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

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Brief Title

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

Official Title

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

Brief Summary

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

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. comparative weekly change in daily worst-itch NRS,

        2. comparative weekly change in average daily NRS itch severity,

        3. 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,

        4. proportion of patients achieving 2 point and 4 point reductions in average daily itch
           severity following two months of treatment,

        5. change in participant-reported outcomes of global assessment of change in itch and
           overall improvement as measured by static participant assessment of itch, participant
           global assessment of change in itch severity, and caregiver global assessment of change
           in itch severity, and

        6. assessment on the safety of Serlopitant in adolescents (≥13 y.o.) and adults with
           pruritus associated with epidermolysis bullosa (EB).
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

The proportion of patients who achieve at least a 3-point reduction in NRS itch severity from baseline and after two months of treatment.


Condition

Epidermolysis Bullosa

Intervention

Serlopitant Tablet

Study Arms / Comparison Groups

 Placebo Oral Tablet
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

29

Start Date

April 18, 2019

Completion Date

May 15, 2022

Primary Completion Date

December 6, 2021

Eligibility 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. Have a history of thyroid cancer, thyroid nodules, inadequately treated thyroid
             disease, or abnormal TSH or free T4 at screening.

          9. Have a history of abnormalities in adrenal or pituitary function (pituitary adenoma,
             adrenal insufficiency, or adrenal nodule).

         10. Screening cortisol level < 3 mcg/dL

         11. Unevaluated abnormalities in cortisol, ACTH, or prolactin.

         12. 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).

         13. 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.

         14. Have taken investigational medications within 30 days prior to Screening.

         15. 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).

         16. 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)

         17. 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.

         18. Within in the past 12 months, have expressed suicidal ideation with some intent to
             act.

         19. 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.
      

Gender

All

Ages

13 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Albert S Chiou, MD/MBA, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03836001

Organization ID

49084


Responsible Party

Principal Investigator

Study Sponsor

Stanford University

Collaborators

 Epidermolysis Bullosa Research Partnership

Study Sponsor

Albert S Chiou, MD/MBA, Principal Investigator, Stanford University


Verification Date

April 2022