). VAS Score is a horizontal line, 100 mm in length, anchored by word descriptors at each end. The subject marked the point on the line that represented his/her perception of his/her current pain status. VAS Score was determined by measuring in millimeters from the left hand end of the line (no pain) to the point that the subject marked. VAS Score range 0 (best) to 100 (Worst). |
| Measured Quality of Life | Up to 1 year. | The wound-quality of life(QoL) questionnaire measures the disease-specific, health related QoL of patients with chronic wounds. It consists of 17 items on impairments that are assessed in retrospect to the preceding 7 days and rated on a 0 (best) to 4 (worse) scale with possible responses from "not at all" to "very much". The total score is the average of the 17 responses. |
| Systemic Infection | Up to 1 year. | Assessed by serum hypersensitive C-reactive protein level(hCRP, mg/L). Increased serum hCRP levels above normal ranges indicate infection. |
| Survival State | From date of treatment until the date of die, assessed up to 1 year. | Survival time from the hAMSCs treatment started(momths) |
Countries
China
Contacts
CONTACTNingning Wang, Professor
CONTACTLianju Qin, Professor
PRINCIPAL_INVESTIGATORNingning Wang, Professor
The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province Hospital
STUDY_DIRECTORJiayin Liu, Professor
The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province Hospital
PRINCIPAL_INVESTIGATORLianju Qin
The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province Hospital
Outcome results
None listed