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Clinical Trials

Clinical trials are crucial to the development of safe and effective treatments for our patients. Our goal is to broaden our efforts in the rare disease space, striving to improve our products. Below is our list of studies currently recruiting in the US. For information on all Clinical Trials that are being conducted you can go to clinicaltrials.gov for futher information.

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Endocrinology

CUSHING’S SYNDROME

A PHASE II, MULTICENTER, OPEN-LABEL, NON-COMPARATIVE STUDY TO EVALUATE THE PHARMACOKINETICS, PHARMACODYNAMICS, AND TOLERABILITY OF OSILODROSTAT IN CHILDREN AND ADOLESCENT PATIENTS WITH CUSHING’S SYNDROME. (LINC 5)

Study Description: Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing’s syndrome.

Therapeutic area: Endocrinology

Phase: 2

Status: Recruiting

Ages Eligible for the Study: 2 to <18 years old

Sexes Eligible for the Study: All

Locations:

   ABMED Clinical Research Corp
   Cape Coral, Florida, United States
   Contact: Dislandys Acosta,
   Principal Investigator: Dr. Paige Vanier Kreegel

   Ann & Robert H. Lurie Children’s Hospital
   Chicago, Illinois, United States
   Contact: Emily Marshall,
   Principal Investigator: Dr. Wendy Brickman

   Texas Valley Clinical Research
   San Francisco, California, United States
   Contact: Luis Gay,
   Principal Investigator: Maya Lodish, MD

   The Regents of the University of California, San Francisco
   Weslaco, Texas, United States
   Contact: Preston Hale,
   Principal Investigator: Dr. Eduardo Dusty Luna

 

ClinicalTrials.gov link: NCT03708900

Oncology

Metabolic

HYPERAMMONEMIA DUE TO ORGANIC ACIDEMIAS: PROPIONIC ACIDEMIA, METHYLMALONIC ACIDEMIA

A NON-INTERVENTIONAL POST-AUTHORIZATION SAFETY STUDY (PASS) OF CARBAGLU® FOR THE TREATMENT OF HYPERAMMONEMIA DUE TO METHYLMALONIC ACIDEMIA (MMA) AND PROPIONIC ACIDEMIA (PA) IN ADULT AND PEDIATRIC PATIENT POPULATIONS

Study Description

This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice.

Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life.

Therapeutic area: Metabolic

Phase: 4

Status: Recruiting

Ages Eligible for the Study: All

Sexes Eligible for the Study: All

Locations:

     Children’s National Hospital
     Washington, District of Columbia, United States
     Contact: Kara Simpson, 202-545-2503,
     Principal Investigator: Nicholas Ah Mew, MD

     University of South Florida
     Tampa, Florida, United States
     Contact: Farideh Oberheu, 813-250-2292,
     Principal Investigator: Amarilis Sanchez-Valle, MD

     Ann & Robert H. Lurie Children’s Hospital of Chicago
     Chicago, Illinois, United States
     Contact: Michael Sawin, 312-227-2816,
     Principal Investigator: Joshua Baker, MD

     Riley Children’s Hospital
     Indianapolis, Indiana, United States
     Contact: Susan Romie, 317-278-6650,
     Principal Investigator: Melissa Lah, MD

     Icahn School of Medicine at Mt. Sinai
     New York, New York, United States
     Contact: Colleen Donnelly, 212-241-5983,
     Principal Investigator: Margo Breilyn, MD

 

ClinicalTrials.gov link: NCT05040178

 

HYPERAMMONEMIA DUE TO UREA CYCLE DISORDERS: N-ACETYLGLUTAMATE SYNTHASE (NAGS) DEFICIENCY

ORPHAN EUROPE CARBAGLU® SURVEILLANCE PROTOCOL

Study Description

The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Much of the FDA-required data is already collected through the Longitudinal Study of Urea Cycle Disorders (RDCRN Protocol #5101). This study will collect additional data on adverse events (interim events), adverse reactions, pregnancy, and fetal outcomes.

