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A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT03836001
Recruitment Status : Recruiting
First Posted : February 11, 2019
Last Update Posted : May 16, 2019
Sponsor:
Collaborators:
Epidermolysis Bullosa Research Partnership
Menlo Therapeutics
Information provided by (Responsible Party):
Albert Chiou, Stanford University

Brief Summary:
To determine if Serlopitant (when taken by mouth) is safe and works on itch in patients aged 13 and above with EB.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: Serlopitant Tablet Drug: Placebo Oral Tablet Phase 2

Detailed Description:

The investigator will determine whether more patients taking serlopitant 5 mg daily as compared to placebo can achieve a 3 point or greater reduction in itch severity as measured by numeric rating scale (NRS) score following two months of treatment.

Secondary objectives include;

  1. continuous change in NRS itch score over time,
  2. the proportion of patients who achieve at a least 30% or 50% reduction in NRS severity from baseline at the end of two months of treatment
  3. proportion of patients achieving 2 point and 4 point reductions in itch severity following two months of treatment, and
  4. change in median NRS score over time as compared to baseline.
  5. assessment on the safety of Serlopitant in adolescents (≥13 y.o.) and adults with pruritus associated with epidermolysis bullosa (EB).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is an investigator-initiated, single-center, randomized, double-blind, placebo controlled, parallel arm trial evaluating the effects of serlopitant at 5 mg by mouth daily on EB-related itch.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: This is a double-blind study.
Primary Purpose: Treatment
Official Title: A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
Actual Study Start Date : April 18, 2019
Estimated Primary Completion Date : September 30, 2021
Estimated Study Completion Date : March 28, 2022


Arm Intervention/treatment
Placebo Comparator: Placebo Oral Tablet
We aim to recruit at least 20 patients who will undergo two months of dosing with placebo (inactive drug or sugar pill), followed by one month of wash-out. All patients will be offered the option of participating in a 12-month open label extension with serlopitant at 5 mg (taken by mouth) daily for continued safety monitoring.
Drug: Placebo Oral Tablet
The placebo is a tablet that looks like a drug but has no drug or other active ingredient in it.
Other Name: Sugar pill

Active Comparator: Serlopitant Tablet
We aim to recruit at least 20 patients who will undergo two months of Serlopitant dosing, followed by one month of wash-out. All patients will be offered the option of participating in a 12-month open label extension with serlopitant 5 mg (taken by mouth) daily for continued safety monitoring.
Drug: Serlopitant Tablet
Serlopitant is a small molecule, highly selective NK1-R (neurokinin-1 receptor) antagonist. Two critical mediators of the urge to scratch are Substance P, or SP, and its receptor, NK1-R. SP is a naturally occurring peptide in the tachykinin neuropeptide family. Tachykinins have a broad range of functions in the nervous and immune systems. SP binding of NK1-R has been shown to be a key mediator of sensory nerve signaling, including the itch-scratch reflex and the vomiting reflex.
Other Name: VPD-737




Primary Outcome Measures :
  1. The proportion of patients who achieve at least a 3-point reduction in NRS itch severity from baseline and after two months of treatment. [ Time Frame: 2 months ]
    Subjects will be asked to complete itch surveys (NRS) and daily before bedtime regarding their daily average itch score and their wound score while bathing/bandage changing.



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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males or females who are at least 13 years of age.
  2. Willing and able to understand and sign informed assent/consent. Adolescents will need a parent or guardian willing and able to give consent.
  3. Clinical diagnosis of epidermolysis bullosa (dystrophic, junctional or simplex).
  4. History of chronic pruritus of at least 6 weeks in duration
  5. On the Screening Visit or Screening phone call, patients must have an NRS pruritus score of at least 5 on average itch score in the past 24 hours
  6. Female subjects must be of non-childbearing potential (ie, post-menopausal for at least 1 year, had a hysterectomy, or had a tubal ligation) or, if of childbearing potential, must have a confirmed negative urine pregnancy test prior to study treatment and be willing to use effective contraception for the duration of the trial. Effective contraception is defined as follows: oral/implant/injectable/ transdermal contraceptives, intrauterine device, condom with spermicide, or diaphragm with spermicide. Abstinence or partner's vasectomy is acceptable if the female agrees to use effective contraception if she decides to discontinue abstinence or to have sexual intercourse with a non-vasectomized partner.
  7. Judged to be in good health based upon the results of a physical examination, medical history, and safety laboratory tests.

