Status:
COMPLETED
The TEMPO (Tracing the Effect of the MC4 Pathway in Obesity) Registry
Lead Sponsor:
Rhythm Pharmaceuticals, Inc.
Conditions:
Genetic Forms of Extreme Obesity
Eligibility:
All Genders
2+ years
Brief Summary
A voluntary prospective study that will allow registration and follow-up of individuals with MC4R pathway genetic obesity.
Detailed Description
Potential patients will be referred to coordinating centers by their healthcare provider (treating physician, diagnosing physician or primary care physician) and if eligible will be invited to enroll ...
Eligibility Criteria
Inclusion
- Extreme obesity patients aged 2 years and older.
- Extreme obesity defined as:
- BMI \> 40 kg/m2 in patients 18 years of age or older.
- BMI value that is \>1.4 times the corresponding age/gender 95th percentile value in patients who are 2 through 17 years of age.
- At least one of the following genotypes:
- Bi-allelic (homozygous or compound heterozygous) POMC, PCSK1, LEPR variants leading to the physician-confirmed diagnosis of either POMC or LEPR deficiency obesity.
- The presence of high-confidence, high-impact genetic variations (homozygote, compound heterozygote, heterozygote or composite heterozygote \[i.e., heterozygous variants in more than a single gene\]) in these same 3 genes (POMC, PCSK1 and LEPR) associated with the clinical presentation of extreme obesity
- The presence of other high-confidence, high-impact genetic variations (homozygote, compound heterozygote, heterozygote or composite heterozygote in the MC4R gene or other upstream MC4R-pathway genes and selected variants downstream in the MC4R-pathway (Table 1) that are carried by extreme obesity patients who do not demonstrate specific syndromic obesity clinical presentations. Selected Bardet-Biedl syndrome (BBS1 through BBS21) genetic variants or Alström syndrome (ALMS) genetic variants possibly contributing to non-syndromic forms of clinical extreme obesity will be eligible for enrollment.
- Study participant and/or parent or caregiver can understand and comply with the requirements of the study, and able to understand and sign the written informed consent (IC)/assent, after being informed about the study.
Exclusion
- Patients with syndromic forms of obesity such as Bardet-Biedl syndrome or Alström syndrome. These patients will be referred to existing registries for these specific syndromic obesity (e.g., Clinical Registry in Bardet-Biedl syndrome \[CRIBBS\] for BBS patients).
- Individual is, in the opinion of the study investigator, not suitable to participate in the study.
Key Trial Info
Start Date :
April 15 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 1 2020
Estimated Enrollment :
303 Patients enrolled
Trial Details
Trial ID
NCT03479437
Start Date
April 15 2018
End Date
December 1 2020
Last Update
May 4 2021
Active Locations (2)
Enter a location and click search to find clinical trials sorted by distance.
1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
2
University of Alberta
Edmonton, Canada, T6G 2B7