Drug Trials Snapshots: DEFENCATH
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.
Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the DEFENCATH Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).
Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).
DEFENCATH (taurolidine 1.35% and heparin 5,000 Units/5 mL [1,000 Units/mL])
de fen’ kath
CorMedix Inc.
Original Approval date: November 15, 2023
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
DEFENCATH is a catheter lock solution (CLS) to reduce the incidence of catheter-related bloodstream infections (CRBSI) in adult patients with kidney failure receiving chronic hemodialysis (HD) through a central venous catheter (CVC).
How is this drug used?
DEFENCATH is a CLS that is instilled into each catheter lumen by a health care professional at the conclusion of each HD session for patients with kidney failure requiring chronic HD. Prior to initiation of the next HD session, it must be aspirated from the catheter and discarded.
Who participated in the clinical trials?
The FDA approved DEFENCATH based on evidence from a single clinical trial of 806 patients with kidney failure receiving HD therapy through a CVC. The trial was conducted at 60 sites in the United States.
How were the trials designed?
The benefits and side effects of AUGTYRO for patients with NSCLC that are ROS1-positive were evaluated in one clinical trial. Some patients were previously treated with a drug that inhibits ROS1 for their ROS1-positive NSCLC (pretreated subgroup) and some had not received prior treatment with a drug that inhibits ROS1 (treatment-naïve subgroup). All patients received AUGTYRO by mouth once daily for 14 days, then increased to 160 mg twice daily until either cancer progression or intolerable side effects.
The benefit of AUGTYRO was evaluated by measuring the percentage of patients who had complete or partial shrinkage of their tumors (overall response rate or ORR) and by measuring the duration of that benefit (duration of response or DOR).
How were the trials designed?
The benefit and side effects of DEFENCATH were evaluated in a single clinical trial of adult patients with kidney failure receiving chronic HD therapy through a CVC. Patients were randomly assigned to receive DEFENCATH or heparin as a CLS at the end of each HD session. Neither the patients nor the health care providers knew which treatment was being given until after the trial was completed. The benefit of DEFENCATH was assessed based on the rate of bloodstream infections, comparing patients in the DEFENCATH and heparin groups.
The efficacy and safety of DEFENCATH for reducing the incidence of CRBSI in patients with kidney failure receiving chronic HD was evaluated in a randomized, double-blind, active‑controlled, multicenter trial. Patients were assigned to receive either DEFENCATH or heparin as a CLS. The assigned study medicine was instilled into the HD CVC at the end of all dialysis sessions and discarded prior to the initiation of the next dialysis session. The cases of CRBSI were assessed by a clinical adjudication committee (CAC). The benefit of DEFENCATH was assessed based on time to CAC-adjudicated CRBSI.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many male and female patients were in the clinical trial used to evaluate efficacy and safety.
Figure 1. Baseline Demographics by Sex
Source: Adapted from FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trial used to evaluate efficacy and safety.
Figure 2. Baseline Demographics by Race
Source: Adapted from FDA Review
Figure 3 summarizes the percentage of patients by age enrolled in the clinical trial used to evaluate efficacy and safety.
Figure 3. Baseline Demographics by Age
Source: Adapted from FDA Review
Figure 4 summarizes the percentage of patients by ethnicity enrolled in the clinical trial used to evaluate efficacy and safety.
Figure 4. Baseline Demographics by Ethnicity
Source: Adapted from FDA Review
Who participated in the trials?
Demographics of patients are presented in Table 1.
Table 1. Baseline Demographics by Age, Race, Sex, and Ethnicity
|
Demographic Parameters |
DEFENCATH N=403 n (%) |
Heparin N=403 n (%) |
Total N=806 n (%) |
| Sex | |||
| Male | 219 (54) | 249 (62) | 468 (58) |
| Female | 184 (46) | 154 (38) | 338 (42) |
| Race | |||
| White | 248 (62) | 262 (65) | 510 (63) |
| Black or African American | 126 (31) | 112 (28) | 238 (30) |
| Asian | 15 (4) | 18 (4) | 33 (4) |
| American Indian or Alaska Native | 3 (1) | 2 (<1) | 5 (1) |
| Native Hawaiian or other Pacific Islander | 10 (2) | 4 (1) | 14 (2) |
| Other | 1 (<1) | 5 (1) | 6 (1) |
| Age, years | |||
| <65 | 239 (59) | 236 (59) | 475 (59) |
| ≥65 | 164 (41) | 167 (41) | 331 (41) |
| Ethnicity | |||
| Hispanic or Latino | 117 (44) | 189 (47) | 366 (45) |
| Not Hispanic or Latino | 226 (56) | 214 (53) | 440 (55) |
Source: Adapted from FDA Review
What are the benefits of this drug?
