HypertriglyceridemiaPhase 2NCT03262610
Setmelanotide in a Single Patient With Partial Lipodystrophy
A Phase 2 study testing Setmelanotide in people with Hypertriglyceridemia and Obesity, run by Rhythm Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
The objective of this single patient study is to assess the safety and efficacy of setmelanotide as a treatment of severe metabolic abnormalities resulting from LD, especially refractory hypertriglyceridemia leading to recurrent bouts of pancreatitis. Read more about this study
- Who is it for?
- Ages 15 to 15 years, women only
- Conditions the study is for
- Hypertriglyceridemia, Obesity
- What is being tested?
- Setmelanotide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Aug 2017to 31 Jan 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 15 to 15 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Signed written informed consent
- Ability to comply with visits and procedures required by program.
- Has physician-confirmed partial lipodystrophy and the following characteristics:
- Has atypical lipodystrophy.
- Has presence of neutralizing antibody to metreleptin
- Patient has life threatening hypertriglyceridemia and has had >8 episodes of pancreatitis, requiring weekly plasmapheresis
- Has Type 1 diabetes mellitus with HbA1c > 10%.
- Female, under the age of 18 years
Who cannot join
- Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study compliance. Neurocognitive disorders affecting ability to consent will not be disqualifying as long as an appropriate guardian able to give consent has been appointed.
- History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocular-cutaneous albinism.
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist.
- Significant hypersensitivity to study drug.
- Inability to comply with QD injection regimen.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Expanded-access for the Use of Setmelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases
The objective of this single patient study is to assess the safety and efficacy of setmelanotide as a treatment of severe metabolic abnormalities resulting from LD, especially refractory hypertriglyceridemia leading to recurrent bouts of pancreatitis.
Read the detailed description
This is a single-patient study to assess the safety and efficacy of setmelanotide in a unique patient with partial LD and severe metabolic abnormalities, most notably refractory hypertriglyceridemia leading to recurrent pancreatitis despite ongoing plasmapheresis treatment.
- Conditions
- Hypertriglyceridemia, Obesity
- Being tested
- Setmelanotide
- Medical subject headings
- Hypertriglyceridemia, Obesity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SetmelanotideDrug
Also called RM-493
daily subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Setmelanotide daily subcutaneous injection | Experimental | Up to 18 weeks setmelanotide treatment. Receives: Drug: Setmelanotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Fasting Triglycerides (TG) levels
The mean change from baseline in fasting triglycerides after setmelanotide treatment.
Measured: 12 to 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2017
- Study start15 Aug 2017
- Primary completion31 Jan 2018
- Study completion31 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypertriglyceridemiaStudies of the same condition, open and under way.
- 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Linda Shapiro, M.D., Ph.D., Study chair, Chief Medical Officer, Rhythm Pharmaceuticals, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03262610
- Sponsor's protocol ID
- RM-493-019
- Status last verified
- Dec 2021
- First submitted
- 22 Aug 2017
- Last updated
- 4 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded-access for the Use of Setmelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases
- ClinicalTrials.gov ID
- NCT03262610
- Acronym / sponsor ID
- RM-493-019
- Conditions
- Hypertriglyceridemia, Obesity
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Aug 2017 (actual)
- Primary completion
- 31 Jan 2018 (actual)
- Study completion
- 31 Jan 2018 (actual)
- First posted
- 25 Aug 2017 (actual)
- 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 objective of this single patient study is to assess the safety and efficacy of setmelanotide as a treatment of severe metabolic abnormalities resulting from LD, especially refractory hypertriglyceridemia leading to recurrent bouts of pancreatitis.
Detailed description
This is a single-patient study to assess the safety and efficacy of setmelanotide in a unique patient with partial LD and severe metabolic abnormalities, most notably refractory hypertriglyceridemia leading to recurrent pancreatitis despite ongoing plasmapheresis treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Setmelanotide daily subcutaneous injection | Experimental | Drug: SetmelanotideUp to 18 weeks setmelanotide treatment. |
Who may qualify
- Ages
- 15 to 15 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Signed written informed consent
- Ability to comply with visits and procedures required by program.
- Has physician-confirmed partial lipodystrophy and the following characteristics:
- Female, under the age of 18 years
Key exclusion criteria (5 listed)
- Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study compliance. Neurocognitive disorders affecting ability to consent will not be disqualifying as long as an appropriate guardian able to give consent has been appointed.
- History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocular-cutaneous albinism.
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist.
- Significant hypersensitivity to study drug.
- Inability to comply with QD injection regimen.
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 |
|---|---|---|
| SetmelanotideAlso: RM-493daily subcutaneous injection | Drug | Setmelanotide daily subcutaneous injection |
Primary outcome
Fasting Triglycerides (TG) levels
Time frame: 12 to 20 weeks
The mean change from baseline in fasting triglycerides after setmelanotide treatment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2022.
Related trials for Hypertriglyceridemia
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Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy
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Plasma Lipids-Dependent Vitamin E Metabolism During Dynamic Hyperlipidemia
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Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia
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A Phase II Study of FXR0906 in Patients With Severe Hypertriglyceridemia or Hypertriglyceridemia With ASCVD.
sHTG, HTG With ASCVD (Diagnosed or at Increased Risk)
Active, not recruitingPhase 3
A Study of SHR-1918 In Participants With Hypertriglyceridemia
Hypertriglyceridemia
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.
Linda Shapiro, M.D., Ph.D. study chair
Chief Medical Officer, Rhythm Pharmaceuticals, Inc
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 Setmelanotide in people with Hypertriglyceridemia and Obesity, run by Rhythm Pharmaceuticals, Inc. at 1 site in United States.
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 8 inclusion and 5 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: finished, as of the sponsor's last update on 4 Jan 2022. 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 United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Fasting Triglycerides (TG) levels (time frame: 12 to 20 weeks). 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 4 Jan 2022.