CLINICAL TRIALS PROFILE FOR HYDROXYPROGESTERONE CAPROATE

All Clinical Trials for Hydroxyprogesterone Caproate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00099164 ↗	Trial of Progesterone in Twins and Triplets to Prevent Preterm Birth (STTARS)	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 3	2004-04-01	Women pregnant with twins or triplets are at high risk of preterm birth, yet no intervention or approach has served to reduce this risk. A recently completed trial by the NICHD sponsored Maternal Fetal Medicine Units (MFMU) Network has, for the first time, demonstrated a treatment that substantially reduces the rate of preterm birth in women at high risk for preterm delivery (i.e. progesterone therapy). Preterm birth was reduced by 35% among progesterone-treated women with a singleton pregnancy when compared with women receiving placebo. The current trial compares weekly treatment by injection of progesterone with placebo in women pregnant with twins or triplets.
NCT00099164 ↗	Trial of Progesterone in Twins and Triplets to Prevent Preterm Birth (STTARS)	Completed	The George Washington University Biostatistics Center	Phase 3	2004-04-01	Women pregnant with twins or triplets are at high risk of preterm birth, yet no intervention or approach has served to reduce this risk. A recently completed trial by the NICHD sponsored Maternal Fetal Medicine Units (MFMU) Network has, for the first time, demonstrated a treatment that substantially reduces the rate of preterm birth in women at high risk for preterm delivery (i.e. progesterone therapy). Preterm birth was reduced by 35% among progesterone-treated women with a singleton pregnancy when compared with women receiving placebo. The current trial compares weekly treatment by injection of progesterone with placebo in women pregnant with twins or triplets.
NCT00120640 ↗	Treatment of Preterm Labor With 17 Alpha-hydroxyprogesterone Caproate	Withdrawn	Yale University	N/A	2005-07-01	The goal of our research will be to determine the effectiveness of 17 alpha-hydroxyprogesterone caproate (17P) in the treatment of preterm delivery. Treatment with progesterone is emerging as the standard of care for prevention of preterm delivery in asymptomatic patients at high risk for preterm birth due to a prior preterm delivery. Our goal is to evaluate whether or not progesterone is also effective in reducing preterm birth in symptomatic patients.
NCT00135902 ↗	Omega-3 Fatty Acid Supplementation to Prevent Preterm Birth in High Risk Pregnancies	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 3	2005-02-01	A recently completed trial of weekly injections of 17 alpha hydroxyprogesterone caproate (17P) found significant effectiveness for 17P in preventing recurrent preterm birth. However, the group who received 17P in this trial still had a high rate of preterm birth. Several reports have shown that dietary supplementation of fish oil, which is rich in Omega-3 fatty acids, reduces the risk of preterm birth. This trial tests whether adding the Omega-3 supplement to 17P therapy has the potential for further reducing the risk of preterm birth in women who have previously had a spontaneous preterm delivery. The trial will compare Omega-3 fatty acid with placebo in women receiving 17P therapy. The hypothesis being tested is: "Among women at high risk for preterm birth receiving weekly injections of 17P, the addition of Omega-3 nutritional supplement will further reduce the rate of preterm birth."

Clinical Trial Conditions for Hydroxyprogesterone Caproate

Condition Name

Condition Name for Hydroxyprogesterone Caproate
Intervention	Trials
Preterm Birth	7
Pregnancy	4
Premature Birth	4
Preterm Delivery	3

Condition MeSH

Condition MeSH for Hydroxyprogesterone Caproate
Intervention	Trials
Premature Birth	17
Rupture	3
Obstetric Labor, Premature	3
Placenta Previa	3

Clinical Trial Locations for Hydroxyprogesterone Caproate

Trials by Country

Trials by Country for Hydroxyprogesterone Caproate
Location	Trials
United States	118
Hungary	6
Egypt	5
Canada	4
Czech Republic	4

Trials by US State

Trials by US State for Hydroxyprogesterone Caproate
Location	Trials
Texas	9
Pennsylvania	8
California	7
Utah	6
North Carolina	6

Clinical Trial Progress for Hydroxyprogesterone Caproate

Clinical Trial Phase

Clinical Trial Phase for Hydroxyprogesterone Caproate
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	9
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for Hydroxyprogesterone Caproate
Clinical Trial Phase	Trials
Completed	17
Recruiting	5
Unknown status	3

Clinical Trial Sponsors for Hydroxyprogesterone Caproate

Sponsor Name

Sponsor Name for Hydroxyprogesterone Caproate
Sponsor	Trials
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	7
AMAG Pharmaceuticals, Inc.	6
The George Washington University Biostatistics Center	3

Sponsor Type

Sponsor Type for Hydroxyprogesterone Caproate
Sponsor	Trials
Other	31
Industry	11
NIH	8

Hydroxyprogesterone Caproate: Clinical Trials Update, Market Analysis, and Projections

Introduction

Hydroxyprogesterone caproate (17-OHPC), a synthetic form of hydroxyprogesterone, has been widely used to prevent premature birth and other pregnancy-related complications. However, recent developments have significantly impacted its clinical use and market standing. This article provides an update on the clinical trials, market analysis, and projections for hydroxyprogesterone caproate.

Clinical Trials Update

Efficacy Concerns

Recent studies have raised substantial concerns about the efficacy of 17-OHPC in preventing premature birth. A large study involving over 1,700 pregnant women with a history of preterm delivery found that 17-OHPC was no more effective than a placebo in preventing recurrent premature birth or medical complications due to prematurity in newborns[1][4].

