Fetal Cystoscopy for Severe Lower Urinary Tract Obstruction

(CYSTO Trial)

Not currently recruiting at 2 trial locations

Overseen By Rodrigo A Ruano, MD, PhD.

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: Rodrigo Ruano M.D., Ph.D

Disqualifiers: Fetal anomaly, Cardiac anomaly, Female fetus, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the use of fetal cystoscopy, a procedure using a small camera to examine and potentially treat severe blockages in the lower urinary tract of unborn babies. The goal is to understand how this intervention affects both mothers and their babies with this specific condition. Pregnant women carrying a male fetus diagnosed with severe lower urinary tract obstruction, a dilated bladder, and certain other medical conditions might be suitable candidates. Participants should have already considered and declined pregnancy termination and must have sufficient social support. As an unphased trial, this study allows participants to contribute to groundbreaking research that could improve outcomes for future patients facing similar challenges.

Do I need to stop my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that fetal cystoscopy is safe for maternal and fetal patients?

Research shows that fetal cystoscopy primarily addresses severe blockages in an unborn baby's urinary tract, especially when the baby's kidneys function well. Studies have examined the safety and effectiveness of this procedure. It remains experimental and targets serious urinary issues in unborn babies.

Previous studies indicate that fetal cystoscopy can be challenging, but it aims to prevent problems at birth. Researchers are assessing its safety during pregnancy. As an experimental treatment, it is still under careful study. Although extensive safety data is not yet available, the procedure undergoes testing to ensure maximum safety for both mother and baby. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Fetal cystoscopy is unique because it offers a direct approach to treating severe lower urinary tract obstruction in unborn babies. Unlike traditional treatments, which might rely on medications or waiting for surgical intervention after birth, fetal cystoscopy allows doctors to intervene while the baby is still in the womb. This method involves using a tiny camera to visualize and potentially relieve the obstruction early in gestation. Researchers are excited about this technique because it could improve outcomes by addressing the problem sooner, potentially reducing complications and improving kidney function in affected infants.

What evidence suggests that fetal cystoscopy is effective for severe lower urinary tract obstruction?

Research has shown that fetal cystoscopy, which participants in this trial will undergo, can help with severe lower urinary tract blockage in babies when their kidney function remains good. This procedure aims to clear the blockage and improve the baby's health. Studies suggest it might help prevent death around the time of birth in these cases. However, fetal cystoscopy is a complex procedure and is mainly used when other options, like creating a drainage path, are unsuitable. While promising, it remains experimental, and more research is needed to confirm its benefits. ^{1 2 3 4 5}

Who Is on the Research Team?

Rodrigo Ruano, MD, Ph.D

Principal Investigator

University of Miami

Are You a Good Fit for This Trial?

This trial is for pregnant women over 18 with a male fetus diagnosed with severe lower urinary tract obstruction (LUTO). The fetus must show specific signs on ultrasound, have favorable urine analysis results, and no chromosomal abnormalities. Gestational age should be between 16-25 weeks. Women must not have medical or pregnancy-related risks that could complicate surgery.

Inclusion Criteria

You have too little or no amniotic fluid in the womb.

Pregnant women with singleton pregnancy

The pregnancy is between 16 and 25 weeks along when the procedure is done.

Exclusion Criteria

I cannot have fetoscopic surgery due to conditions like uterine fibroids or anomalies.

I do not have HIV or Hepatitis, or I have tested negative for them.

I don't have insurance for routine clinical care including prenatal and delivery services.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Fetal Cystoscopy Procedure

Participants undergo fetal cystoscopy procedure to address severe lower urinary tract obstruction

Day 1

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after the fetal cystoscopy procedure

Up to 24 months post delivery

What Are the Treatments Tested in This Trial?

Interventions

• Fetal Cystoscopy

Trial Overview The study tests fetal cystoscopy using fetoscopes to treat severe LUTO in unborn males. It aims to evaluate the outcomes of this prenatal intervention compared to standard care, focusing on both maternal and fetal health after the procedure.

How Is the Trial Designed?

Group I: Fetal Cystoscopy Group Experimental Treatment 2 Interventions

Participants in this group will receive the fetal cystoscopy procedure around 16 weeks 0 days to 25 weeks and 6 days of gestation.

Fetal Cystoscopy is already approved in European Union, United States for the following indications:

Approved in European Union as Fetal Cystoscopy for:

• Severe Lower Urinary Tract Obstruction (LUTO)

Approved in United States as Fetal Cystoscopy for:

• Severe Lower Urinary Tract Obstruction (LUTO)

Who Is Running the Clinical Trial?

