Evaluating Sex and Gender Considerations for Pediatric Rare Condition Clinical Trials: A RareKids-CAN Sex and Gender Sub-Platform Needs Assessment

Evaluating Sex and Gender Considerations for Pediatric Rare Condition Clinical Trials: A RareKids-CAN Sex and Gender Sub-Platform Needs Assessment

This study, as part of the Sex and Gender Sub-Platform of the RareKids-CAN network, aims to learn about the experiences and needs of children with rare conditions who have participated in pediatric rare condition clinical trials, their caregivers, and rare condition researchers when it comes to sex, gender, and Sex and Gender-Based Analysis Plus (SGBA+). This study will help develop more specific support for the design of pediatric rare condition clinical trials that enhance equity, diversity and inclusion related to sex and gender in Canada.

We are recruiting participants to complete a survey and/or an interview. Participants who complete the survey will be compensated.

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The Canadian Skin Patient Alliance (CSPA) is conducting a survey on the impact of dermatological conditions on mental health. 

If you have a dermatological condition or are a caregiver to someone who does, we want to hear from you! Please make your voice heard on this very important topic and be entered into a draw for one of four $50 gift cards for a retailer of your choice. 

Please fill out this 5-10 minute survey. 

English: https://forms.office.com/r/v9brxLUs0S

L'Alliance canadienne des patients en dermatologie (l'ACPD) mène actuellement une sondage sur l'impact des maladies dermatologiques sur la santé mentale.
Si vous souffrez d'une maladie dermatologique ou si vous vous occupez d'une personne qui en souffre, nous voulons connaître votre avis !
Faites entendre votre voix sur ce sujet très important et participez à un tirage au sort pour gagner l'une des quatre cartes-cadeaux d'une valeur de 50 $ à utiliser chez le détaillant de votre choix.
Veuillez remplir ce questionnaire qui ne vous prendra que 5 à 10 minutes.

French: https://forms.office.com/r/5RhydfYpJM

Participant recruitment is now underway for the APOLLO Study, a clinical trial evaluating Bitopertin, an investigational oral medication for individuals with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). The study aims to assess whether Bitopertin can reduce protoporphyrin IX levels and potentially lessen symptoms from sun exposure.

Fast Facts:

• Study Type: Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled

• Who Can Participate? Individuals age 12+ with a confirmed diagnosis of EPP or XLP

• Study Medication: Investigational oral therapy (Bitopertin)

• Study Sites: Alberta

• Travel & related expenses covered by the study sponsor

• Includes an optional open-label extension

If you are interested, please complete the confidential interest form below and be connected to a site near you. Filling out the form does not obligate you to participate.

For more information, contact: president@porphyriacanada.ca.

COMPLETE INTEREST FORM

(Posting on behalf of Inform Rare)

ARE YOU A YOUTH WITH A RARE DISEASE, OR A CAREGIVER TO A CHILD OR YOUTH WITH A RARE DISEASE?

We are a team of researchers from the University of Ottawa working to better understand the experiences of children, youth, and caregivers with clinical trials.

Clinical trials are research studies that evaluate treatments to assess whether they work and are safe We aim to explore how participating in clinical trials may affect the lives of families managing rare diseases.

Eligibility

• Youth aged 14-20 years who live in Canada and have a rare disease diagnosis

• Caregivers residing in Canada (for example, parents) to children aged 0-18 years with rare diseases

• Have participated in or been invited to participate in a clinical trial in the past

• Be able to join an English-speaking focus group

MORE INFORMATION

 Portal Therapeutics is a clinical stage biotechnology company based in California that is developing a novel investigational therapy for people living with EPP and XLP. Portal recently started its Phase 2A clinical trial, GATEWAY, which is currently enrolling adults with EPP (NCT06971900).

Portal has developed PORT-77, which takes a new approach to helping people with EPP and XLP. In EPP or XLP, a molecule that is a part of heme biosynthesis, called protoporphyrin IX (PPIX), builds up in the red blood cells. A protein in red blood cells (called ABCG2) acts like an open gate, moving this PPIX out of red blood cells and into the bloodstream.

When PPIX travels to the skin from the bloodstream, light, especially sunlight, can cause painful reactions. PPIX movement and build-up in the liver and biliary tract can also sometimes create liver complications. PORT-77 is designed to "close" the ABCG2 gate. By blocking the ABCG2 protein, more PPIX stays inside the red blood cells, instead of moving into the bloodstream where it can cause subsequent problems in the skin and liver.

Contact

If you are interested in learning more about the GATEWAY study, please contact the April Goldman, april.goldman@celerion.com, +1 602-437-0097 Ext 66116.

THIS STUDY IS NO LONGER ACCEPTING PARTICIPANTS

An interview study is underway to understand patient experiences with acute hepatic porphyria.

Eligibility

We are looking for individuals ages 15 and over in the United States or Canada who have acute hepatic porphyria (AHP).

Study Structure

As part of this study, you will complete a one-on-one approximately 90-minute telephone or video conference interview scheduled at a time that works well for you, and you will later fill out three online questionnaires that take approximately 20 minutes to complete.

Compensation

You will receive $240.00 USD or Canadian equivalent as compensation for your time to participate.

Information and Disclaimer

This is not a medication-related study. No medication will be given or tested during this study.

The information you share may contribute to knowledge about acute hepatic porphyria (AHP).

Your name and contact information will remain only with the research team and will notbe shared.

Please note, you/your child may not be eligible for the study if you/your child: have taken GIVLAARI (givosiran) or hemin prophylaxis within the past six months; reside in Alberta, Saskatchewan or Newfoundland and Labrador; are between the ages of 15-17 and reside in Quebec; or have certain medical conditions.

Participate

For more information or if you wish to participate, please reference the study code: AX10047A Acute Hepatic Porphyria Study and contact us:

• By email at ResearchStudy@adelphivalues.com to learn more about the study, ask any questions, or schedule a time to be screened.

• By telephone at (617) 720-0001. Please leave a voicemail with your name, telephone number where you can be reached, and the study code (AX10047A), and the recruitment coordinator will follow up to provide more information answer questions or schedule a screening.