CLINICAL TRIALS PROFILE FOR SIKLOS

All Clinical Trials for Siklos

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01976416 ↗	Novel Use Of Hydroxyurea in an African Region With Malaria	Completed	Children's Hospital Medical Center, Cincinnati	Phase 3	2014-09-01	Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.
NCT01976416 ↗	Novel Use Of Hydroxyurea in an African Region With Malaria	Completed	Doris Duke Charitable Foundation	Phase 3	2014-09-01	Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.
NCT01976416 ↗	Novel Use Of Hydroxyurea in an African Region With Malaria	Completed	Makerere University	Phase 3	2014-09-01	Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.
NCT01976416 ↗	Novel Use Of Hydroxyurea in an African Region With Malaria	Completed	Mulago Hospital, Uganda	Phase 3	2014-09-01	Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.
NCT01976416 ↗	Novel Use Of Hydroxyurea in an African Region With Malaria	Completed	Indiana University	Phase 3	2014-09-01	Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.
NCT02042222 ↗	Novel Dose Escalation to Predict Treatment With Hydroxyurea	Completed	Children's Hospital Medical Center, Cincinnati	Early Phase 1	2013-10-01	Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, chronic anemia (a decrease in the number of red blood cells), and damage to body organs. Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs) and an increase in hemoglobin (the oxygen-carrying protein) in the blood. Patients on hydroxyurea who receive a maximum tolerated dose (MTD) that is specific for them have greater clinical benefit than those who receive a standard lower dose. There is, however, no way currently to predict the MTD for individual patients. As such, MTD for each patient is currently determined by gradual increases in the dose over several months. This process is time-consuming, requires monthly clinic visits, and delays the benefits of hydroxyurea therapy. Our research group has come up with an equation that could be used to predict each patient's MTD using baseline clinical and laboratory measures before starting hydroxyurea treatment. The purpose of this research study is to compare the use of our equation for predicting MTD to the current standard practice of gradually increasing the hydroxyurea dose until MTD is reached. We want to see if the use of our predictive equation will allow us to achieve MTD faster and with no more side effects than with the standard practice.

Clinical Trial Conditions for Siklos

Condition Name

Condition Name for Siklos
Intervention	Trials
Sickle Cell Disease	7
Malaria	2
Sickle Cell Anemia	2
Renal Function Disorder	1

Condition MeSH

Condition MeSH for Siklos
Intervention	Trials
Anemia, Sickle Cell	8
Malaria	2
Anemia	2
Albuminuria	1

Clinical Trial Locations for Siklos

Trials by Country

Trials by Country for Siklos
Location	Trials
France	7
Uganda	3
Germany	1
Italy	1
French Guiana	1

Trials by US State

Trials by US State for Siklos
Location	Trials
Texas	1

Clinical Trial Progress for Siklos

Clinical Trial Phase

Clinical Trial Phase for Siklos
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	3
Phase 2	2

Clinical Trial Status

Clinical Trial Status for Siklos
Clinical Trial Phase	Trials
Completed	6
Recruiting	3

Clinical Trial Sponsors for Siklos

Sponsor Name

Sponsor Name for Siklos
Sponsor	Trials
ADDMEDICA SASA	5
Children's Hospital Medical Center, Cincinnati	3
Makerere University	3

Sponsor Type

Sponsor Type for Siklos
Sponsor	Trials
Other	21
Industry	6
NIH	1

SIKLOS: A Comprehensive Update on Clinical Trials, Market Analysis, and Projections

Introduction to SIKLOS

SIKLOS, the brand name for hydroxyurea, is a crucial medication in the treatment of sickle cell disease (SCD), a genetic disorder that affects the production of hemoglobin. Here, we will delve into the clinical trials, market analysis, and future projections for SIKLOS.

Clinical Trials and FDA Approval

FDA Approval for Pediatric Patients

In December 2017, the FDA granted regular approval to hydroxyurea (SIKLOS) for reducing the frequency of painful crises and the need for blood transfusions in pediatric patients with sickle cell anemia. This approval was based on data from the European Sickle Cell Disease Cohort study (ESCORT HU), which involved 405 pediatric patients aged 2-18 years. The study showed a significant reduction in vaso-occlusive episodes, acute chest syndrome, hospitalizations, and blood transfusions after 12 months of hydroxyurea treatment[1].

Efficacy and Safety

Clinical trials have consistently shown that SIKLOS reduces the frequency of vaso-occlusive crises by 66% to 80% in both children and adults. It also decreases the incidence of acute chest syndrome by 25% to 33% and reduces hospital admissions and days spent in hospital. These effects have been sustained for up to seven years in some studies[4].

Market Analysis

Global Market Size and Growth

The global sickle cell disease treatment market is projected to grow significantly. By 2030, the market is expected to reach $9.84 billion from $2.73 billion in 2023, at a compound annual growth rate (CAGR) of 20.1%[5].

