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Trial record 1 of 9 for:    RM-493
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RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2016 by Charite University, Berlin, Germany
Sponsor:
Information provided by (Responsible Party):
PD Dr. Susanna Wiegand, Charite University, Berlin, Germany
ClinicalTrials.gov Identifier:
NCT02507492
First received: May 29, 2015
Last updated: February 19, 2016
Last verified: February 2016
  Purpose
The purpose of this study is to evaluate the effects of a once daily subcutaneous (SC) injection of RM-493, in subjects with POMC (propiomelanocortin) or other related rare genetic mutations, on body weight, metabolic function and blood pressure. Patients who respond during the initial 84 days of treatment can enter into long-term (2-year) extensions. The study drug (RM-493) will be administered in an unblinded fashion.

Condition Intervention Phase
Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation
Drug: RM-493
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients

Further study details as provided by Charite University, Berlin, Germany:

Primary Outcome Measures:
  • Effect of RM-493 on Body weight [ Time Frame: Day 1 through Day 84 ]
    Change of body weight after Treatment with RM-493.


Secondary Outcome Measures:
  • Effect of RM-493 on metabolic serum parameters [ Time Frame: Day 1 through Day 84 ]
    Measurement of the effect of RM-493 on metabolic serum parameters as measured by blood test.

  • Effect of RM-493 on blood pressure [ Time Frame: Day 1 through Day 84 ]
    Measurement of the effect of RM-493 on blood pressure by daily blood pressure measurements.

  • Effect of RM-493 on hunger [ Time Frame: Day 1 through Day 84 ]
    Measurement of the effect of RM-493 on hunger as measured by the Global Hunger Scale

  • Effect of RM-493 on body composition/Energy expenditure [ Time Frame: Day 1 through Day 84 ]
    Measurement of the effect of RM-493 on body composition/Energy expenditure

  • Effect of RM-493 on weight loss after treatment continuation of 2 years [ Time Frame: Day 1 through Day 730 ]
    Measurement of the effect of RM-493 on weight loss after treatment continuation of 2 years


Estimated Enrollment: 10
Study Start Date: January 2015
Estimated Study Completion Date: July 2018
Estimated Primary Completion Date: January 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RM-493 Once Daily
Dose once daily in the morning
Drug: RM-493
Other Name: setmelanotide

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent
  • Homozygous or compound heterozygous (different gene mutation on both alleles) POMC, LEPR or PCSK1 gene mutation
  • Obesity (BMI > 30 kg/m2; + 2 BMI SDS)
  • No other therapeutic option, which might cure the patient (e.g. bariatric surgery (see chapter 8))
  • Negative Pregnancy test
  • Highly effective contraception in women (defined as pearl index < 1), if necessary also for partners of test persons)
  • No participation in other clinical trials according to AMG (Arzneimittelgesetz) (2 months before and after) at the time of this trial
  • Normal or minimally elevated blood pressure (measured in 24RR monitoring or similar methods) according the guidelines of the ESH (European Society of Hypertension) and Deutsche Hochdruckliga: systolic > 159 mmHg/diastolic 99 mmHg
  • sufficient kidney and liver function (Creatinine, ALT, AST)

    • normal values Alanine-Aminotransferase (ALT) (female): < 31 U/l
    • normal values Alanine-Aminotransferase (ALT) (male): < 41 U/l
    • normal values Aspartate-Aminotransferase (AST) (female > 17 years): < 35 U/l; (female < 17 years): 16- 46 U/l
    • normal values Aspartate-Aminotransferase (AST) (male > 17 years): < 50 U/l; (male < 17 years): 16-46 U/l
    • normal values bilirubins (male and female) up to 1,2 mg/dl
    • normal values Creatinine (female > 15 years): 0,51-0,95 mg/dl) ; (female < 15 years): 0,46-0,77 mg/dl
    • normal values Creatinine (male > 15 years): 0,67 - 1,17 mg/dl) ; (male < 15 years): 0,46-0,77 mg/dl

Exclusion Criteria:

  • Pregnancy or Breastfeeding
  • All contraindications against study medication (including auxiliary substances)
  • Interactions with study medication
  • Participation of the patient in a clinical study within the last 2 months
  • Intolerance against albumin
  • Concomitant diseases, impaired organ functions, except for known, concurrent GI disorders or other clinical findings expected in PCSK1 or LEPR gene disorders
  • Renal insufficiency (Creatinine > 0,95 mg/dl (female), > 1,17 mg/dl (male))
  • Impaired liver function (Bilirubins > 1.2 mg/dl)
  • Neurological / psychiatric diseases
  • HIV Infection
  • Active Hepatitis B or C
  • Melanoma or Melanoma occurrence in the family history
  • Non-compliance
  • Subjects who are legally detained in an official institution
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02507492

Contacts
Contact: Susanna Wiegand, PD Dr. +49 30 450 566887 susanna.wiegand@charite.de
Contact: Peter Kühnen, Dr. +49 30 450 450 666839 peter.kuehnen@charite.de

Locations
Germany
Charité - Universitätsmedizin Berlin Recruiting
Berlin, Germany, 13353
Contact: Susanna Wiegand, PD Dr.    +49 30 450 566887    susanna.wiegand@charite.de   
Contact: Peter Kühnen, Dr.    +49 30 450 450 666839    peter.kuehnen@charite.de   
Sub-Investigator: Heiko Krude, Prof. Dr.         
Sub-Investigator: Peter Kühnen, Dr.         
Sponsors and Collaborators
Charite University, Berlin, Germany
  More Information

Responsible Party: PD Dr. Susanna Wiegand, Principal Investigator, Charite University, Berlin, Germany
ClinicalTrials.gov Identifier: NCT02507492     History of Changes
Other Study ID Numbers: RM-493-011
2014-002392-28 ( EudraCT Number )
Study First Received: May 29, 2015
Last Updated: February 19, 2016

Keywords provided by Charite University, Berlin, Germany:
POMC, LEPR, PCSK1

Additional relevant MeSH terms:
Adrenocorticotropic Hormone
Melanocyte-Stimulating Hormones
beta-Endorphin
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on April 28, 2017