CLINICAL TRIALS PROFILE FOR OXANDROLONE

All Clinical Trials for Oxandrolone

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001221 ↗	Effect of Biosynthetic Growth Hormone and/or Ethinyl Estradiol on Adult Height in Patients With Turner Syndrome	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	1987-09-01	Turners Syndrome is a genetic condition in females that is a result of abnormal chromosomes. Girls with Turner syndrome are very short as children and as adults. Although their growth hormone secretion is almost always normal, giving injections of growth hormone to Turner syndrome girls may increase their rate of growth. In addition, most girls with Turner syndrome do not have normal ovaries. In normal girls the ovaries begin producing small amounts of the female sex hormone, estrogen at about 11 - 12 years of age. As girls grow older the level of estrogen increases. Estrogen is responsible for the changes in girls known as feminization. During feminization the hips grow wider, the breasts develop, there is an increase in the rate of growth, and eventually girls experience their first menstrual period. This study was designed to evaluate the effect of low dose estrogen, growth hormone, and the combination of low dose estrogen and growth hormone on adult height in girls with Turner syndrome. Patients will be entered into the study from ages 5 to 12 and will be randomly placed into one of four groups. 1. Group one will receive low dose estrogen 2. Group two will receive growth hormone 3. Group three will receive both low dose estrogen and growth hormone 4. Group four will receive a placebo "sugar pill" Once started, the treatment will continue until the patients approach their adult height, and growth slows to less than 1/2 inch over the preceding year. This usually occurs by the age of 15 or 16. Patients will be seen at the outpatient clinic every 6 months during the study and will receive a routine check-up with blood and urine tests, and hand/wrist X-rays to determine bone age. On patient's yearly visits they will have the density of bone measured in their spine and forearm.
NCT00001343 ↗	The Effects of Hormones in Growth Hormone-Treated Girls With Turner Syndrome	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	1992-12-11	Turners Syndrome is a genetic condition in females that is a result of abnormal chromosomes. Patients with Turner syndrome are typically short, have abnormal physical features, and lack the physical changes normally associated with puberty. In addition, some patients with Turner syndrome have low bone density (osteoporosis) and differences in learning abilities. This study will research the effects of steroid hormones on patients with Turner syndrome. It will look closely at how taking steroid hormones effects the patient's rate of growth as well as the patient's ability to learn. In addition the study will investigate how different hormones (androgen and estrogen) work when given together as a combination. All patients asked to participate in this study will receive growth hormone injections. However, half of the patients will receive an additional sex steroid hormone (oxandrolone) in the form of a pill. The other half of the patients will receive a placebo or "sugar pill". This will allow the researchers to determine if the combination of the hormones produces different results than growth hormone alone. The study will last approximately 2 years. After 2 years of research the patients may qualify for an additional 2 years of treatment. Patients may benefit directly from this research with increased growth and improved ability to learn.
NCT00004275 ↗	Oxandrolone Compared With a Placebo on Growth Rate in Girls With Growth Hormone-Treated Turner's Syndrome	Completed	Jefferson Medical College of Thomas Jefferson University	Phase 2	1999-10-01	RATIONALE: Turner's syndrome is a disease in which females are missing all or part of one X chromosome and do not produce the hormones estrogen and androgen. Giving growth hormone may help girls with Turner's syndrome attain a more normal height. It is not yet known if growth hormone is more effective with or without oxandrolone for Turner's syndrome. PURPOSE: Randomized phase II trial to study the effectiveness of oxandrolone in girls who have growth hormone-treated Turner's syndrome.
NCT00004275 ↗	Oxandrolone Compared With a Placebo on Growth Rate in Girls With Growth Hormone-Treated Turner's Syndrome	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	1999-10-01	RATIONALE: Turner's syndrome is a disease in which females are missing all or part of one X chromosome and do not produce the hormones estrogen and androgen. Giving growth hormone may help girls with Turner's syndrome attain a more normal height. It is not yet known if growth hormone is more effective with or without oxandrolone for Turner's syndrome. PURPOSE: Randomized phase II trial to study the effectiveness of oxandrolone in girls who have growth hormone-treated Turner's syndrome.
NCT00006167 ↗	Nutrition Intervention in AIDS Wasting	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 4	1998-01-01	There are no guidelines for appropriate nutritional management of weight loss or wasting in HIV infection. Some treatments may increase weight, but without improving muscle mass or quality of life. In this clinical trial AIDS patients with wasting are randomized to one of three nutritional strategies and studied over a 12-week period: 1) optimal oral nutrition with counseling and protein and calorie supplementation, and a placebo pill; 2) optimal oral nutrition with the oral androgen, oxandrolone at 20 mg daily; and 3) optimal oral nutrition with progressive resistance training (PRT). In all participants, dietary intervention is maximized by weekly personalized counseling to address individual issues and concerns. Two primary outcomes are assessed: thigh muscle mass and quality of life. Our findings can be used to develop guidelines for standards of nutritional care among AIDS patient with the wasting syndrome.
NCT00027391 ↗	Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)	Completed	FDA Office of Orphan Products Development	N/A	2001-09-01	This is a study to determine whether albuterol or oxandrolone, alone or in combination, are able to increase strength and muscle mass in patients with FSHD. It also will determine if albuterol given in "pulsed" fashion will have more effect than when given continuously.

