Pediatric CLINICAL TRIAL Link

[Linda Burke]

Some news has been publically posted regarding the clinical trial of Soliris (eculizumab) for pediatric patients with atypical HUS. As an independent and unaffiliated organization devoted to education, support, and outreach for aHUS patients and families, this interactive website of the Foundation for Children with Atypical HUS recognizes that some in our community are highly interested in this trial.

On August 31, the National Institutes of Health posted an overview of the trial on www.clinicaltrials.gov. Here is the link: http://www.clinicaltrials.gov/ct2/results?term=NCT01193348. You will notice that the trial is not yet enrolling patients, but some basics of the study do appear and I'd encourage you to read the full text. Children from 6 months to 18 years of age are eligible for this clinical trial. Pediatric aHUS patients must meet four requirements for inclusion in the clinical trial while three factors would exclude the pediatric aHUS patient from trial participation.

Inclusion:

1. Patient's parent/legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].

2. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease.

3. Patients from 6 months up to 18 years of age and body weight ≥ 5 kg.

4. Patients exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine.

Exclusion:

1. Plasma therapy for > 5 weeks prior to enrollment.

2. Chronic dialysis.

3. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.

Since only your child's doctor or specialist team can completely and appropriately assess your child's status and make conclusive decisions regarding Soliris and this clinical trial, we urge you to consult your health provider regarding your child's specific situation. Physicians are encouraged to contact Alexion Pharmaceuticals at 1.888.SOLIRIS (1.888.765.4747) for specific information about the trials locationed in North America and Europe, but please note that additional information will be publically released at www.clinicaltrials.gov (clinical trial identifier: NCT01193348) as details become public. Please monitor that site for enrollment dates as The Foundation for Children with Atypical HUS will not be privy to any confidential information or any pre-release of trial information.

[Paula Blanchard Lamigo]

Had our appointment on Friday, Dr. Kher is going to start trying to contact people regarding the trial & he told me he would let me know what he finds out in about a week. Lisa, if he finds out anything that might be helpful, I'll let you know.

[Paula Blanchard Lamigo]

Our appointment is tomorrow, I'll let you know if Dr. Kher has anything new to add.

[lisa ann peterson]

chloe is in a contstant state of hemolysis cause her disease is 13% acitive at all times and we have to give her weekly shots of epo to sustain normal blood levels.. dr. h said that she would have to relapse to use the solaris. paula if you find out let me know

[Paula Blanchard Lamigo]

Okay so the way I read #4 of the inclusions is that the patient must be in a state of hemolysis (well that is one of the factors we have with Devin) He has been in a continuous state of low-level hemolysis, even when he had episodes & was treated with plasma-pheresis & then ffp, his body remains in a constant state of hemolysis. I'm not sure on the elevated serum creatinine though. I'll have to confirm this with Dr. Kher at our next appt. on Sept. 24th. I am reading that several people are saying that their children must relapse before they can start the trial, but what if their children are in a constant state of hemolysis? Or, does Alexion want a "full-blown" relapse? I have not heard this from Dr. Kher on the few occasions he was able to speak with someone from Alexion.

[Zofia]

Thanks Cheryl.
But does it means that if We stop giving Ania plasma , and She will relapse before the trial begin, then no mater what we do (give Soliris, or FFP), She will be excluded?

[Jessica Olivia Frysz]

very good info on the trial, but it doesn't help me, because I'm no longer a pediatric patient and I don't have a transplant yet, but it has been discussed at my eval that this drug would be used.

[Cheryl Biermann]

The plasma condition means that if your child has had plasma in the 5 weeks priort to enrollment of the study they will not be able to participate. This will allow everyone in the study to have the same starting point and will not cloud the study with questions such as, "Was it really the Eculizumab that helped or was it the FFP?"

According to our nephrologists there have been children who have been successfully transplanted with a new kidney and Eculizumab, so no, Jose wouldn't be the first in the world, but would you really want him to be? I for one am glad that others have taken this step before us, I have a tremendous respect and gratitude for everyone who has been trail-blazing the way, especially for the people who have had so much damage due t to the long-term effects of living with the disease and then on chronic dialysis! We are truly living in a time where miracles are happening, due to the dedication of the medical field and the medical researchers as well as the countless hours of volunteers have contributed in order to raise money to pay for the research!

[Zofia]

"Plasma therapy for > 5 weeks prior to enrollment"
Does this mean that children treated witch FFP in the past (for years in ours case) won't be able to use it?
Or just the last FFP should be given >5 weeks prior to enrollment?

[Kerri Grey]

Thanks Linda for keeping us all informed!!! This puts Ash out of being able to use it in the trials as he is already using it... oh well

[maria vicenta carratala rios]

Hi Linda.
We are waiting yet. The next week Jose he will be a month who is waiting a new kidney with Eculizumab.will Be he the first child in the world?
Thanks for the update