Phase I/IIa Study Evaluating Safety and Efficacy of an Intratympanic Dose of PIPE-505 in Subjects With Hearing Loss

A Phase I/IIa, Randomized, Double-Blind, Placebo-Controlled, Safety, Pharmacokinetic, and Preliminary Efficacy Study of Unilateral Intratympanic PIPE-505 in Subjects With Sensorineural Hearing Loss Associated With Speech-in-Noise Impairment

This is a randomized, double-blind study of PIPE-505, or placebo, in subjects with hearing loss associated with speech-in-noise impairment.

Study Overview

• Status: Completed
• Conditions: Sensorineural Hearing Loss
• Intervention / Treatment: Drug: Placebo; Drug: PIPE-505

Detailed Description

This is a randomized-controlled, double-blind study of PIPE-505 or placebo given as an injection one time in subjects with sensorineural hearing loss associated with speech-in-noise difficulty. Visits to the clinic will occur at baseline, dosing, and days 1, 7, 14, 30, 60 and 90 after treatment. Safety will be assessed by periodic measurement of vital signs, ear examination, electrocardiogram (ECG), blood laboratory analyses and occurrence of adverse events (AE). Efficacy will be assessed by periodic audiometry and other audiological tests.

• Study Type: Interventional
• Enrollment (Actual): 28
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Torrance, California, United States, 90503
      • Breathe Clear Institute
  • Florida
    • Boca Raton, Florida, United States, 33487
      • ENT and Allergy Associates of Florida
  • Indiana
    • New Albany, Indiana, United States, 47150
      • Advanced ENT & Allergy
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas; Dept of Otolaryngology Head & Neck Surgery
  • Kentucky
    • Louisville, Kentucky, United States, 40220
      • Advanced ENT & Allergy
  • North Carolina
    • Charlotte, North Carolina, United States, 28210
      • Charlotte Eye Ear Nose Throat Associates
    • Winston-Salem, North Carolina, United States, 27103
      • Piedmont Ear Nose Throat and Associates
  • Utah
    • Saint George, Utah, United States, 84790
      • Chrysalis Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 71 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Subject's primary language is English.
• Male or female between 18 and 75 years of age, inclusive, at randomization.
• Diagnosis of bilateral sensorineural hearing loss (SNHL).
• Normal tympanogram in the ear intended for injection (as defined in protocol) at Screening.
• Male or female subjects with reproductive potential agree to comply with protocol-approved double barrier contraceptive method during and for 3 months after study drug administration.
• The subject is in general good medical health with no clinically significant or relevant abnormalities, including medical history, physical exam, vital signs, ECG, and laboratory evaluations (hematology, chemistry, and urinalysis) as assessed by the Investigator.

Exclusion Criteria:

• History of chronic otitis externa or media, other chronic middle ear disorders, barotrauma, Meniere's disease, endolymphatic hydrops, perilymph fistula, herpes zoster oticus or other infectious etiology of hearing loss.
• Presence of a genetic, syndromal or developmental auditory disorder or of an autoimmune or serious neurological disorder that could contribute to auditory loss.
• Evidence of current conductive hearing loss, mixed hearing loss or otosclerosis.
• History of auditory loss related to exposure to known high-dose ototoxic drugs; any prior exposure to platinum-based medications.
• Otological disorders that would preclude safe tympanic injection.
• Presence of a cochlear implant.
• Evidence of bothersome tinnitus as determined by the Investigator.
• Intratympanic injection within 6 months of randomization.
• Use of an investigational product or intervention other than a non-interventional registry study (including vaccine studies) within the greater of 30 days or 5 half-lives (if known) prior to Screening or expected during the study.
• History of malignancy under current active treatment or considered at substantial risk for progression or recurrence during the study interval, as determined by the Investigator. Note, central nervous system neoplasms or head and neck cancer are excluded from eligibility regardless of treatment status.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Intratympanic injection
Experimental: PIPE-505	Drug: PIPE-505; Intratympanic injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety: Treatment-Emergent Adverse Events (TEAE)	Number of participants with TEAEs	From baseline to 3 months follow up

Secondary Outcome Measures

Outcome Measure	Time Frame
Pharmacokinetics: Area under the curve (AUC)	From baseline to 3 months follow up
Pharmacokinetics: t1/2 (half life)	From baseline to 3 months follow up

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory: Speech-in-noise assessments	Hearing performance in the setting of background noise	1 month, 2 months, and 3 months after drug administration
Exploratory: Audiogram tests	Hearing sensitivity and thresholds with a quiet background	1 month, 2 months, and 3 months after drug administration
Exploratory: Auditory brainstem potential testing	Electrophysiological assessment of auditory nerve and brainstem responses to auditory signals	1 month, 2 months, and 3 months after drug administration

Collaborators and Investigators

• Sponsor: Contineum Therapeutics
• Investigators: Study Director: Stephen Huhn, MD, Chief Medical Officer, Pipeline Therapeutics, Inc

Study record dates

Study Major Dates

• Study Start (Actual): October 15, 2020
• Primary Completion (Actual): June 17, 2021
• Study Completion (Actual): June 17, 2021

Study Registration Dates

• First Submitted: June 29, 2020
• First Submitted That Met QC Criteria: July 4, 2020
• First Posted (Actual): July 8, 2020

Study Record Updates

• Last Update Posted (Actual): April 1, 2025
• Last Update Submitted That Met QC Criteria: March 13, 2025
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Keywords: Hearing Loss; Deafness; Cochlear synaptopathy; Speech-in-noise hearing difficulty
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Ear Diseases; Hearing Disorders; Hearing Loss; Deafness; Hearing Loss, Sensorineural
• Other Study ID Numbers: PTI-505-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No