Fetal Cystoscopy for Severe Lower Urinary Tract Obstruction

(CYSTO Trial)

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

10 Participants Needed

Trial Summary

What is the purpose of this trial?

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 obstruction (LUTO).

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 data supports the effectiveness of the treatment Fetal Cystoscopy for Severe Lower Urinary Tract Obstruction?

Research shows that fetal cystoscopy can accurately diagnose and treat lower urinary tract obstructions in unborn babies, improving outcomes by allowing for early intervention. Studies have demonstrated its ability to confirm diagnoses and successfully perform procedures to relieve obstructions, suggesting it is a promising treatment option. ^{1 2 3 4 5}

Is fetal cystoscopy generally safe for humans?

Fetal cystoscopy has been performed successfully in some cases to treat lower urinary tract obstructions in fetuses, but more studies are needed to fully understand its risks and limitations. It is considered less invasive than open fetal surgery, which may reduce the risk of preterm labor. ^{1 2 4 5 6}

How is fetal cystoscopy different from other treatments for severe lower urinary tract obstruction?

Fetal cystoscopy is unique because it allows doctors to directly look inside the fetus's bladder and urinary tract to diagnose and treat blockages before birth, unlike other treatments that may only involve shunting fluid around the obstruction. This approach can help identify the exact cause of the blockage and potentially treat it directly, which is not possible with standard ultrasound or shunting techniques. ^{1 2 3 4 5}

Research Team

Rodrigo Ruano, MD, Ph.D

Principal Investigator

University of Miami

Eligibility Criteria

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

My recent urine tests show low levels of sodium, chloride, and osmolality.

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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 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]

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

In-utero percutaneous cystoscopy in the management of fetal lower obstructive uropathy. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Effectiveness of fetal cystoscopy as a diagnostic and therapeutic intervention for lower urinary tract obstruction: a systematic review. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Two-year outcomes after diagnostic and therapeutic fetal cystoscopy for lower urinary tract obstruction. [2017]

4. United States pubmed.ncbi.nlm.nih.gov

Percutaneous fetal cystoscopy and endoscopic fulguration of posterior urethral valves. [2019]

5. England pubmed.ncbi.nlm.nih.gov

Fetal intervention for severe lower urinary tract obstruction: a multicenter case-control study comparing fetal cystoscopy with vesicoamniotic shunting. [2016]

6. United States pubmed.ncbi.nlm.nih.gov

Fetoscopic surgery for the treatment of congenital anomalies. [2019]