Summary
Cyclosporine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Alpha-1 Antitrypsin Deficiency (AATD) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Alpha-1 Antitrypsin Deficiency (AATD). No registered clinical trial in this database tests Cyclosporine specifically in Alpha-1 Antitrypsin Deficiency (AATD). The evidence below comes from published literature only, so any clinical signal has not yet been tested prospectively against this condition in a registered study. The literature layer holds 2 publications for this pair: 2 clinical trial publications. Publication years run from 1995 to 2011. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Alpha-1 Antitrypsin Deficiency ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Progressive lung and liver disease; ZZ genotype: FEV1 decline accelerated (80-100 mL/year vs 30 mL/year in non-smokers); liver cirrhosis progressive; no spontaneous resolution without augmentation therapy.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 24.0 (trials 0.0, literature 4.0, tier 15, approved bonus 5.0) |
| Registered trials | 0 total: 0 recruiting, 0 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 2 (2 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | ABCB4 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
No registered trial links Cyclosporine to Alpha-1 Antitrypsin Deficiency (AATD) in the current OSMF trial extract.
Published literature
- Clinical trial publication Long-term outcome in 42 pediatric liver transplant patients with alpha 1-antitrypsin deficiency: a single-center experienceHughes MG Jr, Khan KM, Gruessner AC et al. · Clinical transplantation · 2011 · PMID 21077958
- Clinical trial publication Weaning of immunosuppression in long-term liver transplant recipientsRamos HC, Reyes J, Abu-Elmagd K et al. · Transplantation · 1995 · PMID 7839442
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target ABCB4. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Cyclosporine approved for Alpha-1 Antitrypsin Deficiency (AATD)?
Cyclosporine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Alpha-1 Antitrypsin Deficiency (AATD) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Alpha-1 Antitrypsin Deficiency (AATD). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Cyclosporine in clinical trials for Alpha-1 Antitrypsin Deficiency (AATD)?
No registered clinical trial in this database tests Cyclosporine specifically in Alpha-1 Antitrypsin Deficiency (AATD). The evidence below comes from published literature only, so any clinical signal has not yet been tested prospectively against this condition in a registered study.
What does the evidence show for Cyclosporine in Alpha-1 Antitrypsin Deficiency (AATD)?
The record for Cyclosporine in Alpha-1 Antitrypsin Deficiency (AATD) rests on 2 publications without a registered trial. This is a lead for hypothesis generation, not evidence of efficacy. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target ABCB4. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.