Metreleptin for Lipodystrophy
Recruiting in Palo Alto (17 mi)
+4 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Amryt Pharma
Must not be taking: Immunomodulatory drugs
Disqualifiers: HIV, Immunocompromised, eGFR <30, Malignancy, others
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 2 Jurisdictions
Trial Summary
What is the purpose of this trial?This Phase 3 study is an Open Label Extension of the APG-20 Study To Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects with Partial Lipodystrophy
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does mention that subjects on oral contraceptives will not be required to discontinue them. It is best to discuss your specific medications with the study team.
How does the drug Metreleptin differ from other treatments for lipodystrophy?
Metreleptin is unique because it is a synthetic form of the hormone leptin, which helps regulate energy balance and fat storage, making it particularly effective for treating lipodystrophy, a condition characterized by abnormal fat distribution. Unlike other treatments, Metreleptin directly addresses the hormone deficiency that contributes to the symptoms of lipodystrophy.
12345Eligibility Criteria
This trial is for individuals with Partial Lipodystrophy, specifically those who have already participated in the APG-20 Study. It's designed to assess long-term safety and effectiveness of Metreleptin when taken daily.Inclusion Criteria
I am postmenopausal, surgically sterile, or I use effective birth control.
Subjects who are willing to follow the dietary restrictions recommended by the Investigator
I am not pregnant.
+5 more
Exclusion Criteria
Severe hypersensitivity reactions to the study treatment of the Parent study APG-20
I have tested positive for HIV.
I am immunocompromised or taking drugs that affect my immune system.
+5 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Open-label Extension
Participants receive daily subcutaneous metreleptin treatment to evaluate long-term safety and efficacy
24 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The study tests the ongoing effects of a drug called Metreleptin given by daily injection under the skin. As an open-label extension, all participants know they are receiving Metreleptin and there is no comparison group.
1Treatment groups
Experimental Treatment
Group I: MetreleptinExperimental Treatment1 Intervention
Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake
Metreleptin is already approved in United States, Canada for the following indications:
🇺🇸 Approved in United States as Myalept for:
- Complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy
🇨🇦 Approved in Canada as Myalept for:
- Complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy
- Patients with partial lipodystrophy
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
UT Southwestern Medical CenterDallas, TX
University of MichiganAnn Arbour, MI
Massachusetts General HospitalBoston, MA
University of MichiganAnn Arbor, MI
More Trial Locations
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Who Is Running the Clinical Trial?
Amryt PharmaLead Sponsor
References
Development of Myostatin Inhibitory d-Peptides to Enhance the Potency, Increasing Skeletal Muscle Mass in Mice. [2023]Myostatin is a key negative regulator of skeletal muscle growth, and myostatin inhibitors are attractive tools for the treatment of muscular atrophy. Previously, we reported a series of 14-29-mer peptide myostatin inhibitors, including a potent derivative, MIPE-1686, a 16-mer N-terminal-free l-peptide with three unnatural amino acids and a propensity to form β-sheets. However, the in vivo biological stability of MIPE-1686 is a concern for its development as a drug. In the present study, to develop a more stable myostatin inhibitory d-peptide (MID), we synthesized various retro-inverso versions of a 16-mer peptide. Among these, an arginine-containing derivative, MID-35, shows a potent and equivalent in vitro myostatin inhibitory activity equivalent to that of MIPE-1686 and considerable stability against biodegradation. The in vivo potency of MID-35 to increase the tibialis anterior muscle mass in mice is significantly enhanced over that of MIPE-1686, and MID-35 can serve as a new entity for the prolonged inactivation of myostatin in skeletal muscle.
Synthesis and biological activity of ovine beta-lipotropin-(41--91)-henkaipentekontapeptide. [2019]The 51-residue peptide ovine beta-lipotropin-(41--91) has been synthesized by the solid-phase method in about 5% overall yield. The synthetic product was characterized by partition chromatography on agarose gel, thin-layer chromatography in two solvent systems, paper electrophoresis at two pH values, polyacrylamide gel electrophoresis, amino acid analyses of acid and enzymic hydrolysates, and bioassay for lipolytic and melanotropic activities. The synthetic peptide is about 5.4 times as active on a weight basis as ovine beta-lipotropin in the lipolytic assay. In the melanotropic assay, it was about 2.4 times more active than the beta-lipotropin but only 5% as active as bovine beta-melanotropin. It had negligible opiate activity in the guinea pig ileum assay.
Synthesis of a pentekontapeptide with high lipolytic activity corresponding to the carboxyl-terminal fifty amino acids of ovine beta-lipotropin. [2019]The synthesis of a peptide, composed of fifty amino-acid residues, corresponding to positions 42-91 in ovine beta-lipotropin has been accomplished by the solidphase method. The preformed symmetrical anhydride and active ester coupling methods were used exclusively. The synthetic product was purified by gel filtration, carboxymethylcellulose chromatography, and partition chromatography on Sephadex G-50. Its lipolytic activity in isolated rabbit fat cells was about six times that of beta-lipotropin on a weight basis.
beta-Lipotropin: primary structure of the hormone from the ostrich pituitary gland. [2019]The amino acid sequence of beta-lipotropin from the ostrich pituitary has been determined. It consists of 79 amino acids. The amino acid sequence has been determined as follows: H-(1)AlA-Leu-Pro-Pro-Ala-Ala-Met-Leu-Pro-(10)Ala-Ala-Ala-Glu-Glu-Glu-Glu-Gly-Gl u-Glu-(20)Glu-Glu-Glu-Gly-Glu-Ala-Glu-Lys-Glu-Asp-(30)Gly-Gly-Ser-Tyr-Arg-Met-A rg-His-Phe-Arg-(40)Trp-Gln-Ala-Pro-Leu-Lys-Asp-Lys-Arg-Tyr-(50)Gly-Gly-Phe-Met- Ser-Ser-Glu-Arg-Gly-Arg-(60)Ala-Pro-Leu-Val-Thr-Leu-Phe-Lys-Asn-Ala-(70)Ile-Val -Lys-Ser-Ala-Tyr-Lys-Lys-Gly-(79)Gln-OH. When compared with the primary structures of other known beta-lipotropins, the sequence at the NH2-terminal, beta-melanotropin and beta-endorphin portions of the molecule exhibit considerable variability.
Isolation of a new lipolytic-melanotropic peptide from human pituitary glands. [2016]A new peptide having both lipolytic and melanotropic properties has been isolated from human pituitary glands. It has a molecular weight around 11,000, an amino acid composition different from the known lipolytic-melanotropic hormones, and an isoelectric point of 8.5. Although it is not entirely pure, there is no doubt that it differs from other known lipolytic-melanotropic substances in its total lack of methionine and tryptophan and its unusually high content of lysine and histidine. It has a melanocyte-stimulating hormone activity of 19 units/mug. As a lipolytic agent it is active with rabbit adipocytes, only slightly active with human cells and inactive with adipocytes from the rat.