Thalassemia

Sickle Cell

A life beyond thalassemia or sickle cell disease is worth fighting for.

If you have been diagnosed with thalassemia or transfusion-dependent sickle cell disease (SCD), the Gladiolus Study™ may interest you.

Find a participating site

This Phase 2, open-label clinical research study is investigating the safety and effectiveness of a once-daily oral investigational medication (taken as two tablets) that may improve anemia and reduce the need for red blood cell (RBC) transfusions. All participants enrolled in this study will receive the investigational medication, which means no participants will receive a placebo (an inactive substance also known as a “sugar pill”).

We are currently enrolling participants who meet the following criteria:*

Are 12 to 65 years of age.
Have one of the following diagnoses:
- SCD and receiving regular RBC transfusions to prevent stroke or recurrence of stroke (Group A).
- Thalassemia and receiving regular RBC transfusions to prevent symptomatic anemia (Group B).
- Thalassemia and not receiving regular RBC transfusions (Group C).

*There are additional eligibility requirements, which the investigator will explain to you.

About the Gladiolus Study

For the transfusion-dependent sickle cell disease (SCD) and thalassemia groups (Groups A and B), the Gladiolus Study is testing how well the investigational medication works to reduce the number of red blood cell (RBC) transfusions required during the study treatment period compared to the number of RBC transfusions needed prior to taking part in the study. For the thalassemia participants not receiving regular RBC transfusions (Group C), the Gladiolus Study is testing how well the investigational medication works to improve hemoglobin levels and anemia.

The Gladiolus Study will also assess:

The safety of the investigational medication based on side effects, laboratory tests, physical exams, and other clinical measures in the study.
The effects of the investigational medication on the amount of iron in the body.

How the investigational medication behaves in the body (called pharmacokinetics [PK]) during the study.
How the investigational medication impacts participants’ quality of life.

The total length of participation is up to 58 weeks, which is just over one year. During this time, participants will attend approximately 14 study visits over three periods: the screening period, the study treatment period, and an end-of-study visit four weeks after the last dose of the investigational medication.

Screening

This period lasts up to six weeks and includes one visit to the study site. During this time, the study team will confirm eligibility for enrollment into the study for interested individuals.

Open-label study treatment

This period lasts up to 48 weeks and requires approximately 12 visits to the study site. “Open-label” means participants and the study team will know what study treatment the participant received. All participants receive the investigational medication in this study.

End-of-study visit

This visit happens approximately four weeks after the last dose of the investigational medication. This visit is important because the study team will check on participants’ health after stopping the investigational medication.

Throughout the study, the investigator will perform a number of tests and procedures, including but not limited to:

A physical exam

This will monitor the participant’s overall health while they take part in the study.

An electrocardiogram (ECG)

This will measure the participant’s heart rhythm and electrical activity to make sure the heart is functioning correctly.

Collection of blood samples

The study team will collect blood samples to see how the participant’s body is reacting to the investigational medication, as well as to monitor the participant’s health and safety.

A pregnancy test (if applicable)

If a female participant is of childbearing age, a sample of their blood and/or urine will be collected and tested to check if they are pregnant – women who are pregnant will not be able to participate in the study.

Questionnaires

The participant will be asked to complete questionnaires to record any changes in their condition and answer questions about their quality of life.

Collection of urine samples

Routine laboratory tests will be run to monitor the participant’s health.