Clinical Trial Conditions for Oxandrolone

Condition Name

Condition Name for Oxandrolone
Intervention	Trials
Turner's Syndrome	3
Burns	2
Muscular Dystrophies	1
Dwarfism	1

Condition MeSH

Condition MeSH for Oxandrolone
Intervention	Trials
Syndrome	4
Wounds and Injuries	3
Turner Syndrome	3
Primary Ovarian Insufficiency	3

Clinical Trial Locations for Oxandrolone

Trials by Country

Trials by Country for Oxandrolone
Location	Trials
United States	43
Brazil	1
France	1
Puerto Rico	1
Pakistan	1

Trials by US State

Trials by US State for Oxandrolone
Location	Trials
Ohio	5
Texas	4
Pennsylvania	4
Utah	3
Florida	3

Clinical Trial Progress for Oxandrolone

Clinical Trial Phase

Clinical Trial Phase for Oxandrolone
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	3
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for Oxandrolone
Clinical Trial Phase	Trials
Completed	14
Not yet recruiting	3
Terminated	2

Clinical Trial Sponsors for Oxandrolone

Sponsor Name

Sponsor Name for Oxandrolone
Sponsor	Trials
FDA Office of Orphan Products Development	2
National Institutes of Health (NIH)	2
The University of Texas Medical Branch, Galveston	2

Sponsor Type

Sponsor Type for Oxandrolone
Sponsor	Trials
Other	21
NIH	10
U.S. Fed	9

Oxandrolone: Clinical Trials, Market Analysis, and Projections

Introduction

Oxandrolone, a synthetic anabolic steroid, has been used in various medical contexts, including the treatment of muscle loss, bone pain in osteoporosis, and recently, in the management of severe burn injuries. This article will delve into the current state of clinical trials, market analysis, and future projections for oxandrolone.

Clinical Trials and Efficacy

Use in Burn Patients

Recent studies, such as the post hoc analysis of the RE-ENERGIZE trial, have explored the efficacy of oxandrolone (OX), beta blockers (BB), and the combination of both (BBOX) in severely burned adults. The trial involved 1200 adult patients and showed that the use of OX, BB, and BBOX was common, although with variable usage across different sites worldwide. The study found mixed associations between these treatments and patient outcomes, with beta blockers associated with lower in-hospital and 6-month mortality, while oxandrolone was linked to longer times to discharge alive[1].

Pediatric Burn Patients

In pediatric burn patients, oxandrolone has been shown to have positive effects on hypermetabolism, catabolism, and hyperinflammation. A study published in the Journal of Burn Care & Research demonstrated that long-term administration of oxandrolone improved lung function, increased height percentile, bone mineral content, lean body mass, and strength in severely burned pediatric subjects[3].

Market Analysis

FDA Approval and Withdrawal

Oxandrolone, marketed under the brand name Oxandrin, was initially approved by the FDA in 1964 for relieving bone pain in osteoporosis. However, in June 2023, the FDA withdrew its approval for oxandrolone tablets due to serious potential problems associated with the drug, including peliosis hepatitis, live cell tumors, and blood lipid changes that increase the risk of atherosclerosis[2].

