A Study Evaluating Mibavademab Treatment of Obesity due to Leptin (LEP) Gene Mutations in Children, Adolescents and Adults

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Regeneron Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Decision date (initial)

2025-11-06

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Regeneron Pharmaceuticals, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the effect of mibavademab on body weight.

Secondary objectives 6

1. To characterize time course of body weight changes and effect on central adiposity in response to mibavademab.
2. To evaluate the effect of mibavademab on body composition.
3. To evaluate the impact of mibavademab on hunger and related symptoms of obesity.
4. To evaluate the safety and tolerability of mibavademab.
5. To evaluate the PK of mibavademab in participants with MOB.
6. To assess the immunogenicity of mibavademab.

Conditions and MedDRA coding

Monogenic obesity

Regulatory references

Scientific advice from competent authorities

Medicines And Healthcare Products Regulatory Agency

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Has documented medical history of biallelic loss of function variants of the LEP gene prior to the screening visit.
2. Has class ≥2 obesity at screening and at baseline, as defined in the protocol.
3. NOTE: Other Protocol-defined inclusion criteria apply.

Exclusion criteria 9

1. Unwilling or unable to provide, or have the treating physician provide, documented historical weight for at least 6 months prior to screening.
2. History of bariatric surgery within approximately the past 12 months of study screening.
3. History of weight loss of ≥5% of body weight in approximately the past 3 months of study screening.
4. History of genetic causes of obesity other than/in addition to biallelic loss of function variants of the LEP gene.
5. History of schizophrenia, bipolar disorder, or other mental illness that, in the opinion of the investigator, might pose a safety risk to participation in a clinical study of an investigational drug.
6. Any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS) during screening or baseline, or any lifetime history of a suicide attempt, or any lifetime history of suicidal behavior, including ideation.
7. Treatment with medications for weight loss or medications with known side effects of weight loss within the past approximately 3 months of study screening.
8. Participants currently being treated with metreleptin, or a history of prior treatment with metreleptin as defined in the protocol.
9. NOTE: Other Protocol-defined exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percent change in Body Mass Index (BMI) from baseline to week 24.

Secondary endpoints 17

1. Percent change in BMI from baseline to week 52.
2. Absolute change in BMI from baseline, through week 52.
3. Percent change in body weight from baseline, through week 52.
4. Absolute change in body weight from baseline, through week 52.
5. Change in waist circumference from baseline, through week 52 in participants aged ≥18 years.
6. Change in absolute total fat mass by whole body DXA from baseline, through week 52.
7. Change in percent total fat mass by whole body DXA from baseline, through week 52.
8. Change in absolute regional fat mass by whole body DXA from baseline, through week 52.
9. Change in percent regional fat mass by whole body DXA from baseline, through week 52.
10. Change in scores in the symptoms of hyperphagia questionnaire from baseline, through week 52.
11. Change in scores in the impacts of hyperphagia questionnaire from baseline, through week 52.
12. Change in scores in the hunger questionnaire from baseline, through week 52.
13. Number of treatment-emergent adverse events (TEAEs) through week 65.
14. Severity of TEAEs through week 65.
15. Concentrations of total mibavademab in serum through week 65.
16. Incidence of anti-drug antibody (ADA) to mibavademab through week 65.
17. Titer of ADA to mibavademab through week 65.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Regeneron Pharmaceuticals Inc.

Sponsor organisation

Regeneron Pharmaceuticals Inc.

Address

777 Old Saw Mill River Road

City

Tarrytown

Postcode

10591-6717

Country

United States

Scientific contact point

Organisation

Regeneron Pharmaceuticals Inc.

Contact name

Medical Affairs

Public contact point

Organisation

Regeneron Pharmaceuticals Inc.

Contact name

Medical Affairs

Third parties 7

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 23 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications