Clinical Trial: Renal Agenesis Fetal Therapy

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Renal Agenesis Fetal Therapy (RAFT) Trial

Brief Summary: This study offers eligible pregnant patients with a fetal diagnosis of congenital bilateral renal agenesis (CoBRA) serial amnioinfusions with normal saline. All eligible patients will be offered intervention but may also elect expectant management.

Detailed Summary:

In fetal life, urine production by the kidneys is responsible for sustaining amniotic fluid in the uterus. This fluid is critical for fetal lung development during gestation. Complete lack of amniotic fluid, known as anhydramnios, leads to lethal pulmonary hypoplasia in 100% of patients. A common cause of complete anhydramnios is congenital bilateral renal agenesis or CoBRA. CoBRA is relatively common complicating nearly 1 in every 3000 pregnancies. CoBRA has traditionally been considered a lethal condition as affected fetuses have pulmonary hypoplasia and in addition require renal transplant to sustain life. Several reports have suggested a beneficial effect of amnioinfusion for lung development in fetal states of low amniotic fluid. Accordingly, restoration of normal amniotic fluid volume with amnioinfusion may improve pulmonary mortality in patients with CoBRA. In terms of neonatal renal failure, however, the outlook has significantly improved over the last decade. Large studies have demonstrated that peritoneal dialysis and renal transplant have become highly successful strategies for neonates with little or no kidney function. Accordingly, there is a need to evaluate the efficacy of serial amnioinfusion to produce life-sustaining pulmonary function specifically in CoBRA patients.

The aim of this study is to evaluate the safety and efficacy of serial amnioinfusion to reliably prevent pulmonary hypoplasia in patients with isolated CoBRA. It is unknown if serial amnioinfusions can reliably prevent pulmonary hypoplasia in the setting of CoBRA. Similarly, it is not known if serial amnioinfusions will give neonates enough pulmonary reserve to be candidates for renal replacement therapy with acceptable levels of morbidity. The investigators hypothesize that serial amnioinfusions will reliably prevent lethal pulmonary hypoplasia in fetuses diagnosed with CoBRA with minimal m
Sponsor: Johns Hopkins University

Current Primary Outcome:

• Maternal safety: the rate of maternal adverse events [ Time Frame: These outcomes will be measured at every prenatal visit, after every prenatal procedure, at delivery and 30 days after delivery. ]
  To evaluate the safety of serial saline amnioinfusions in patients with CoBRA. Safety of the infusions will be determined by the rate of maternal adverse events (if any) with infusion. These events include bleeding, deep infection (chorioamnionitis), and superficial wound infection requiring antibiotics.
• Survival to dialysis [ Time Frame: Birth to either successful dialysis or nonsurvival. ]
  To evaluate the efficacy of serial saline amnioinfusions to prevent lethal pulmonary hypoplasia in patients with CoBRA. Survival or nonsurvival to the endpoint of successful dialysis will be measured.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Survival to transplant [ Time Frame: birth to either successful renal transplant (usually around 2 years of life) or nonsurvival ]
  To determine the proportion of patients treated with RAFT who successfully receive renal transplant.
• Quality of life using Pediatric Quality of Life Inventory 4.0 (PedsQL TM) survey [ Time Frame: 1 and 2 years of life and then 1, 2 3 and 4 years after transplant ]
  To determine the quality of life of patients that undergo RAFT. The parent proxy score will be used until the child is 5 years of age.

Original Secondary Outcome: Same as current

Information By: Johns Hopkins University

Dates:
Date Received: March 27, 2017
Date Started: March 18, 2017
Date Completion: March 18, 2026
Last Updated: March 30, 2017
Last Verified: March 2017