Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene MutationPhase 2NCT02507492
RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients
A Phase 2 study testing RM-493 in people with Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation, run by Charite University, Berlin, Germany at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation
- What is being tested?
- RM-493
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteGermany
- Study dates
- Jan 2015to Jul 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
Charité - Universitätsmedizin Berlin
Berlin, State of Berlin, 13353
Susanna Wiegand, PD Dr., +49 30 450 566887, susanna.wiegand@charite.de
Peter Kühnen, Dr., +49 30 450 450 666839, peter.kuehnen@charite.de
About this study
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.
- Being tested
- RM-493
- Keywords
- POMC, LEPR, PCSK1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RM-493Drug
Also called setmelanotide
| Study group | Role | What they receive |
|---|---|---|
| RM-493 Once Daily | Experimental | Dose once daily in the morning Receives: Drug: RM-493 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of RM-493 on Body weight
Change of body weight after Treatment with RM-493.
Measured: Day 1 through Day 84
Secondary outcomes (5)
Effect of RM-493 on metabolic serum parameters
Measurement of the effect of RM-493 on metabolic serum parameters as measured by blood test.
Measured: Day 1 through Day 84
Effect of RM-493 on blood pressure
Measurement of the effect of RM-493 on blood pressure by daily blood pressure measurements.
Measured: Day 1 through Day 84
Effect of RM-493 on hunger
Measurement of the effect of RM-493 on hunger as measured by the Global Hunger Scale
Measured: Day 1 through Day 84
Effect of RM-493 on body composition/Energy expenditure
Measurement of the effect of RM-493 on body composition/Energy expenditure
Measured: Day 1 through Day 84
Effect of RM-493 on weight loss after treatment continuation of 2 years
Measurement of the effect of RM-493 on weight loss after treatment continuation of 2 years
Measured: Day 1 through Day 730
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2015 (estimated)
- Study startJan 2015
- Primary completionJan 2018 (estimated)
- Study completionJul 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene MutationStudies of the same condition, open and under way.
- More trials run by Charite University, Berlin, GermanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Charite University, Berlin, Germany
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, PD Dr. Susanna Wiegand, Charite University, Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02507492
- Sponsor's protocol ID
- RM-493-011
- Other IDs
- 2014-002392-28, 2014
- Status last verified
- Feb 2016
- First submitted
- 29 May 2015
- Last updated
- 22 Feb 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients
- ClinicalTrials.gov ID
- NCT02507492
- Acronym / sponsor ID
- RM-493-011
- Keywords
- POMC, LEPR, PCSK1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Charite University, Berlin, GermanyUniversity, hospital or other
- Study start
- Jan 2015
- Primary completion
- Jan 2018 (estimated)
- Study completion
- Jul 2018 (estimated)
- First posted
- 24 Jul 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RM-493 Once Daily | Experimental | Drug: RM-493Dose once daily in the morning |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RM-493Also: setmelanotide | Drug | RM-493 Once Daily |
Primary outcome
Effect of RM-493 on Body weight
Time frame: Day 1 through Day 84
Change of body weight after Treatment with RM-493.
5 secondary outcomes
Effect of RM-493 on metabolic serum parameters
Time frame: Day 1 through Day 84
Effect of RM-493 on blood pressure
Time frame: Day 1 through Day 84
Effect of RM-493 on hunger
Time frame: Day 1 through Day 84
Effect of RM-493 on body composition/Energy expenditure
Time frame: Day 1 through Day 84
Effect of RM-493 on weight loss after treatment continuation of 2 years
Time frame: Day 1 through Day 730
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2016.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RM-493 in people with Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation, run by Charite University, Berlin, Germany at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Susanna Wiegand, PD Dr. (+49 30 450 566887, susanna.wiegand@charite.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of RM-493 on Body weight (time frame: Day 1 through Day 84). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Feb 2016.