Exclusion Criteria:

  1. Have any medical condition or disability that would interfere with the assessment of safety or efficacy in this trial or would compromise the ability of the subject to travel to Stanford or to undergo study procedures or to give informed consent.
  2. Have a history of sensitivity to any components of the study material.
  3. Are females of childbearing potential who are unwilling to use adequate contraception or who are breast feeding.
  4. Have any chronic or acute medical condition that, in the opinion of the investigator, might interfere with the study results or place the subject at undue risk.
  5. Have chronic renal disease, i.e., serum creatinine greater than 2 times the upper limit of normal.
  6. Have chronic liver disease. Subjects with hepatitis B and C who have normal liver function may be enrolled.
  7. Have a current malignancy (such as Hodgkin's lymphoma, B or T cell lymphoma, or myeloma) or blood cell dyscrasia (e.g., polycythemia or myelofibrosis) that would lead to systemic chronic pruritus.
  8. Subjects with untreated hyperthyroidism.
  9. Have pruritus of psychogenic etiology (delusions of parasitosis, obsessive compulsive disorder and major depression) or neuropathic etiology (due to shingles, spinal cord injury or with neurologic deficit).
  10. Have pruritus due to urticaria, drug allergy, or infection (such as pityriasis rosacea or tinea or active human immunodeficiency virus [HIV]). Note: Subjects with HIV who have undetectable viral load, and stable retro-viral therapy may enroll.
  11. Have taken investigational medications within 30 days prior to Screening.
  12. Are unwilling to discontinue specific medications that, in the view of the investigator may have significant interactions with the trial drug, for at least two weeks prior to initiation of study and throughout the study period (this includes miconazole, delavirdine, conivaptan, Clarithromycin, telithromycin, nefazodone, itraconazole, ketoconazole, indinavir, lopinavir, nelfinavir, ritonavir, saquinavir).
  13. Are unable or unwilling to maintain their current anti-itch and opioid-based pain medications at a stable dosage through the course of the two months of active treatment (including but not limited to opioid pain medications, antihistamines, and gabapentin)
  14. Started or changed medications, creams, or emollients including over-the-counter (OTC) preparations or bath oil treatment specifically for relief of pruritus within 30 days prior to Screening.
  15. Within in the past 12 months, have expressed suicidal ideation with some intent to act.
  16. Have any social or medical condition (e.g. alcoholism, drug dependency, psychotic state) that, in the investigator's opinion, might interfere with the subject's ability to comply with the requirements of the protocol.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03836001


Contacts
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Contact: Clinical Trial Coodinator 650-721-7166 ceteng7@stanford.edu
Contact: Clinical Trial Coordinator 6507217149 baileyhi@stanford.edu

Locations
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United States, California
Stanford University Recruiting
Redwood City, California, United States, 94063
Contact: Clinical Trial Coordinator    650-721-7149    baileyhi@stanford.edu   
Sponsors and Collaborators
Stanford University
Epidermolysis Bullosa Research Partnership
Menlo Therapeutics
Investigators
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Principal Investigator: Albert S Chiou, MD/MBA Stanford University

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Responsible Party: Albert Chiou, Assistant Professor of Dermatology., Stanford University
ClinicalTrials.gov Identifier: NCT03836001     History of Changes
Other Study ID Numbers: 49084
First Posted: February 11, 2019    Key Record Dates
Last Update Posted: May 16, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Pruritus
Skin Diseases
Skin Manifestations
Signs and Symptoms
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous
Serlopitant
Neurokinin-1 Receptor Antagonists
Neurokinin A
Substance P
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs