A Phase 2 interventional study of Obeticholic Acid and Placebo in Familial Partial Lipodystrophy, sponsored by Abhimanyu Garg. Completed at 2 sites in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2024-08-27.
Sponsored by Abhimanyu Garg · Phase 2, Interventional, and Treatment
Lipodystrophies are rare disorders characterized by selective loss of adipose tissue and predisposition to insulin resistance and its metabolic complications. Hepatic steatosis is a common complication in patients with partial and generalized lipodystrophies.Despite aggressive management of diabetes and hyperlipidemia, hepatic steatosis and its complications present a therapeutic challenge in many patients. Due to this large disease burden, it is important to assess the efficacy and safety of novel therapies for hepatic steatosis in patients with lipodystrophies.There are, however, no systematic studies evaluating various therapeutic interventions for reducing hepatic steatosis in patients with lipodystrophies. A variety of drugs have been investigated in nonlipodystrophic patients with non-alcoholic hepatic steatosis and steatohepatitis (NASH) or non-alcoholic fatty liver disease (NAFLD). Recent data support the activation of the farnesoid X receptor (FXR, NR1H4), a nuclear hormone receptor regulated by bile acids, for treatment of NASH and NAFLD. FXR activates transcription of several genes particularly the atypical nuclear receptor small heterodimer partner (SHP, NR0B2) and thus can influence triglyceride metabolism within hepatocytes.Both cholic acid (CA) and chenodeoxycholic acid (CDCA) are ligands for FXR, however, UDCA which is the 7 hydroxy β-epimer of CDCA, does not activate FXR. Obeticholic acid (OCA) is a first-in-class selective FXR agonist which has approximately 100 fold greater FXR-agonistic activity in the nanomolar range, as compared to CDCA .It therefore appears that FXR modulation offers interesting therapeutic possibilities in treating hepatic steatosis. This study is primarily designed to study efficacy of OCA, a strong FXR ligand, in reducing hepatic triglyceride levels in patients with hepatic steatosis and Familial Partial Lipodystrophy (FPLD). If proven to be effective, it may reduce morbidity and mortality as a result of sequelae of hepatic steatosis in patients with lipodystrophies.
This study will be a randomized, placebo-controlled cross-over trial. Patients who are considered eligible for the study will undergo screening evaluation to determine their eligibility for the trial. For those who are found to be eligible, during the baseline period, they will continue their usual diet and other lifestyle measures without changing any medications for 1 month in order to establish a baseline state. Three blood samples will be obtained during this period at the Clinical and Translational Research Center. Following the baseline period, the patients will receive obeticholic acid (OCA) or an identical placebo in the dose of 25 mg/day for a period of 4 months and then will receive the other treatment (OCA or placebo) for 4 months. There will be a wash-out period of 4 months in-between the two study periods.
Patients will be educated to maintain their usual physical activities and diet during the study. The subjects will be admitted to the Clinical and Translational Research Center for the baseline evaluations (at the beginning of the two study periods), and at the end of four months during each study period.
20 studies on the registry are indexed under Lipodystrophy, Familial Partial; 5 are open to participants now.
This study's enrollment of 10 is below the median of 16 across 14 interventional studies indexed under Lipodystrophy, Familial Partial.
Browse Lipodystrophy, Familial Partial studies →This is the only study on the registry with Abhimanyu Garg as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patient will receive obeticholic acid (OCA) in the dose of 25 mg/day for a period of 4 months.
Drug: Obeticholic Acid
Patient will recieve placebo in the dose of 25 mg/day for a period of 4 months.
Drug: Placebo
Capsules of obeticholic acid (OCA) or an identical placebo in the dose of 25 mg/day for a period of 4 months .
Also known as: NEW DRUG APPLICATION
Identical to Obeticholic Acid - placebo drug
Also known as: Identical to Obeticholic Acid placebo drug
Change in the Liver Triglycerides (TG).
The primary end-point variable was the change in the liver TG content assessed using proton-density fat fraction mapping by Magnetic Resonance Imaging (MRI).
Time frame: Baseline, 4 months
Change in Serum Triglyceride Levels
Change in the serum levels of Triglycerides from baseline to month 4 is being assessed.
Time frame: Baseline, Month 4
Change in Serum Levels of Alanine Aminotransferase
Change in serum levels of Alanine Aminotransferase from baseline to month 4 is assessed
Time frame: Baseline, Month 4
Change in Serum Levels of Aspartate Aminotransferases
Change in serum levels of Aspartate Aminotransferases from baseline to month 4 is assessed
Time frame: Baseline, Month4
Change in Serum Levels of Gamma-Glutamyl Transpeptidase
Change in serum levels of Gamma-Glutamyl Transpeptidase from baseline to month 4 is assessed
Time frame: Baseline, Month 4
Changes in Serum Insulin Levels
Changes in serum Insulin levels is assessed
Time frame: Baseline, Month 4
Change in Hepatic Insulin Sensitivity
Change in hepatic insulin sensitivity is assessed by suppression of hepatic glucose output during the low-dose and high-dose insulin infusions during the euglycemic clamp study.
