Current Studies

Certain studies conducted by Engage Health are closed, and recruitment is targeted to certain individuals. Other studies are open, and allow recruitment from a number of sources.

The following are current market research projects that are open to participation.

Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families

While there are a number of publications/guidance/consensus statements related to the diagnosis and management of Duchenne Muscular Dystrophy (DMD) from the point of view of the health-care providers, there is a need for more research to define the key aspects of life with DMD from the perspective of the patient and caregivers, using the patients’ own voice and description of life impact.

To that end, please participate in Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families. The objectives of this study include;

  • To improve the understanding of the treatment goals that a DMD patient or caregiver may be most interested in based on the severity of their disease
  • To collect evidence, in the patients’ own terms, of the functional burden and the self-­ identified treatment goals of Duchenne patients and caregivers from their perspective and use this evidence to help identify things important to Duchenne patients to measure in clinical trials.
  • To collect information that will be used to inform the selection of key concepts of interest and development of future clinical outcome measures, including observer reported outcomes/patient reported outcomes

The study will be conducted in individuals 11 years or older living with DMD as well as caregivers for boys and men of all ages. To qualify you must meet the following criteria;

  • Participant must have a confirmed diagnosis of DMD

OR

  • The parent/legal guardian of a child with a confirmed diagnosis of DMD. Proof of diagnosis will be required
  • Resident of the U.S.
  • Able to read, write and communicate in English
  • Able to grant informed consent
  • Participant must be 11 years or older. Participants over the age of 18 years of age will provide informed consent. Parents/legal guardian of participants under the age of 18 will provide informed consent for their child. Participants under the age of 18 will provide informed assent.
  • Willing to participate in a one -hour telephone interview between now and January, 2019
  • Ability to view or receive a document from the interviewer before or during the interview (web browser, ability to receive a text, fax or document by mail)

During the interview participants will be asked key aspects of life with DMD, including things that each patient/parent wishes that they or their child could do, but cannot do because of

DMD. Participants will also be asked about how these issues impact their life or the life of their child. Participants will be paid an honorarium of $100 (US dollars) at the completion of the interview.

To participate, please visit Your Voice: lmpact of DMD RSVP. If you are unable to reach the RSVP from this link, please copy and paste the following URL into your browser bar; http://www.engagehealth.com/survey/TakeSurvey.aspx?SurveyID=984L694

Assessing the Need for Higher Education in Patient Advocacy (US and other non-EU countries)

As the biotech/pharma stage is pushed toward a patient-focused path, the field of patient advocacy has grown.  Engage Health is helping Professional Patient Advocates in the Life Science (PPALS), a non-profit professional patient advocacy organization conduct a feasibility study to determine the need for higher education in this field. The study allows for three major groups to voice their views on this important idea;

  • Patients and families
  • HR/hiring professionals
  • Patient advocates (both those who are unaware of PPALS and those who have participated in the PPALS’s Patient Advocacy Certificate Training)

Please participate in this study to help determine the future of patient advocacy!  Please participate between now and July 20th.  The survey will take approximately 10 minutes to complete and can be found at this link or by accessing the URL below:

http://www.engagehealth.com/survey/TakeSurvey.aspx?SurveyID=9l4M3m3.

Cystinosis Patients—Free Genetic Testing for Nonsense Mutations

McGill University invites cystinosis patients to join their study concerning a specific type of genetic alteration called a “Nonsense Mutation”.  Although cystinosis is caused by many different disruptions of the CTNS gene, they are particularly interested in “nonsense mutations” which trick the cell into stopping production of Cystinosin protein. Several pharmaceutical companies are working hard to develop medications related to a well-known antibiotic (gentamicin) that permits the cell to disregard nonsense mutations. To participate in the free genetic testing study and learn if you have this nonsense mutation, contact the study coordinator, Murielle Akpa, PhD  either by phone or email; phone: (514) 412-4400 ext. 22953 email: murielle.akpa@muhc.mcgill.ca.

Our client is interested in learning more from physicians about their cystinosis patients and their interest in clinical trials. To denote your interest and share your experiences, please click this link.

Genetic Testing in Glucose Type-1 Deficiency Syndrome (Glut1 DS) – For Physicians

Our client is sponsoring a program for physicians offering a no-cost genetic testing for their patients with symptoms of Glut1 DS or a family history of the disease. The genetic test panel includes 3 key mutations which may account for up to 47% of paroxysmal dyskinesia cases, and takes between 2-4 weeks. If you are a physician who would like to participate in this free testing program, please click on this link to complete a brief survey and request the test kits.

Physician Survey Regarding the Diagnosis and Treatment of Galactosialidosis and Sialidosis Type I 

Our client would like to learn more about patients with galactosialidosis and sialidosis type I, including how they are diagnosed and treated. If you are a physician who sees these patients and would like to participate in this brief survey please click here: survey. Your participation, which does not involve sharing confidential information, will help our client design future clinical trials.