Considering a Clinical Trial for a Child: A Caregiver's View

Narrator (00:02) 
A child's diagnosis of cancer or another serious disease is difficult. Families, guardians, and loved ones experience a range of emotions and often need support related to their child's diagnosis and treatment. St. Jude Children's Research Hospital brings you Caregivers SHARE, a St. Jude podcast. SHARE stands for support, honor, advise, reflect, encourage. In this series, you'll hear stories and insights directly from the experiences of St. Jude families and care providers. 

Clay Culp (00:37) 
Hi, I'm Clay Culp, Social Work Manager at St. Jude Children's Research Hospital. Welcome to the second episode of a two-part series on clinical trials. For more basic information on clinical trials, listen to our previous episode, What is a Clinical Trial?

Today, we'll focus on the social, emotional, and logistical challenges that families face when considering enrolling on a clinical trial. Carina, could you introduce yourself and tell us a little bit about your journey? 

Carina Richardson (01:05) 
Yes, I would love to. Thank you, Clay. My son Nolan is the youngest of three children. He has an older brother and an older sister. And when Nolan was eight years old, he began experiencing recurrent vomiting and a handful of headaches, which were severe at times. This went on over the course of three weeks. And when we still didn't have any answers, my husband requested an MRI, mainly for reassurance.  

But to our surprise, we learned that Nolan had a golf ball-sized mass in the lower portion of his brain, and that was explaining his symptoms. So we quickly connected with a pediatric neurosurgeon in our state. Nolan had surgery, and once the pathology came back, we learned he had a medulloblastoma, which is a fast-growing and aggressive form of brain cancer. And suddenly, we were faced with decisions about his treatment.  

We were devastated. Nolan had gone from being healthy, funny, athletic, to suddenly having a very serious illness. And it not only affected Nolan, it affected the whole family as we were trying to make decisions about enrolling him in a medical trial and what that would look like for our family.  

I will say that today, Nolan is 18, and he is cancer-free, and he is about to graduate high school this spring. Yes. 

Clay (02:52) 
Exciting. Yeah. Well, congrats to Nolan. Pre-congrats on graduating high school. And thank you for being here to share your story and a little bit of Nolan's story. So, Dr. Seth Karol, we're glad to have you here as well. Can you introduce yourself and tell us a little bit about why it's so important that we talk about clinical trials? 

Seth Karol (03:14) 
Yeah, thank you, Clay. And thank you for the chance to be here and join Carina. So my name is Seth Karol, and I'm one of the leukemia lymphoma oncologists. That means I'm a doctor who also takes care of patients with children with leukemia, a form of blood cancer and lymphoma, as well as writes clinical trials and runs those trials so that we can try to improve treatments and improve outcomes for children with those types of cancer. And I think that clinical trials are really foundational and fundamental to how we make treatment better for children with any serious illness. And so very glad to be here and to join the conversation. 

Clay (03:49) 
All right, so let's dive right in. Carina. When your care provider first mentioned the word clinical trial, what went through your mind? 

Carina (03:60) 
Well, we were still reeling from the diagnosis following surgery and trying to wrap our heads around what this would mean for Nolan and our family. I will say my husband is a physician and I have worked as a genetic counselor in the past. So medical terminology was familiar, but we were not experts by any means in pediatric oncology. So it was a lot to take in. We wanted to know first, how is this clinical trial different than the standard treatment? And what benefit could it give our son for his survival?  

At the end of the day, that was one of the most important things we were considering is, is this the right decision for Nolan? And a clinical trial seemed to be another option for treatment that was being offered. We were having to choose. 

Clay (05:10) 
Right. So all the while, these ideas of everything you've heard in your life, maybe about clinical trials and what is that going to mean for Nolan, all that's going through your head. But then you're faced with the reality of a clinical trial. So I'm wondering, what was it actually like to have Nolan on a clinical trial? 

Carina (05:31) 
So one of the things we learned early on with this clinical trial is that we would have to move away from home for a time to have the ability to complete the treatment. So at first, we really did think long and hard, is this something our family could do? We were a family of five. We had two children. In school, back in our home state. And we wanted to try to not just keep our family together, but also make life as normal as possible. And this was not accomplishing either of those two things. So that was something we had to think long and hard about. 

Clay (06:21) 
Well, Dr. Karol, Seth, you've been in so many of these conversations with families hearing maybe their initial reaction to the word clinical trial, but then to also see how maybe that perspective shifts a little bit as they hear a little bit more. Can you share some about your impression of what this is like for caregivers, how those conversations go when it's actual reality of a clinical trial? 