Current Market Status

The withdrawal of FDA approval significantly impacts the market for oxandrolone. Despite its historical use, the drug is no longer approved for any indications in the United States. This move is likely to reduce its global market presence and availability.

Safety and Adverse Effects

Known Risks

Oxandrolone is associated with several serious adverse effects, including peliosis hepatitis, live cell tumors, blood lipid changes, cholestatic hepatitis, hypercalcemia in patients with breast cancer, and an increased risk of prostatic carcinoma and prostatic hypertrophy in geriatric patients. These risks were a key factor in the FDA's decision to withdraw its approval[2].

Mechanism of Action and Uses

Anabolic Properties

Oxandrolone interacts with androgen receptors in target tissues, promoting anabolic effects such as weight gain, muscle growth, and bone density improvement. It is used as adjunctive therapy to promote weight gain after extensive surgery, chronic infections, or severe trauma, and to offset protein catabolism associated with prolonged corticosteroid administration[5].

Future Projections

Research and Development

Given the recent withdrawal of FDA approval, future research on oxandrolone may focus on mitigating its adverse effects or exploring alternative anabolic steroids with better safety profiles. However, the drug's potential benefits in specific medical conditions, such as severe burn injuries, may still be investigated in clinical trials outside the U.S. market.

Global Market Impact

The global market for oxandrolone is expected to decline significantly following the FDA's withdrawal of approval. Manufacturers may need to seek approvals in other countries or reformulate the drug to address the safety concerns raised by the FDA.

Regulatory Environment

FDA and International Regulations

The FDA's decision sets a precedent for regulatory bodies in other countries to reevaluate the safety and efficacy of oxandrolone. International regulatory agencies may follow suit, leading to a global reduction in the drug's availability and use.

Key Takeaways

• Clinical Trials: Oxandrolone has shown mixed results in clinical trials, particularly in the management of severe burn injuries.
• Market Analysis: The FDA's withdrawal of approval significantly impacts the global market for oxandrolone.
• Safety Concerns: Serious adverse effects associated with oxandrolone led to its withdrawal from the market.
• Future Projections: Research may focus on alternative anabolic steroids, and the global market is expected to decline.
• Regulatory Environment: The FDA's decision may influence regulatory actions in other countries.

FAQs

Q: What is oxandrolone used for?

A: Oxandrolone is used to treat muscle loss from prolonged corticosteroid treatment, bone pain associated with osteoporosis, and as adjunctive therapy to promote weight gain after severe trauma or surgery[5].

Q: Why was the FDA approval for oxandrolone withdrawn?

A: The FDA withdrew its approval due to serious potential problems associated with the drug, including liver issues, live cell tumors, and blood lipid changes[2].

Q: What are the adverse effects of oxandrolone?

A: Adverse effects include peliosis hepatitis, live cell tumors, blood lipid changes, cholestatic hepatitis, hypercalcemia, and increased risk of prostatic carcinoma and prostatic hypertrophy[2].

Q: Is oxandrolone still used in clinical trials?

A: Yes, oxandrolone may still be used in clinical trials, particularly in the context of severe burn injuries, although its use is expected to be more limited following the FDA's withdrawal of approval.

Q: How does the FDA's decision impact the global market for oxandrolone?

A: The FDA's decision is likely to lead to a global reduction in the availability and use of oxandrolone, as other regulatory bodies may follow suit in reevaluating the drug's safety and efficacy.

Sources

1. Burns & Trauma, "A post hoc analysis of the RE-ENERGIZE trial," 2024.
2. Horizon Blue, "The FDA has withdrawn approval for OXANDRIN (oxandrolone) tablets," 2023.
3. Journal of Burn Care & Research, "Long-Term Administration of Oxandrolone Improves Lung Function in Severely Burned Pediatric Subjects," 2016.
4. ClinicalTrials.gov, "OXANDROLONE."
5. DrugBank, "Oxandrolone: Uses, Interactions, Mechanism of Action."