Time frame: Baseline, Month 4
| Milestone | Active Capsule of Obeticholic Acid, Then Placebo | Placebo for Obeticholic Acid, Then Active Drug |
|---|---|---|
| Started | 5 | 5 |
| Completed | 5 | 5 |
| Not completed | 0 | 0 |
| Milestone | Active Capsule of Obeticholic Acid, Then Placebo | Placebo for Obeticholic Acid, Then Active Drug |
|---|---|---|
| Started | 5 | 5 |
| Completed | 5 | 5 |
| Not completed | 0 | 0 |
| Milestone | Active Capsule of Obeticholic Acid, Then Placebo | Placebo for Obeticholic Acid, Then Active Drug |
|---|---|---|
| Started | 5 | 5 |
| Completed | 5 | 5 |
| Not completed | 0 | 0 |
The primary end-point variable was the change in the liver TG content assessed using proton-density fat fraction mapping by Magnetic Resonance Imaging (MRI).
| percentage of liver triglycerides | Baseline (Month 0) | Obeticholic Acid (Month 4) | Placebo (Month 0) | Placebo (Month 4) |
|---|---|---|---|---|
| Change in the Liver Triglycerides (TG). | 13.2 (2.8 to 28.5) | 6.4 (2.4 to 18.0) | 12.3 (3.6 to 33.3) | 10.6 (3.4 to 29.3) |
Change in the serum levels of Triglycerides from baseline to month 4 is being assessed.
| mg/dL | Baseline (Month 0) | Obeticholic Acid (Month 4) | Placebo (Month 0) | Placebo (Month 4) |
|---|---|---|---|---|
| Change in Serum Triglyceride Levels | 199.2 (78 to 392) | 182.3 (102 to 309.3) | 169.3 (103.5 to 562.0) | 161.3 (93 to 655) |
Change in serum levels of Alanine Aminotransferase from baseline to month 4 is assessed
| U/L | Baseline (Month 0) | Obeticholic Acid (Month 4) | Placebo (Month 0) | Placebo (Month 4) |
|---|---|---|---|---|
| Change in Serum Levels of Alanine Aminotransferase | 16.3 (13.5 to 31) | 15.8 (13 to 42.5) | 19.0 (11 to 33.5) | 18.5 (12.6 to 33) |
Change in serum levels of Aspartate Aminotransferases from baseline to month 4 is assessed
| U/L | Baseline (Month 0) | Obeticholic Acid (Month 4) | Placebo (Month 0) | Placebo (Month 4) |
|---|---|---|---|---|
| Change in Serum Levels of Aspartate Aminotransferases | 15.5 (13.5 to 35.5) | 16.8 (13.7 to 27) | 18.7 (13.5 to 28.5) | 16.5 (12.5 to 31) |
Change in serum levels of Gamma-Glutamyl Transpeptidase from baseline to month 4 is assessed
| U/L | Baseline (Month 0) | Obeticholic Acid (Month 4) | Placebo (Month 0) | Placebo (Month 4) |
|---|---|---|---|---|
| Change in Serum Levels of Gamma-Glutamyl Transpeptidase | 27.5 (16.3 to 64) | 17 (10.6 to 45.7) | 21.6 (14.6 to 109) | 21.2 (13.5 to 66) |
Changes in serum Insulin levels is assessed
No measurements were reported for this outcome.
Change in hepatic insulin sensitivity is assessed by suppression of hepatic glucose output during the low-dose and high-dose insulin infusions during the euglycemic clamp study.
No measurements were reported for this outcome.
Collected over 4 months for each intervention separated by a 4-month washout period, approximately 12 months total. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Active Capsule of Obeticholic Acid | 0/10 (0%) | 0/10 (0%) | 4/10 (40%) |
| Placebo for Obeticholic Acid | 0/10 (0%) | 0/10 (0%) | 2/10 (20%) |
| Event | Active Capsule of Obeticholic Acid | Placebo for Obeticholic Acid |
|---|---|---|
| ItchingSkin and subcutaneous tissue disorders | 4/10 | 2/10 |
A total of thirteen subjects were consented in the study. Ten adult female subjects completed the study. Two subjects did not qualify based on the enrollment criteria (screen failed). One subject withdrew due to personal reasons prior to being randomized. Hence only 10 subjects considered enrolled. Population Description: Baseline Characteristics of 10 patients
| Age, Continuous(years) | All Study Participants |
|---|---|
| Mean | 37.5 ± 15 |
| Sex: Female, Male(Participants) | All Study Participants |
|---|---|
| Female | 10 |
| Male | 0 |
| Race (NIH/OMB)(Participants) | All Study Participants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 10 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Liver Fat(percentage of liver fat) | All Study Participants |
|---|---|
| Mean | 13 ± 8 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Obeticholic acid
Lipodystrophy, Familial Partial →