Seth (06:48) 
Yeah, I think Carina really described beautifully some of the challenges that families face as they think through treatment for any serious illness for their child. And certainly, thinking through the options of a trial are part of that for many families and children. I think most of the conversations I have with families focus on sort of two aspects of whether to be on a trial or not. And so one of those is the medical piece of it, which is to say, how is this medicine or how is this treatment different if I do the clinical trial than if I do the standard treatment, the standard protocol?  

And then I think the other piece is connected to the other part that you talked on, which is to say, what does being on the trial mean in terms of what I have to do? Do I have to be away from home for a period of time to get that treatment somewhere else? Do I have to come to the hospital to get a test done that was only being done as part of the trial?  

And so when we think through or talk through trial options with families, I think that first piece, how is the treatment different? What might that mean for my child is often the biggest piece of it. But we try to make sure we touch on that other piece as well, because that second half is really a big factor in terms of whether families say yes now, but even more importantly, how realistic it is that we're going to make it through this whole plan that we developed together. Because I want to make sure that we are thoughtful about the fact that that child's care is the center of everything we do at the hospital.  

And yet, there is the world outside the hospital that is such a big part of families' lives too, and we can't separate them and need to make sure that we take perfect care of the kid as best we can, and yet also be aware that coming to clinic to get checked on extra times just so we can feel extra safe for them also means the family is traveling, and that there's all these little pushes and pulls. And families willingly do that all the time for children. That's part of being a parent. But we try to minimize how challenging that is to be on a trial compared to our usual care. 

Carina (08:51) 
And I will add that part of what helped us decide to enroll in a clinical trial is the support of our family and community at home, because I knew I was going to have to be separated from the rest of the family, and I wanted to make sure the rest of my family was being taken care of as well while we were here treating Nolan's condition. 

Clay (09:19) 
Yeah, it's so important. And I want to hear a little bit more about how you personally came to that decision. But before we get to the logistics of that, I'm curious about just the emotional toll. I know in my role as a social worker, I hear a lot from parents, caregivers about just how heavy it is to make all these critical decisions that could affect the child for the rest of their life. Can you share about that emotional aspect of decision-making? 

Carina (09:52)
Yes. And you are spot on with the word heavy. It weighs on. Your mind tremendously to not only know that all of a sudden your child's life has changed because of an illness, but also that you are responsible for the decision that comes next.  

So that is a lot to think about. In the early days, there was little sleep, lots of tears, just emotions all over the place. One moment we would be so scared, another hopeful, well, maybe this is not as bad as we think. And then when those pathology results came back, that was a hard reality to sit down and face.  

So, all of those things together do impact the decision-making process. And most families need guidance in that—we don't know the information that we don't know about the ins and outs of standard treatment or a clinical trial. We never expected to make a decision like this for our son. 

Clay (11:15) 
Yeah. So take me into that decision-making process a little bit. You're feeling all the heaviness, the emotions, the lack of sleep, and you're trying to make this decision. So what were some of the things, some of the questions even that were most important to you, the people that you did lean on to try to make that decision? 

Carina (11:33) 
Yes. We met with the oncologists at the hospital where the surgery was completed, and we were given the options of standard treatment or potentially a clinical trial. The standard treatment differed in various ways from the clinical trial.  

And so, those were the questions we asked. How is it different? How much information do you have about this in terms of cure rates, complications, risks, things like that we wanted to know. Then we also learned during that conversation that the clinical trial was not available at this institution, so we would have to travel out of state if we wanted to participate. So that was overwhelming because all of a sudden, we have a very sick child who's not feeling well, and just the thought of traveling was really difficult at that time.  

We fortuitously were connected, just the universe set this up, that we were able to speak to another family initially who was on this clinical trial we were considering. And they were diagnosed a few months before, so they were ahead. So, we connected with this family and spoke about the day-to-day. What does this look like for you? They were also out of state traveling to this institution for the trial. And they had other children at home. So, there were things we could relate to here.  

And that really helped not necessarily guide our decision, but tell us that, okay, this may be hard, but it's doable. So, that gave us some hope. And from that conversation, we did end up speaking to one of the doctors in charge of the trial. He ended up calling us at home on a weekend and explaining the trial, what it would offer our child specifically, how much time it would involve, and what next steps might be if we were seriously considering the trial. So that was helpful to have that medical piece in conjunction with the family piece.  

I can say that in addition, we did consult other institutions about treatment just to ask, hey, what do you think? Should we stick with the standard treatment? Should we do the trial? And ultimately, when we put all of that information together and sat down in person to have that consent discussion, we did decide to enroll Nolan. 

