RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients

NCT ID: NCT02507492

Last Updated: 2016-02-22

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-01-31
• Study Completion Date: 2018-07-31

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.

Conditions

Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

RM-493 Once Daily

Dose once daily in the morning

Group Type: EXPERIMENTAL

RM-493

Intervention Type: DRUG

Interventions

RM-493

Intervention Type: DRUG

Other Intervention Names

setmelanotide

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Charite University, Berlin, Germany

OTHER

Sponsor Role: lead

Responsible Party

PD Dr. Susanna Wiegand

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Charité - Universitätsmedizin Berlin

Berlin, State of Berlin, Germany

Site Status: RECRUITING

Countries

Germany

Central Contacts

Susanna Wiegand, PD Dr.

Role: CONTACT

susanna.wiegand@charite.de

+49 30 450 566887

Peter Kühnen, Dr.

Role: CONTACT

peter.kuehnen@charite.de

+49 30 450 450 666839

Facility Contacts

Susanna Wiegand, PD Dr.

Role: primary

susanna.wiegand@charite.de

+49 30 450 566887

Peter Kühnen, Dr.

Role: backup

peter.kuehnen@charite.de

+49 30 450 450 666839

Other Identifiers

2014-002392-28

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

RM-493-011

Identifier Type: -

Identifier Source: org_study_id