Therapeutic area: Metabolic

Phase: 4

Status: Recruiting

Ages Eligible for the Study: Child, Adult, Older Adult

Sexes Eligible for the Study: All

Locations:

     Children’s National Hospital
     Washington, District of Columbia, United States
     Contact: Kara Simpson, 202-545-2503,
     Principal Investigator: Nicholas Ah Mew, MD
     Children’s Hospital Boston (UCDC New England Center)
     Boston, Massachusetts, United States
     Contact: Debbie Fu, 617-919-7631,
     Principal Investigator: Gerry Berry, MD
     Icahn School of Medicine at Mt. Sinai
     New York, New York, United States
     Contact: Alison Horn, 212-659-8540,
     Principal Investigator: Margo Breilyn, MD

 

ClinicalTrials.gov link: NCT03409003

Investigator-Sponsored Studies

Who is eligible to receive support:

Recordati Rare Diseases (RRD) is dedicated to supporting independent research that advances medical and scientific understanding related to RRD products or therapeutic areas of interest that leads to promising medical findings. RRD may offer study drugs and/or financial support for Investigator-Sponsored Studies (ISS), subject to approval by the RRD ISS Committee. Support for an ISS will only be provided if the study is aimed to meet a legitimate and genuine medical need.


What RRD can support:

RRD may support an approved study with funding and/or RRD medicinal product. RRD ensures safety reporting requirements are communicated to the investigator or institution prior to the start of an ISS and as needed throughout the duration of the study. RRD ensures that current information for the investigator or institution is available for distributing safety-related information. Any support provided by RRD to an ISS will represent fair market value remuneration for the research services performed.


How to submit an Investigator-Sponsored Study request

Requestors should send written requests for grants, including a budget, to the designated ISS email inbox. The ISS Review Committee at RRD will collectively review concept submission based on scientific merit and alignment with corporate research and development plans. The requestor will be informed about the outcome; if the concept submission is approved, the investigator will be invited to submit a final ISS protocol and budget to be considered for full approval. RRD requires that the following documents are in place before ISS support may be provided:

  • A fully executed ISS agreement between the sponsor and RRD
  • An EC/IRB and/or Health Authority approval

For ISS inquiries and submissions please email:

Scientific Library

Explore select presentations and posters from Congress events and access publications related to Recordati Rare Diseases products categorized by therapeutic area.
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Educational Grants

RRD is dedicated to supporting Corporate Social Responsibility through our Grants. We provide educational and scientific research grants to support initiatives that advance medical knowledge, improve the quality of health care, and patient outcomes.
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Medical Inquiries

Use this form to submit your medical inquiry. Pharmacists from our Medical Information team will respond to your request as quickly as possible.
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This Website is intended to help health care professionals in the United States and its territories find scientifically-balanced information and educational resources related to RRD treatments and therapeutic areas of interest, including clinical data, research developments, congress materials and publications. The Website is made available by RRD’s Medical Affairs organization and is provided as an educational resource for informational, non-promotional and scientific exchange purposes.

 

This Website may contain information about products or uses that have not been approved by the U.S. Food and Drug Administration. RRD does not recommend the use of its products other than as described in the approved prescribing information. The content of this Website is not medical advice and does not replace independent medical judgment. Healthcare providers are responsible for individual patient care on the basis of their professional knowledge, experience, and licensure.

 

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A clean environment is essential for people’s well-being: the health of the planet and the health of people is tightly interconnected. Environmental elements, such as air, water, land and climate, all have an impact on the well-being of humans. Placing a focus on people’s health and being sustainable therefore also means prioritising environmental protection and a responsibility towards future generations. This is why the Group ensures that it conducts business in a socially responsible manner and in accordance with sustainable practices, national and international laws, and the expectations of stakeholders.
Recordati for the community
We believe that contributing to the well-being of the community and dedicating part of our resources to acts of solidarity is not merely the fulfilment of company obligations or professional duty, but rather a moral imperative, an essential part of a healthy business capable of growth but at the same time able to support and develop the community in which it operates and make its employees proud.
Value and diversity of our people
At Recordati, we believe in equal opportunities and we guarantee that everyone can achieve their potential. We see diversity as a value and will not tolerate any discrimination based on ethnicity, nationality, gender, sexual orientation, disability, age, political or religious belief, or any other personal characteristics. At Recordati, we work hard to create a safe and inclusive work environment, where we all have our rights to physical and psychological integrity respected on a daily basis, as well as our right to freedom of opinion and association. We recognise that we each have a role to play in the success of our business and we implement staff development policies through which everyone’s contribution and achievements can be appropriately rewarded.
Multiple opportunities
Recordati is uniquely structured to bring treatment options across specialty and primary care, consumer healthcare, and rare diseases. We have fully integrated operations across research and development, chemical and finished product manufacturing through to commercialisation and licensing.
Luigi Longinotti
Managing Director and General Manager EMEA

Luigi Longinotti is Managing Director and General Manager for EMEA at Recordati Rare Diseases, leading the European and Middle Eastern Commercial Operations. He is responsible for Marketing, Medical, Market Access, Regulatory, Manufacturing and Logistics, as well as the enabling functions to support the business, including Compliance and Quality.