Clay (14:49) 
It sounds like you really pulled in a lot of different sources of information, but ultimately went with the decision that was right for you. And I think that's important to emphasize for our listeners that there's no one right decision. And I'm curious, Seth, if you can speak to from a physician's perspective, when you're laying out options and families may feel like, wait, I'm supposed to pick the right option. What does it actually mean? If you're laying out options, are they all good options? Are some of them good, some of them not good? Can you address that? 

Seth (15:27)
Yeah, I think it's one of the most important things that I do when I'm having these conversations is trying to figure out how to present the information in a way that gives families the knowledge to feel comfortable with their decision. It's definitely my job to not offer something that I don't think is reasonable. So I will never sit down in a conversation and offer to Carina or another family something that I wouldn't want someone to at least offer to me if we were in different shoes.  

And so that's where my knowledge and my experience comes into play, is knowing what those options are and what things are reasonable to offer. And that doesn't mean that I don't think one might be a little better than another, but it means that if you chose the thing that I think isn't number one, that it's a 1B, it's really close in a way that I think it's still very appropriate.  

And then as we lay out those differences between either multiple trials or between a standard treatment and any trial, I try to emphasize those differences and then really have a conversation with the family and learn about their kid and their family and their preferences. And that really helps me understand, you know, which, if I've got multiple reasonable choices, might make sense. And I'll, we'll talk through those aspects of things together.  

And it could be that they've talked to other doctors or other hospitals and have heard other opinions. And then I really value hearing what they're thinking about all those opinions, because that makes a big difference in terms of what they're thinking and the conversations we have. 

Carina, you said it perfectly, right? These are heavy decisions. And ultimately, I can make a recommendation to a family. It's going to be their ink on the paper. And even more importantly, it's their child. And so, they're going to go home to bed at some point and lay their head on the pillow, and I want them to be able to sleep with the decision that they've made. It's my job to help them feel comfortable making a choice in a terrible circumstance where their child's faced with a life-threatening illness. 

Carina (17:25)
And I will add that for us, getting all this information and being able to ask questions about risks, benefits, differences, timelines. All of these things, cure rates potentially. This was very helpful. And at the end of the day, as information seekers, my husband and I felt comfortable making the decision.  

Now, do we wish we never were in that situation? Of course. But we felt we had as much information as we could possibly have at that time to make the best decision for our child. And it really did give me, at that time, just a sense of peace. Like, okay, now we've made the decision. Let's get going on this treatment. 

Seth (18:21) 
Yeah, and I see that in a lot of other families as well, where it can be very, very difficult to get to that point where we're comfortable making a decision because these are heavy choices. And we're talking about a serious illness and often treatments that have potentially serious side effects.  

And at the same time, we know that we need to do some treatment because that's the way we make this better ultimately. And so getting to that point where we can be comfortable getting there and then sort of moving forward is often a weight off of family shoulders.  

And then the other thing that I think we allow families to get to that point and to feel comfortable with that is knowing that we can always have another conversation in the future, that the decision we make today does not have to be the decision that we take forward for the rest of treatment or the rest of our time together but If there are things that change, and that can mean a night's sleep, and I feel differently when I wake up in the morning, or that can be something that happens either to the child from a side effect or something going really well, or from something out in the world where I learned something from other people getting that medicine. We may have to sit down and have a conversation and make that change. 

Clay (19:28) 
Right. And it's not just one conversation. It's really an ongoing dialogue with this holistic perspective of the needs of the family, the patient, maybe siblings, so many different factors that come into play.  

That makes me think about the logistics of this. You've made the decision and now you're thinking about, okay, how is this going to change our daily life? How do we meet the requirements to be on the trial? What was that like for you and how hard was it to do everything that you needed to do? 

Carina (20:04) 
It was overwhelming. When we sat down to discuss the trial in detail and sign the consent forms, we were given a protocol that spanned 72 months. My child was eight, and in my head, I was trying to do the math like, how old, he's going to be a teenager by the time this is finished, like that is a long period of commitment.

With that said, the trial looked different at different stages. So the first stage was active treatment. That was the stage where we would be required to move and to be on site or as close to on site as possible. And that was difficult because we were separated from the rest of the family for six weeks for radiation therapy. And then we came home for a bit. And then we came back to start the chemotherapy treatments.  

The chemotherapy treatments consisted of four intense cycles of chemotherapy for Nolan. It can look different for other kids, but for Nolan, it was four, which would involve hospitalization to administer these very intense medications, as well as labs weekly, as well as clinic visits weekly with our medical team. And in addition, there were requirements for heart function studies, lung function studies, and more.  

