METRE-PL: Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Patients With PL

• Sponsor: Amryt Pharma (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05164341
• Collaborator: (none)
• Enrollment: 80
• Location: 1
• Arms: 2
• Anticipated Duration (Months): 49.5
• Patients Per Site Per Month: 1.6

Study Details

Study Description

Brief Summary

This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.

Condition or Disease	Intervention/Treatment	Phase
Partial Lipodystrophy	Drug: metreleptin; Drug: Placebo	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 80 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy
• Actual Study Start Date: Dec 17, 2021
• Anticipated Primary Completion Date: Jun 30, 2025
• Anticipated Study Completion Date: Jan 31, 2026

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: metreleptin Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake	Drug: metreleptin; Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency
Placebo Comparator: placebo Placebo for daily injection is a sterile, white, solid lyophilised cake	Drug: Placebo; Placebo

Outcome Measures

Primary Outcome Measures

1. Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL) [6 months]

   To evaluate the efficacy (HbAa1c) of daily SC metreleptin treatment

2. Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL) [6 months]

   To evaluate the efficacy (TGs) of daily SC metreleptin treatment

Secondary Outcome Measures

1. Safety analysis of AEs, AESIs, SAEs by treatment arm [12 months]

   To evaluate the safety of daily SC metreleptin treatment in subjects with PL

2. Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL) [12 months]

   To assess the effect of metreleptin on HbA1c

3. Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL) [12 months]

   To assess the effect of metreleptin on TGs

4. Change from Baseline to each assessment time point in quality of life (QoL) in all subjects [12 months]

   To assess the effect of metreleptin on quality of life (QoL) in all subjects

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Diagnosis of Partial Lipodystrophy

Exclusion Criteria:

• Previous treatment with metreleptin

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Amryt Research Site	Columbus	Ohio	United States	43201

Sponsors and Collaborators

• Amryt Pharma

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Amryt Pharma
• ClinicalTrials.gov Identifier: NCT05164341
• Other Study ID Numbers: APG-20
• First Posted: Dec 20, 2021
• Last Update Posted: Mar 3, 2022
• Last Verified: Feb 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Amryt Pharma: metreleptin
• Additional relevant MeSH terms: Lipodystrophy

Study Results

No Results Posted as of Mar 3, 2022