 

He joined Recordati in July 2014 as Corporate Portfolio Management Director for Orphan Drugs. In such a role he has been responsible for driving the growth of the rare disease portfolio at Recordati, with his duties ranging from business development to strategic marketing and pipeline project management.

 

Before he joined Recordati, he held senior business and corporate development roles at Chiesi Farmaceutici and Menarini Group where he executed several key asset deals and transactions.

Luigi gained a degree in Economics from the University of Florence and a Master’s degree in Business Innovation from Sant’Anna School of Advanced Studies in Pisa.

Bruno Parenti
Head of LAC Region, Head of Global Endocrinology Franchise

Bruno Parenti is Head of LAC Region at Recordati Rare Diseases, leading the business in Latin America, Asia Pacific and Russia. He has been in this position since 2011, where he spearheaded the expansion of operations around the globe. He recently assumed the additional role of Head of Global Endocrinology Franchise, establishing the new HQ in Basel and building a dedicated organization.

 

Prior to these positions, Bruno spent two years at Recordati Ireland overseeing international sales in the Asia-Pacific region. He served at Chiesi Farmaceutici for two years as Area Manager for the Far East. Earlier in his career, he held commercial roles at Kedrion Biopharmaceuticals where he was responsible for international markets with a focus on Latin America and the Middle East.

Mohamed Ladha
President and General Manager, North America

Mohamed Ladha is the President and General Manager at Recordati Rare Diseases where he is   responsible for the US and Canadian business. During his career,  Mohamed has held numerous leadership positions globally in hematology/oncology and specialty care to oversee business in the US, Canada, EU, China, Emerging Markets, and Japan.

 

Mohamed  joins Recordati Rare Diseases from Oncopeptides, where he was the General Manager and Executive Vice President for the US Region Business Unit. Prior to Oncopeptides, he served in a series of leadership positions of increasing responsibility at companies including Vertex Pharmaceuticals, Pfizer, Schering-Plough, Merck & Company, Hospira, ARIAD Pharmaceuticals, Takeda Oncology, and Tocagen. He started his career in basic science research focused in oncology at the Dana-Farber Cancer Institute.

 

Mohamed graduated from Hampshire College with a Bachelor of Arts degree. He also holds professional/graduate degrees from Harvard University’s Kennedy School of Government and Northwestern University’s Kellogg School of Management.

 

Changing to first name to be consistent with bio below, also feel that first name as opposed to Mr has a more informal and friendly tone.   [EC1]

Scott Pescatore
Executive Vice President

Scott Pescatore is Vice President and Head of Global Operations at Recordati Rare Diseases.

He holds a Doctor of Pharmacy degree and completed his post-doctoral fellowship in Pharmacology and Drug Development. Dr Pescatore has spent over 20 years working internationally in the pharmaceutical industry, specializing in oncology, haematology and rare diseases.

 

He joined Novartis Oncology US in 2001 where he served in various medical, sales and marketing roles of increasing responsibility. In 2008, he moved to Novartis Oncology UK as Business Franchise Head for solid tumours where he also managed the New Products portfolio. In 2010 he moved to Milan to manage the Region Europe Haematology Franchise where he led the joint venture between Novartis Oncology and Incyte to launch a novel treatment for myeloproliferative disorders. In 2014 he was appointed the Oncology General Manager in Ireland and after three years returned to Milan as General Manager of the Region Europe Rare Disease Business Unit, overseeing operations in 37 markets and focusing on the endocrinology portfolio.

 

Prior to joining Recordati Rare Diseases in 2020, he was Vice President Oncology Business Unit for AstraZeneca Italy where he was responsible for the portfolio of oncology/haematology products including two joint ventures with MSD and Daiichi Sankyo.