Rodrigo Ruano M.D., Ph.D

Lead Sponsor

Trials

2

Recruited

20+

Rodrigo Ruano

Lead Sponsor

Trials

5

Recruited

40+

Published Research Related to This Trial

Fetal cystoscopy can change the ultrasound diagnosis of congenital lower urinary tract obstruction in a significant percentage of cases, with alterations noted in 36.4% and 25.0% of fetuses in two studies.

While fetal cystoscopy showed a high odds ratio for improved perinatal survival compared to no treatment (20.51), it did not demonstrate a significant advantage over vesicoamniotic shunt, indicating that its effectiveness as a therapeutic intervention remains uncertain and requires further research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of fetal cystoscopy as a diagnostic and therapeutic intervention for lower urinary tract obstruction: a systematic review. Morris, RK., Ruano, R., Kilby, MD.[2022]

Fetal cystoscopy and vesicoamniotic shunting significantly improve the 6-month survival rate in fetuses with severe lower urinary obstruction (LUTO), with adjusted relative risks of 1.86 and 1.73 respectively compared to no intervention.

Fetal cystoscopy is particularly effective in preserving normal renal function in cases of posterior urethral valves, showing a significant improvement in both survival and renal function, while vesicoamniotic shunting only improved survival without affecting renal outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fetal intervention for severe lower urinary tract obstruction: a multicenter case-control study comparing fetal cystoscopy with vesicoamniotic shunting. Ruano, R., Sananes, N., Sangi-Haghpeykar, H., et al.[2016]

Fetal cystoscopy accurately diagnosed the cause of lower urinary tract obstruction (LUTO) in 91.4% of cases, allowing for targeted prenatal treatment.

Among fetuses diagnosed with posterior urethral valves (PUV), 56.7% survived to infancy, with 76.5% of those having normal renal function at 1 year, indicating that fetal cystoscopy can lead to improved outcomes for certain conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Two-year outcomes after diagnostic and therapeutic fetal cystoscopy for lower urinary tract obstruction. Sananes, N., Cruz-Martinez, R., Favre, R., et al.[2017]

Percutaneous fetal cystoscopy was successfully performed in 11 out of 13 fetuses with lower obstructive uropathy, allowing for direct visualization of the urinary tract and identification of conditions that ultrasound could not clarify.

The procedure not only helped diagnose issues like ureteral dilation and webs but also facilitated treatment options, such as urethral vesicoamniotic shunting, demonstrating its potential as a valuable tool in managing fetal urinary obstructions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In-utero percutaneous cystoscopy in the management of fetal lower obstructive uropathy. Quintero, RA., Johnson, MP., Romero, R., et al.[2019]

Fetoscopic techniques allow for safer prenatal surgical interventions by avoiding the risks associated with traditional open surgery, making it possible to treat conditions like congenital obstructive uropathy earlier in gestation.

In a study involving midgestation fetal lambs, an endoscopic approach using a new expandable wire mesh stent successfully corrected obstructive uropathy, suggesting that this method could improve bladder drainage and reduce the risks of preterm labor in fetuses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fetoscopic surgery for the treatment of congenital anomalies. Estes, JM., MacGillivray, TE., Hedrick, MH., et al.[2019]

A successful case of percutaneous fetal cystoscopy was performed on a male fetus at 19 weeks of gestation, confirming the diagnosis of posterior urethral valves and allowing for therapeutic intervention at 22 weeks.

This case demonstrates the potential of fetal cystoscopy as a feasible method for diagnosing and treating congenital urinary tract anomalies, although further studies are necessary to evaluate its overall effectiveness and safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Percutaneous fetal cystoscopy and endoscopic fulguration of posterior urethral valves. Quintero, RA., Hume, R., Smith, C., et al.[2019]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11717740/

Indications and Outcomes of Fetal Cystoscopy for Lower ... The evidence suggests that fetal cystoscopy is a challenging procedure, mainly indicated for severe LUTO with preserved fetal renal function.

2. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT03281798

Fetal Cystoscopy for Severe Lower Urinary Tract ... The purpose of the study is to study the outcomes of maternal and fetal patients who are undergoing fetal intervention for severe isolated lower urinary tract ...

3. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/abs/10.1002/pd.6721

Indications and Outcomes of Fetal Cystoscopy for Lower ... Current management strategies, including fetal vesicoamniotic shunting (VAS) and fetal cystoscopy, aim to alleviate obstruction and mitigate ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02446184

Fetal Cystoscopy for Lower Urinary Tract Obstruction This is a pilot study to evaluate the safety, feasibility, and effectiveness of fetal cystoscopy as an experimental procedure in avoiding perinatal death and ...

5. mayo.edu mayo.edu/research/clinical-trials/cls-20317022

Fetal Cystoscopy for Severe Lower Urinary Tract ... The purpose of the study is to study the outcomes of maternal and fetal patients who are undergoing fetal intervention for severe, isolated lower urinary tract ...