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Potassium Channel Blocker

Senicapoc for Dehydrated Stomatocytosis

Phase 1 & 2
Waitlist Available
Led By Carlo Brugnara, MD
Research Sponsored by Boston Children's Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients carrying KCNN4 mutations in V282M as described in 1981 by Snyder et al and Sauberman et al. and characterized molecularly by Andolfo et al. in 2015, and other patients with V282 mutations with demonstrated in-vitro sensitivity to senicapoc
Have a diagnosis of dehydrated stomatocytosis with a molecularly confirmed mutation in KCNN4
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 months
Awards & highlights

Study Summary

This trial is for a disorder called Dehydrated Stomatocytosis, which is a genetic disorder that causes chronic hemolysis, variable anemia, and erythrocyte dehydration. The goal of this trial is to see if Senicapoc, a drug that has been shown to be safe with limited side-effects, can help treat this disorder.

Who is the study for?
This trial is for adults over 21 with a specific genetic blood disorder called dehydrated stomatocytosis, confirmed by certain mutations. They must be able to follow the study plan and have signs of the disease like high mean corpuscular hemoglobin concentration (MCHC) or low haptoglobin. People can't join if they've had recent strokes, transfusions, major surgeries, drug/alcohol dependence, severe anemia, are pregnant/breastfeeding or have liver/renal issues.Check my eligibility
What is being tested?
The trial tests Senicapoc—a Gardos channel blocker—on patients with familial dehydrated stomatocytosis due to V282 mutations in KCNN4. It's a daily treatment aiming to show how well it works as proof-of-concept since previous trials in sickle cell disease didn't meet goals despite being safe.See study design
What are the potential side effects?
Senicapoc has been found safe with limited side effects in past studies on sickle cell disease. Specific side effects aren't listed but are expected to be mild based on previous research.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a specific KCNN4 gene mutation sensitive to senicapoc.
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I have dehydrated stomatocytosis with a confirmed KCNN4 mutation.
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I have blood issues related to dehydrated stomatocytosis, meeting at least 3 of the specific criteria listed.
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I am 21 years old or older.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
chronic hemolysis biomarkers
Secondary outcome measures
Decrease in the frequency and intensity of pain
Improved functional health and well-being
spleen volume

Trial Design

1Treatment groups
Experimental Treatment
Group I: TreatmentExperimental Treatment1 Intervention
This is a pivotal trial in members of the same family carrying the V282M nutation in the Gardos channel (KCNN4) and other patients with V282 mutations with demonstrated in-vitro sensitivity to senicapoc. These mutations lead to hyperactivation of the channel and red cell dehydration. Up to 6 patients are eligible to enroll in this study, which will assess effectiveness based on individual changes of primary endpoints over individually established baselines.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Senicapoc (synonyms: ICA-17043; 2,2-bis-(4-fluorophenyl)-2-phenylacetamide)
2021
Completed Phase 2
~10

Find a Location

Who is running the clinical trial?

Boston Children's HospitalLead Sponsor
756 Previous Clinical Trials
5,579,159 Total Patients Enrolled
Carlo Brugnara, MDPrincipal Investigator - Boston Children's Hospital
Boston Children's Hospital

Media Library

Senicapoc (Potassium Channel Blocker) Clinical Trial Eligibility Overview. Trial Name: NCT04372498 — Phase 1 & 2
Stomatocytosis Research Study Groups: Treatment
Stomatocytosis Clinical Trial 2023: Senicapoc Highlights & Side Effects. Trial Name: NCT04372498 — Phase 1 & 2
Senicapoc (Potassium Channel Blocker) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04372498 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this experiment still recruiting participants?

"Affirmative. Clinicaltrials.gov reveals that the medical study, which was first posted on April 15th 2021, is actively recruiting participants. 6 individuals from one centre must be enrolled in order to complete the trial."

Answered by AI

Are there any studies that have utilized the drug ICA-17043 (Senicapoc; 2,2-bis-(4-fluorophenyl)-2-phenylacetamide) in their research?

"Presently, there are two clinical trials for Senicapoc (ICA-17043; 2,2-bis-(4-fluorophenyl)-2-phenylacetamide) with no Phase 3 studies being conducted. There is a concentration of these studies in Boston, MA but three medical facilities across the United States have been approved to run trials for this drug molecule."

Answered by AI

How many participants are participating in this experiment?

"Affirmative. Information accessible on clinicaltrials.gov verifies that this research effort, initially posted to the website in April 2021, is currently enrolling participants. The study requires 6 individuals from a single location."

Answered by AI
~1 spots leftby Mar 2025