Market Segmentation

The market is segmented by treatment type, with originator drugs holding a larger share in 2022. However, the generic drugs segment is also expected to grow. The pharmacotherapy segment, which includes SIKLOS, is anticipated to grow at the fastest CAGR during the forecast period due to the launch of new branded pharmacotherapy drugs and increasing government initiatives[5].

Distribution Channels

The distribution channels for SCD treatments include direct tender, hospital pharmacies, retail pharmacies, online pharmacies, and others. The direct tender segment held the largest market share in 2022, but online pharmacies are expected to register the highest CAGR due to their convenience, better pricing, and improved consumer compliance[2].

Key Players and Market Competition

Dominant Players

Companies such as Global Blood Therapeutics Inc. (Pfizer Inc.), Novartis AG, and Emmaus Medical, Inc. are prominent players in the SCD treatment market. Global Blood Therapeutics Inc. (Pfizer Inc.) holds a significant market share due to strong sales from Oxbryta, another SCD treatment[5].

Regional Market Share

The U.S. dominates the global SCD treatment market with a 64% share in 2022, attributed to better access to treatment, potential pipeline candidates, strong government support, and rising collaborations. Europe also holds a significant market share and is projected to witness considerable growth due to favorable reimbursement policies and increasing prevalence of the disease[5].

Future Projections

Pharmacotherapy Segment Growth

The pharmacotherapy segment, which includes SIKLOS, is expected to lead the market by treatment modality. The growth is driven by the launch of new branded pharmacotherapy drugs and increasing government initiatives for early launches of these drugs[5].

Online Pharmacies and Direct Tender

Online pharmacies are anticipated to grow significantly due to their convenience and better pricing. The direct tender segment, while currently dominant, will continue to play a crucial role in the distribution of SCD treatments[2].

Global Expansion

The market in regions such as the Middle East, Africa, the Mediterranean, and South America is expected to expand significantly due to the high incidence of SCD and rising disposable income. Increasing awareness about SCD and a strong pipeline of branded drugs will also fuel market growth in these regions[5].

Adverse Reactions and Safety Considerations

Common Adverse Reactions

The most common adverse reactions to SIKLOS include infections and neutropenia, with an incidence of more than 10%[1].

Reporting Adverse Events

Healthcare professionals are advised to report all serious adverse events suspected to be associated with the use of SIKLOS to FDA’s MedWatch Reporting System[1].

Conclusion

SIKLOS has been a game-changer in the treatment of sickle cell disease, particularly with its approval for pediatric patients. The clinical trials have shown significant efficacy in reducing painful crises and other complications associated with SCD.

Key Takeaways

• FDA Approval: SIKLOS was approved by the FDA in 2017 for pediatric patients with sickle cell anemia.
• Clinical Efficacy: SIKLOS reduces vaso-occlusive crises, acute chest syndrome, and hospital admissions.
• Market Growth: The global SCD treatment market is projected to reach $9.84 billion by 2030.
• Pharmacotherapy Segment: This segment is expected to grow at the fastest CAGR due to new drug launches and government initiatives.
• Distribution Channels: Online pharmacies are expected to grow significantly due to their convenience and better pricing.
• Adverse Reactions: Common adverse reactions include infections and neutropenia.

FAQs

What is SIKLOS used for?

SIKLOS (hydroxyurea) is used to reduce the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia.

What was the basis for FDA approval of SIKLOS for pediatric patients?

The FDA approval was based on data from the European Sickle Cell Disease Cohort study (ESCORT HU), which showed a significant reduction in vaso-occlusive episodes and other complications.

What are the common adverse reactions to SIKLOS?

The most common adverse reactions include infections and neutropenia.

How is the global SCD treatment market expected to grow?

The global SCD treatment market is projected to grow from $2.73 billion in 2023 to $9.84 billion by 2030 at a CAGR of 20.1%.

Which companies are dominant in the SCD treatment market?

Global Blood Therapeutics Inc. (Pfizer Inc.), Novartis AG, and Emmaus Medical, Inc. are the prominent players in the market.

Sources

1. FDA: FDA approves hydroxyurea for treatment of pediatric patients with sickle cell anemia.

   • https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-hydroxyurea-treatment-pediatric-patients-sickle-cell-anemia

2. GlobeNewswire: Sickle Cell Disease Treatment Market Worth $4.69 Billion Globally by 2030.

   • https://www.globenewswire.com/news-release/2023/11/08/2775839/0/en/Sickle-Cell-Disease-Treatment-Market-Worth-4-69-Billion-Globally-by-2030-Exclusive-Report-by-The-Insight-Partners.html

3. Grand View Research: Clinical Trials Market Size, Share And Growth Report, 2030.

   • https://www.grandviewresearch.com/industry-analysis/global-clinical-trials-market

4. European Medicines Agency: Siklos | European Medicines Agency (EMA).

   • https://www.ema.europa.eu/en/medicines/human/EPAR/siklos

5. Fortune Business Insights: Sickle Cell Disease Treatment Market Size & Analysis [2030].

   • https://www.fortunebusinessinsights.com/industry-reports/sickle-cell-disease-treatment-market-101009

Last updated: 2025-01-02