The Clinical Trial
We Got Here.
Now We Need to Get Our Kids Treated.
After years of work, the clinical trial is ready to begin. Now comes the funding part.
For Barbara, Makeda’s mom, the journey began with a diagnosis no parent wants to hear.
AGU is a rare genetic disease that progressively takes things away from children—development, abilities, independence and eventually, their future.
But Barbara and our other AGU families didn’t give up.
They organized. They raised money. They found researchers. They built a global community. They kept pushing forward year after year.
Rare Trait Hope set out to help make a clinical trial possible. Today, it's a reality.
It’s the moment our global families have been working toward for years.
We Have Done the Hard Part.
Now We Need the Money for the Next Part.
Rare Trait Hope and our global AGU families have already raised more than US $4 million to develop, manufacture and safety-test the medicine for our kids. Almost US$1 million was raised here in Canada. Here’s where the funding has taken us so far in our journey:
The U.S. Food and Drug Administration (FDA) has authorized the clinical trial to proceed under an Investigational New Drug application. The Institutional Review Board at the U.S. university where participants will be treated has also approved the study.
Now we need to fund it and help families with the costs of participating.
The estimated clinical cost: Approximately US$170,000 per child.
Anticipated Costs During the Clinical Trial
Including:
- Travel
- Lodging
- Local transportation
- Meals
- Time away from work
- Childcare and support for other family members
- Other costs associated with staying near the clinical site for several months
What Do We Hope The Clinical Trial Achieves for Our Kids?
- Pre-clinical studies show it’s possible to reduce the toxic materials associated with AGU.
- The investigational medicine is designed to be given as a one-time treatment.
- We hope the treatment will slow or stop the progression of AGU and give treated children the opportunity to maintain or improve their development and abilities. The clinical trial will help us understand these benefits.
- The clinical trial is currently designed to enroll nine participants.
Think About What This Funding Means
A treatment has been developed. Millions of dollars have been invested.
The regulatory approvals have been obtained. The clinical team is ready.
Let's Get Our AGU Kids to
The Clinical Trial!
Your gift will help Rare Trait Hope fund the trial and support the families facing the costs of getting their children to the clinical site and participating in the study.
YOU can be part of a remarkable journey that families began years ago—and help get them across the finish line.
Additional Payment Options
You can also e-transfer funds directly to Rare Trait Hope Society by sending your donation to info@raretraithope.ca. Please include your full name, email address and address if you require a tax receipt. We cannot identify your email address based on your e‑transfer and will be unable to follow up with a tax receipt without this information.
Cheques
Mail to:
Rare Trait Hope Society
2768 W Broadway #573
Vancouver, BC
V6K 4P4
Donation of Securities
Tax Receipts
Rare Trait Hope Society is a registered Canadian non‑profit organization and registered charity (CRA#71150 6071 RR0001).
Tax receipts are issued through our donation management system (Keela). If you donate online by credit card, your tax receipt will be emailed to you automatically. If you donate by cheque or e-transfer, your receipt will be emailed once your donation has been processed.
Monthly donors will receive one consolidated tax receipt at the beginning of the following calendar year.
US Donors
If you are in the United States, Rare Trait Hope Fund is a 501(c)(3) registered charity and you can donate at Rare Trait Hope Fund.