Find a participating site

Children’s Hospital Los Angeles

4650 Sunset Boulevard
Los Angeles, CA 90027

Obdulio Carreras

UCLA – David Geffen School of Medicine at UCLA

10833 Le Conte Avenue
42-121 CHS
Los Angeles, CA 90095

Bruck Habtemariam

UCSF – Benioff Children’s Hospital, Oakland

747 52nd Street
Oakland, CA 94609

Mary Peoples

UCI Health

101 The City Drive South
Orange, CA 92868

Nancy Ortega

714-509-6558

CHOC – Children’s Hospital of Orange County

1201 West La Veta Avenue
Orange, CA 92868

Eliud Espinal

Children's National

111 Michigan Ave NW
Washington, DC, MD 20010

Regine Hyppolite

Cornell - Weill Cornell Medicine/New York-Presbyterian

525 East 68th Street
Payson-695
New York, NY 10065

Kathryn Lang

Duke University

315 Trent Drive
Room 270
Durham, NC 27710

Lindsey Ford

ECU – East Carolina University - Brody School of Medicine

600 Moye Boulevard
Suite 3E57 - Pediatrics
Greenville, NC 27834

Amy Hawkins

CCHMC – Cincinnati Children’s Hospital Medical Center

3333 Burnet Avenue
Cincinnati, OH 45229

Adriane Hausfeld

Children’s Hospital of Philadelphia

3401 Civic Center Boulevard
Philadelphia, PA 19104

Jaladhikumar Patel

About thalassemia¹

Thalassemia occurs when the body does not make enough hemoglobin (a protein that carries oxygen in the blood), which is an important part of red blood cells (RBCs). A lack of hemoglobin in the body causes a decreased number of RBCs, which don’t function as they should. RBCs carry oxygen to all cells in the body, and cells use oxygen to function. So, when there are not as many healthy RBCs, there isn’t enough oxygen, which can often cause fatigue, weakness, shortness of breath, and anemia.

About sickle cell disease (SCD)²

SCD causes red blood cells (RBCs) to form into a hard sickle or “C” shape due to an abnormal hemoglobin type. This abnormality causes sickled RBCs to not live as long as normal RBCs. Sickled RBCs block blood flow and the cells die early, leaving a shortage of healthy RBCs. This often causes pain, fatigue, swelling, anemia, and jaundice, among other symptoms.

What you should know about clinical research studies

All new treatment options must go through a series of clinical research studies before being approved for use by the US Food and Drug Administration (FDA).

Participation in any clinical research study is completely voluntary and participants may withdraw at any time for any reason.
Studies are conducted by doctors who are responsible for the study participants’ care.
Clinical research studies must first be approved by an institutional review board (IRB) or ethics committee (EC) before individuals are allowed to participate.

Every study participant is monitored during and sometimes after the study.
Clinical research studies provide information needed to develop new treatments for diseases.
All new treatment options must be evaluated in clinical research studies before being available for use by patients outside a clinical research study.

Clinical research studies are conducted in phases.

Phase 1

The investigational medication is usually tested in a small group of healthy people to evaluate its safety, its side effects, and the correct dose. Some Phase 1 studies also include people with the disease that is being studied.

Phase 2

The investigational medication is tested in a larger group of people with the disease that is being studied to evaluate its effectiveness and to continue to assess its safety and side effects. The Gladiolus Study is a Phase 2 clinical research study.

Phase 3

The investigational medication is tested in larger groups of people with the disease being studied to assess its effectiveness compared to approved treatment options (or a placebo), and to further evaluate its safety and side effects.

Participation in any clinical research study is completely voluntary, and participants may choose to leave the study at any time for any reason. If you would like to leave the study, you should discuss this with the investigator, who will give you information about how to do this safely.

Before you can take part in a research study, the investigator will explain the informed consent form to you, along with any potential risks and benefits, and your rights as a participant. The decision to participate is yours and entirely voluntary. If you decide to take part and sign the informed consent form, the investigator will conduct screening assessments to determine whether you qualify to enroll. After all necessary tests and assessments have been completed, and if you are eligible to participate, you may enter the study.

The sponsor of this study is Forma Therapeutics, Inc.

Minority under-representation in research continues to be a major barrier to the availability of treatment options for rare diseases like thalassemia and sickle cell disease (SCD). It goes without saying that minorities are historically under-represented and under-studied in clinical research studies, and past experiences have worsened feelings of distrust toward study participation. To address distrust toward clinical research within the thalassemia and SCD communities, Forma Therapeutics has made a concerted effort to be transparent about our drug development process by engaging in the following actions:

Active listening and acknowledgement of the expressed questions, concerns, and fears regarding clinical research and participation.
Understanding and empowerment of the communities to identify their unmet treatment needs and access to research opportunities.

Collaboration with the communities on best practices to establish relationships and trust between the community and researchers.
Ongoing education regarding clinical research studies and safety.

Find a participating site

References

Access date: April 28, 2021

A life beyond thalassemia or sickle cell disease is worth fighting for.

We are currently enrolling participants who meet the following criteria:*

About the Gladiolus Study