Over the course of the study, patients treated with DEFENCATH as a CSL experienced fewer CRBSIs in comparison to patients who received heparin as a CSL.
What are the benefits of this drug (results of trials used to assess efficacy)?
Table 2 summarizes efficacy results for the evaluated patients in the clinical trial.
Table 2. Efficacy Results, Efficacy Population
|
Parameter |
DEFENCATH N=397 |
Heparin N=398 |
| CAC-adjudicated CRBSI, n (%) | 9 (2.3) | 32 (8.0) |
| Event rate per 1000 catheter-days (95% CI) | 0.13 (0.07, 0.26) | 0.46 (0.33, 0.66) |
| Risk reduction (95% CI)* | 71 (38, 86) | |
| Log-rank test p-value | 0.0006 |
Source: Adapted from FDA Review
Based on 1 – Hazard Ratio
Abbreviations: CAC, clinical adjudication committee; CI, confidence interval; CRBSI, catheter-related bloodstream infection
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: The observed effect of DEFENCATH was similar for females and males.
- Race: DEFENCATH worked similarly in White and Black or African American patients. The number of patients in other races were limited; therefore, differences in how DEFENCATH worked among other races could not be determined.
- Age: The observed effect of DEFENCATH was larger in patients older than 65 years of age than in patients younger than 65 years of age. Because of limited data, this difference may be due to chance.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
Table 3 summarizes efficacy results by sex, race, age, and ethnicity.
Table 3. Efficacy Results by Sex, Race, Age, and Ethnicity, Efficacy Population
| DEFENCATH, N=397 | Heparin, N=398 | |||
| Subgroup | Events1 n/N (%) | Event1 Rate Per 1000 Catheter‑Days (95% CI) | Events1 n/N (%) | Event1 Rate per 1000 Catheter‑Days (95% CI) |
| Age, years | ||||
| <65 | 6/235 (2.6) | 0.15 (0.07, 0.34) | 14/233 (6.0) | 0.36 (0.21, 0.60) |
| ≥65 to <75 | 2/98 (2.0) | 0.12 (0.03, 0.47) | 10/94 (10.6) | 0.63 (0.34, 1.16) |
| <75 | 1/64 (1.6) | 0.09 (0.01, 0.65) | 8/71 (11.3) | 0.58 (0.29, 1.17) |
| Race | ||||
| White | 6/244 (2.5) | 0.14 (0.06, 0.32) | 19/259 (7.3) | 0.41 (0.26, 0.65) |
| Black or African American | 3/124 (2.4) | 0.15 (0.05, 0.46) | 9/110 (8.2) | 0.50 (0.26, 0.96) |
| Other | 0/29 (0) | 0 | 4/29 (13.8) | 0.82 (0.31, 2.18) |
| Sex | ||||
| Male | 3/215 (1.4) | 0.08 (0.03, 0.25) | 14/246 (5.7) | 0.33 (0.19, 0.55) |
| Female | 6/182 (3.3) | 0.19 (0.09, 0.43) | 18/152 (11.8) | 0.69 (0.44, 1.10) |
Source: Adapted from FDA Review
1 Event refers to the CAC-adjudicated CRBSI.
2 Other races included Asian, American Indian or Alaska Native, Native Hawaiian or other Pacific Islander, and other.
Abbreviations: CAC, clinical adjudication committee; CI, confidence interval; CRBSI, catheter-related bloodstream infection
What are the possible side effects?
The most common side effect was malfunctioning of the hemodialysis catheter. Other side effects include bleeding, low platelet count, nausea, vomiting, dizziness, and musculoskeletal chest pain.
What are the possible side effects (results of trials used to assess safety)?
Table 4 summarizes safety results for the evaluated patients in the clinical trial.
