KM-001 Cream for the treatment of adult patients with PPPK1 and PC

• Research type

  Research Study

• Full title

  Phase 1b, open label study to evaluate the safety, tolerability, and efficacy of a 1% topical formulation of KM-001 for the treatment of type I Punctate Palmoplantar Keratoderma or Pachyonychia Congenita

• IRAS ID

  1006297

• Contact name

  Anke Mueller

• Contact email

  anke.mueller@bioskincro.com

• Sponsor organisation

  Kamari Pharma Ltd

• ISRCTN Number

  ISRCTN67285394

• Research summary

  The planned trial intends to evaluate whether a newly developed drug is safe, tolerable, and efficacious for the treatment of type I Punctate Palmoplantar Keratoderma (PPPK1) or Pachyonychia Congenita (PC). Both conditions are rare diseases for which standard therapies are available, which can alleviate the symptoms but cannot cure the disease. The sponsor of the trial - Kamari Pharma Ltd. - is developing a new therapy to cure these diseases. The test product is KM-001 cream 1%., which has not yet been approved for treatment. In this trial, it is tested for the duration of 84 days. All participants are treated with the same test product.
  Patients diagnosed for PPPK1 and PC may participate in the trial which is conducted at the clinical research facility of the Royal London Hospital, Whitechapel, London.
  Overall, the trial will take up to 17 weeks with 8 on-site visits and 4 phone calls. Except for 2 visits the visits will take approximately 90 min with several examinations and assessments including but not limited to blood sampling, blood pressure and heart rate measurement, questionnaires for itch and pain assessment. At two visits, blood samples will be taken at different timepoints after application of the test drug to examine drug levels. These stays will take approximately 6.5 hours.
  Certain medications are not permitted prior to the start and during the trial, i.e., participants may need to discontinue any medications they are taking. Concomitant medications during the trial are allowed after consultation with the investigator if they do not affect the trial results or participants’ safety.
  Based on the research results so far, the sponsor and investigator hope that the treatment with KM-001 cream reduces symptoms, e.g., pain, itch, and the disease severity in treated lesions. However, this is to be proven in the trial

• REC name

  Wales REC 1

• REC reference

  22/WA/0272

• Date of REC Opinion

  28 Oct 2022

• REC opinion

  Further Information Favourable Opinion