NCT04324411

Sirolimus Combined With ATRA for the Treatment of Auto-Immune Anemia

Status unknown Phase 2, PHASE3 Last updated 27 March 2020

What this trial tests

Phase 2, PHASE3 trial testing sirolimus and ATRA in Autoimmune Anemia in 50 participants. Status unknown.

Timeline

1 April 2020

Primary endpoint
1 April 2022

1 December 2022

Quick facts

Lead sponsor	Peking Union Medical College Hospital
Phase	Phase 2, PHASE3
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	50
Start date	1 April 2020
Primary completion	1 April 2022
Estimated completion	1 December 2022
Sites	1 location across China

Drugs / interventions tested

• sirolimus and ATRA — full drug profile →

Conditions studied

• Autoimmune Anemia — all drugs for Autoimmune Anemia →

Sponsor

Peking Union Medical College Hospital

Who can join

Adults 18 to 70, any sex, with Autoimmune Anemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Autoimmune anemia (AIA), including autoimmune hemolytic anemia (AIHA), EVENs' syndrome (ES), acquired pure red aplastic anemia (PRCA), is a kind of anemia disease mediated by autoimmunity, which can be primary or secondary to other diseases including autoimmune disease, malignant tumor, infection, etc. Glucocorticoid is the first-line treatment. However, the recurrence rate is very high and some patients may not response to steroids, the latter defined as refractory autoimmune anemia (RAIA). Second-line therapies include cyclosporine A (CSA), cyclophosphamide, 6-mercaptopurine, CD20 monoclonal antibody, anti human lymphocyte immunoglobulin (ATG), and even splenectomy. Cyclosporine A is easy to accept while some patients may have side effects such as renal function damage, gingival hyperplasia, hypertension and so on. Other second-line drugs also have many problems, such as low effective rate, slow onset, expensive price, and large side effects, and some patients do not response to these treatments. The refractory/relapsed AIA patients have increased cardiovascular events, increased opportunities for infections, decreased quality of life, and even death. At present, there is still no effective treatment for these patients. Our previous retrospective study showed that sirolimus was effective in cyclosporine refractory PRCA with an effective rate of 70% and slight side effects. In addition, we used sirolimus in refractory AIHA and ES, with an effective rate of 60-70%. However, there are still some non-responsive patients. Recently, it has been reported that all trans retinoic acid (ATRA) combined with danazol was effective in the treatment of refractory immune thrombocytopenic purpura (ITP). Therefore, we plans to combine sirolimus and ATRA in the treatment of refractory AIA to improve the efficacy. Since both sirolimus and ATRA are cheap and have slight side effects, this combination may reduce the economic burden of patients and reduce the side effects related to treatment.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Autoimmune hemolytic anemia: current knowledge and perspectives.
   Michalak SS, Olewicz-Gawlik A, Rupa-Matysek J, Wolny-Rokicka E, et al · · 2020 · cited 72× · PMID 33292368 · DOI 10.1186/s12979-020-00208-7
2. All-trans retinoic acid in hematologic disorders: not just acute promyelocytic leukemia.
   Chen Y, Tong X, Lu R, Zhang Z, et al · · 2024 · cited 16× · PMID 39027338 · DOI 10.3389/fphar.2024.1404092