Table 4. Safety Results, Safety Population
| Adverse Reactions | DEFENCATH N=398 n (%) | Heparin N=399 n (%) |
| Product Issues | ||
| Hemodialysis catheter malfunction | 68 (17) | 47 (12) |
| Blood and lymphatic system disorders | ||
| Hemorrhage or bleeding | 27 (7) | 34 (9) |
| Thrombocytopenia | 7 (2) | 4 (1) |
| Gastrointestinal disorders | ||
| Nausea | 28 (7) | 44 (11) |
| Vomiting | 24 (6) | 32 (8) |
| Nervous system disorders | ||
| Dizziness | 22 (6) | 16 (4) |
| Musculoskeletal and connective tissue disorders | ||
| Musculoskeletal chest pain | 11 (3) | 7 (2) |
Source: Adapted from FDA Review
Were there any differences in side effects among sex, race, and age?
- Sex: The occurrence of side effects was similar in males and females.
- Race: The majority of patients were White or Black or African American. The occurrence of hemodialysis catheter malfunction, vomiting, and hypertension was higher in the Black or African American subgroup. Because of limited data these differences may be due to chance.
- Age: The occurrence of hemodialysis catheter malfunction was similar across all age groups. The ≥75-year-old age group had higher incidence of bleeding compared to the other age groups. Because of limited data this difference may be due to chance.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Table 5, Table 6, and Table 7 summarize the adverse events by sex, race, and age, respectively.
Table 5. Side Effects by Treatment Group and Sex, Safety Population
| DEFENCATH | Heparin | |||
| Adverse Event | Female N=183 n (%) | Male N=215 n (%) | Female N=153 n (%) | Male N=246 n (%) |
| Hemodialysis catheter malfunction | 29 (16) | 39 (18) | 20 (13) | 27 (11) |
| Diarrhea | 15 (8) | 23 (11) | 12 (8) | 18 (7) |
| Hypotension | 13 (7) | 23 (11) | 6 (4) | 23 (9) |
| Hypertension | 13 (7) | 15 (7) | 9 (6) | 30 (12) |
| Nausea | 12 (7) | 17 (8) | 17 (11) | 29 (12) |
Source: Adapted from FDA Review
Table 6. Side Effects by Treatment Group and Race, Safety Population
| DEFENCATH | Heparin | |||
| Adverse Event | Black or African American N=125 n (%) | White N=244 n (%) | Black or African American N=111 n (%) | White N=259 n (%) |
| Hemodialysis catheter malfunction | 29 (23) | 33 (14) | 17 (15) | 27 (10) |
| Hypotension | 13 (10) | 20 (8) | 7 (6) | 17 (6) |
| Vomiting | 13 (10) | 11 (5) | 7 (6) | 22 (9) |
| Diarrhea | 12 (10) | 25 (10) | 11 (10) | 17 (7) |
| Hypertension | 12 (10) | 15 (6) | 13 (12) | 23 (9) |
| Muscle spasms | 11 (9) | 15 (6) | 12 (11) | 21 (8) |
| Nausea | 7 (6) | 21 (9) | 11 (10) | 32 (12) |
| Hemorrhage or bleeding | 7 (6) | 19 (8) | 12 (11) | 22 (9) |
Source: Adapted from FDA Review
Table 7. Side Effects by Treatment Group and Age, Safety Population
| DEFENCATH | Heparin | |||||
| Adverse Event | <65 N=236 n (%) | ≥65 to <75 N=98 n (%) | ≥75 N=64 n (%) | <65 N=234 n (%) | ≥65 to <75 N=94 n (%) | ≥75 N=71 n (%) |
| Hemodialysis catheter malfunction | 43 (18) | 14 (14) | 11 (17) | 28 (12) | 6 (6) | 13 (18) |
| Diarrhea | 20 (9) | 13 (13) | 5 (8) | 20 (9) | 8 (9) | 2 (3) |
| Hypotension | 19 (8) | 13 (13) | 4 (6) | 19 (8) | 6 (6) | 4 (5) |
| Nausea | 19 (8) | 5 (5) | 5 (8) | 25 (11) | 15 (16) | 6 (9) |
| Hypertension | 19 (8) | 7 (7) | 2 (3) | 26 (11) | 12 (13) | 1 (1) |
| Hemorrhage or bleeding | 13 (6) | 7 (7) | 8 (13) | 12 (5) | 9 (10) | 13 (18) |
| Vomiting | 13 (6) | 8 (8) | 3 (5) | 15 (6) | 13 (14) | 5 (7) |
| Pneumonia | 10 (4) | 3 (3) | 5 (8) | 11 (5) | 8 (9) | 9 (13) |
Source: Adapted from FDA Review
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
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