Clinical Trials of Liposomes in Children’s Anticancer Therapy: A Comprehensive Analysis of Trials Registered on ClinicalTrials.gov

Figures & data

Figure 1 Flowchart of trial selection.

Table 1 Characteristics of All Included Trials

Variable	Subgroup	N (%)
Year
	Prior to 2007	37 (50.0%)
	2007–2011	15 (20.3%)
	2012–2016	11 (14.9%)
	2017–2021	11 (14.9%)
Status
	Active, not recruiting	5 (6.8%)
	Completed	35 (47.3%)
	Recruiting	9 (12.2%)
	Suspended	1 (1.4%)
	Terminated	8 (10.8%)
	Withdrawn	3 (4.1%)
	Unknown status	13 (17.6%)
Study results
	Has results	17 (23.0%)
	No results available	57 (77.0%)
Funded by
	Industry	8 (10.8%)
	NIH	5 (6.8%)
	Other	30 (40.5%)
	Other + NIH	21 (28.4%)
	Other + industry	9 (12.2%)
	Industry + NIH	1 (1.4%)
Locations
	Asia	9 (12.2%)
	Europe	11 (14.9%)
	North America	51 (68.9%)
	Other	3 (4.1%)

Abbreviation: NIH, National Institutes of Health.

Table 2 Study Design Elements of Interventional Trials (n = 70)

Variable	Subgroup	N (%)
Primary purpose
	Prevention	2 (2.9%)
	Diagnostic	2 (2.9%)
	Supportive care	3 (4.3%)
	Treatment	63 (90.0%)
Allocation
	Randomized	21 (30.0%)
	Non-randomized	15 (21.4%)
	N/A	25 (35.7%)
	Unknown	9 (12.9%)
Intervention model
	Single group assignment	37 (52.9%)
	Parallel assignment	16 (22.9%)
	Crossover assignment	1 (1.4%)
	Sequential assignment	1 (1.4%)
	Unknown	15 (21.4%)
Masking
	Single	1 (1.4%)
	Double	4 (5.7%)
	Quadruple	2 (2.9%)
	None (open label)	50 (71.4%)
	Unknown	13 (18.6%)
Phases
	Phase 1	21 (30.0%)
	Phase 1/Phase 2	6 (8.6%)
	Phase 2	17 (24.3%)
	Phase 2/Phase 3	2 (2.9%)
	Phase 3	20 (28.6%)
	Phase 4	2 (2.9%)
	Not applicable	2 (2.9%)
Enrollment
	≤ 50	36 (51.4%)
	50–100	5 (7.1%)
	100–500	12 (17.1%)
	≥ 500	5 (7.1%)
	Unknown	12 (17.1%)

Table 3 Overview of Drugs for Prevention

Name and Number of the Investigated Drug	PubChem CID	Solubility (in Water)	Types (MeSH Tree)
Amphotericin B (3)	5280965	750 mg/L at 25 °C	Macrolides/Organic Chemicals
Annamycin (2)	115212	Not Available	Anthracyclines/Organic Chemicals
Bupivacaine (1)	2474	9.17 × 10^−5 mg/L at 25 °C	Anilides/Organic Chemicals
Cisplatin (2)	5702198	1 mg/mL	Platinum Compounds/Inorganic Chemicals
Cytarabine (4)	6253	1 × 10^6 mg/L at 25 °C	Nucleosides/Nucleic Acids
Daunorubicin (3)	30323	3.0 × 10^4 mg/L at 25 °C	Anthracyclines/Organic Chemicals
Doxorubicin (32)	31703	2600 mg/L at 25 °C	Anthracyclines/Organic Chemicals
Irinotecan (2)	60838	0.11 g/L	Alkaloids/Heterocyclic Compounds
Paclitaxel (1)	36314	5.56× 10^−3 g/L	Cycloparaffins/Organic Chemicals
Topotecan (1)	60700	2350 mg/L at 25 °C	Alkaloids/Heterocyclic Compounds
Tretinoin (1)	444795	2.48 × 10^−2 mg/L at 25 °C	Cycloparaffins/Organic Chemicals
Vincristine (12)	5978	2.27 mg/L at 25 °C	Alkaloids/Heterocyclic Compounds
W_ova1 Vaccine (1)	Not Available	Not Available	Biological Products/Complex Mixtures
DOTAP: Chol-fus1 (1)	Not Available	Not Available	Biological Products/Complex Mixtures
T4N5 lotion (1)	Not Available	Not Available	Bacteriophages & Enzymes
Daunorubicin-Cytarabine (2)	Complex Compounds	Complex Compounds	Complex Compounds
Doxorubicin-Daunorubicin(1)	Complex Compounds	Complex Compounds	Complex Compounds

Notes: Data from PubChem CID and Solubility (In water), http://www.ncbi.nlm.nih.gov/pccompound/; Data from MeSH Tree, https://meshb.nlm.nih.gov/.

Figure 2 Overview cancer types assessed for liposomal treatment for prevention (n = 123). The following cancers appeared only once: advanced cancer, bone cancer, germ cell tumors, glioma, invasive pulmonary aspergillosis, kidney tumor, lung cancer, multiple myeloma, nasopharyngeal carcinoma, pancreatic cancer, pediatric cancer, plasma cell neoplasm, precancerous condition, and prostate cancer.