NCT02507492

Brief Summary

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2015

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2015

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

May 29, 2015

Completed
2 months until next milestone

First Posted

Study publicly available on registry

July 24, 2015

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2018

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2018

Completed
Last Updated

February 22, 2016

Status Verified

February 1, 2016

Enrollment Period

3 years

First QC Date

May 29, 2015

Last Update Submit

February 19, 2016

Conditions

Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation

Keywords

POMC, LEPR, PCSK1

Outcome Measures

Primary Outcomes (1)

  • Effect of RM-493 on Body weight

    Change of body weight after Treatment with RM-493.

    Day 1 through Day 84

Secondary Outcomes (5)

  • Effect of RM-493 on metabolic serum parameters

    Day 1 through Day 84

  • Effect of RM-493 on blood pressure

    Day 1 through Day 84

  • Effect of RM-493 on hunger

    Day 1 through Day 84

  • Effect of RM-493 on body composition/Energy expenditure

    Day 1 through Day 84

  • Effect of RM-493 on weight loss after treatment continuation of 2 years

    Day 1 through Day 730

Study Arms (1)

RM-493 Once Daily

EXPERIMENTAL

Dose once daily in the morning

Drug: RM-493

Interventions

RM-493DRUG
Also known as: setmelanotide
RM-493 Once Daily

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • +1 more criteria

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Charité - Universitätsmedizin Berlin

Berlin, State of Berlin, 13353, Germany

RECRUITING

MeSH Terms

Interventions

setmelanotide

Central Study Contacts

Susanna Wiegand, PD Dr.

CONTACT

+49 30 450 566887susanna.wiegand@charite.de

Peter Kühnen, Dr.

CONTACT

+49 30 450 450 666839peter.kuehnen@charite.de

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

May 29, 2015

First Posted

July 24, 2015

Study Start

January 1, 2015

Primary Completion

January 1, 2018

Study Completion

July 1, 2018

Last Updated

February 22, 2016

Record last verified: 2016-02

Locations

DE(1)