Safety Concerns

In addition to efficacy issues, there are also safety concerns associated with 17-OHPC. The European Medicines Agency's (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) reviewed a large population-based study that suggested people exposed to 17-OHPC in the womb may have an increased risk of cancer. Although the study had limitations and a low number of cancer cases, it was enough to raise a "possible but unconfirmed risk of cancer"[1][4].

Regulatory Actions

Following these findings, the EMA recommended the suspension of the marketing authorizations for medicines containing 17-OHPC in the European Union. This decision was based on the conclusion that the benefits of 17-OHPC do not outweigh its risks in any authorized use[1][4].

Market Analysis

Current Market Size

As of 2023, the global hydroxyprogesterone caproate market size was approximately USD 0.27 billion. Despite the recent regulatory setbacks, the market is projected to grow, albeit at a slow pace[2].

Market Segmentation

The hydroxyprogesterone caproate market is segmented based on the type of formulation (1 ml and 5 ml injections) and application (hospitals and clinics). The market has seen significant use in hospitals and clinics, particularly for preventing pregnancy loss and premature birth[2].

Driving Factors

The market has been driven by factors such as rising maternal age and growing awareness about preterm birth prevention. However, the lack of efficacy and safety concerns have dampened the growth prospects[2].

Market Projections

Growth Rate

The hydroxyprogesterone caproate market is expected to exhibit a Compound Annual Growth Rate (CAGR) of 1.5% by 2032. This modest growth is largely due to the availability of alternative treatments and the ongoing regulatory challenges[2].

Future Market Size

By 2032, the market is projected to reach USD 0.30 billion. This growth, although slow, indicates that while the drug may not be as dominant as it once was, it still maintains some market presence[2].

Impact of Regulatory Actions

FDA and EMA Decisions

In April 2023, the FDA pulled its approval for Covis Pharma’s preterm birth prevention drug Makena, which contains 17-OHPC, due to its lack of efficacy. Similarly, the EMA's recommendation to suspend the marketing authorizations for 17-OHPC medicines in the EU has further reduced the drug's market viability[1][4].

Alternative Treatments

The EMA and FDA have highlighted that alternative treatments are available for preventing preterm birth, including progesterone, tocolytics, and corticosteroids. These alternatives are likely to capture a larger share of the market as 17-OHPC's use diminishes[1][4].

Historical Use and Trends

Usage Patterns

Historical data shows that the use of hydroxyprogesterone caproate increased significantly from less than 11,000 fills per quarter between 2010 and 2014 to more than 30,000 fills per quarter in the first quarter of 2019. However, following the announcement of Makena’s failed postapproval trial, the use declined substantially[5].

Brand vs. Generic

Despite the availability of generic versions since 2018, brand-name Makena has maintained a significant market share, particularly due to its newer branded autoinjector formulation. This has resulted in continued high spending on hydroxyprogesterone caproate[5].

Expert Insights and Future Directions

Lack of Innovation

Experts in maternal and fetal health have expressed concerns about the lack of innovation in drug development to prevent spontaneous preterm birth. This lack of innovation, combined with the efficacy and safety issues of 17-OHPC, underscores the need for new and effective treatments[4].

Patient and Healthcare Provider Perspectives

Patient representatives and healthcare providers have been involved in the review process, emphasizing the importance of safe and effective treatments. The shift towards alternative treatments is expected to be driven by both regulatory recommendations and patient safety concerns[1].

Key Takeaways

• Efficacy and Safety Concerns: Recent studies have shown that 17-OHPC is not effective in preventing premature birth and may pose a risk of cancer to those exposed in the womb.
• Regulatory Actions: The EMA and FDA have taken steps to limit or suspend the use of 17-OHPC due to these concerns.
• Market Projections: Despite regulatory setbacks, the market is expected to grow at a CAGR of 1.5% to reach USD 0.30 billion by 2032.
• Alternative Treatments: Progesterone, tocolytics, and corticosteroids are being recommended as alternative treatments for preventing preterm birth.
• Need for Innovation: There is a pressing need for innovative and effective treatments to prevent spontaneous preterm birth.

FAQs

What is the current status of hydroxyprogesterone caproate in the European Union?

The EMA has recommended the suspension of the marketing authorizations for medicines containing 17-OHPC in the European Union due to concerns over its efficacy and safety[1][4].

Why has the FDA pulled its approval for Makena?

The FDA pulled its approval for Makena in April 2023 because studies showed that the drug was not effective in preventing premature birth[4].

What are the alternative treatments available for preventing preterm birth?

Alternative treatments include progesterone, tocolytics, and corticosteroids, which are recommended to slow contractions and speed up lung development before delivery[1][4].

How has the market size of hydroxyprogesterone caproate been affected by recent developments?

The market size has been impacted by regulatory actions, but it is still projected to grow at a CAGR of 1.5% to reach USD 0.30 billion by 2032[2].

What are the key driving factors for the hydroxyprogesterone caproate market?

The market is driven by factors such as rising maternal age and growing awareness about preterm birth prevention, although these are now tempered by efficacy and safety concerns[2].

Sources

1. European Pharmaceutical Review: EMA recommends suspension of hydroxyprogesterone caproate medicines[1].
2. Business Research Insights: Hydroxyprogesterone Caproate Market Size - Forecast To 2032[2].
3. FDA Presentation: Vaginal progesterone compared with intramuscular 17-alpha-hydroxyprogesterone caproate for prevention of recurrent preterm birth[3].
4. BioSpace: EU Regulator Pulls Approval for Preterm Labor Drug One Year After FDA[4].
5. JAMA Network: Changes in the Use of Hydroxyprogesterone Caproate Injection After Confirmatory Trial Failure[5].