So, there were a lot of moving pieces with the trial in terms of scheduling these appointments and making that happen over the course of the four months. This is in addition to the, I want to almost say, normal things that happen on any standard treatment, like you might need to go in for blood transfusions, or there might be some other health concern that arises during that time. So, these were all in addition to that. It was quite a commitment. 

Clay (22:35) 
Well, yeah, as I'm sitting here listening to you talk about that, I'm thinking, wow, that does sound somewhat overwhelming. And I've heard a lot of parents, caregivers share about that.  

I wonder if this is going to resonate with you, but some talk about this balance of, yes, we need to plan and think about these things and also give up some sense of control and planning for everything and kind of taking things day by day. Whether you're on a clinical trial or not, that that might be something that resonates. 

Carina (23:08) 
Absolutely. I am a planner and this was not in my plan at any point of my life to have a child with a serious illness. And most of all, I didn't want it for him. I was so worried about how he was going to come through this. I have learned that sometimes the best laid plans, what's that expression? “The best laid plans?” 

Clay (23:35) 
“Of Mice and Men.” 

Carina (23:35) 
Yes, yes. So it has taught me sometimes we need to just let go of some of the planning and just take things day by day, because each day can look quite different when you have a child on a clinical trial. 

Clay (23:54) 
Right. It's so true. And it's a “both and”. There's this day-by-day aspect and this other piece. People do need to prepare and plan to take part in a clinical trial. So I want to bring you in, Seth, to share a little bit about maybe some thoughts, some considerations that you encourage caregivers to think about? 

Seth (24:15
Yeah, as we think about what it means to be on a clinical trial or sort of the requirements that are there, there's sort of two layers. The first is before you get on the clinical trial. And so clinical trials are treatments or they're protocols that do similar or the same things for everyone. And so we want to make sure that the patients meet their criteria to be on that trial so that we're treating, for example, one type of cancer, all the same. And I wouldn't want to treat a brain tumor with blood cancer medicines.  

And at the same time, that helps us increase the safety of the trial, because we know that the child meets some criteria, their body is healthy enough that we think that trial can be done as safely as possible.  

And then Carina just did a beautiful job of talking through all the challenges that happen when you're on a trial. So there are things that we do for all children that we take care of. We check up on them in the office. We may hospitalize them when they need it. We give them medicines. We do blood work or x-rays or things like that to understand how they're doing.  

But then we may do extra of those for the clinical trials so that we better understand how this treatment is different than the standard treatment. And we try to compact that as much as possible so that if I'm going to see you in clinic for a visit, that we’ll also have you see the physical therapist to do their evaluation on the same day. Or if I'm drawing blood work to make sure your child does or doesn't need a transfusion, that I draw the little bit of research blood at the same time so that we're not poking them more than once or using their line more than once for that sample.  

Those little things can add up over time. And so instead of being able to leave at noon, you may have an appointment at one o'clock that's for the research, and that could extend your day, or you may have to come back some other day for one of those pieces. And so those are the sort of just living through life as a patient getting therapy for a serious illness, but also like those little changes to that that can occur as a result of being on a trial. 

Carina (26:10) 
And I will expand a little bit on how a trial can change over time. So I talked a little bit about active treatment when we were away from home, but then at the end of the treatment, you do get to go back home.  

A clinical trial may have families come back at different points. For us, it was every three months initially, and then every six months for a time. And then it changed to every year until we completed those 72 months of the protocol. So we did have to plan, okay, when are we going back to do this? What's going to happen when we're there? Will we be able to speak to our doctor?  

Fortunately, every time during those appointments, we did have a clinic visit and were able to address any concerns that happened along the way. And we really appreciated that line of communication that continued even after the active treatment stopped. 

Clay (27:24) 
I'm glad you're sharing that too, because the needs of a family may shift throughout the treatment trajectory and after treatment. So the things that were working for you, the preparation that you did with your family, that may need to shift in different stages of your child's illness journey.  

The other thing too that I was thinking is with so many of these serious illnesses, routine follow-up is needed, but the difference was that follow-up would have been at home if you hadn't done this clinical trial. So that is something that parents should think about. 

Carina (28:00) 
Yes, that is a big difference. And the other is that some of the lab tests or diagnostic imaging or other tests that were done during these follow-ups may have been part of the clinical trial and not standard treatment. So, there were components of those follow-ups that differed from standard treatment. 

Clay (28:27) 
Right. And parents will know that upfront. They'll be able to see, okay, what is the difference in follow-up care for standard versus clinical trial? 

Seth (28:39) 
That's right. So when we go through and talk to a family about a trial, we'll have a consent document, which is the stack of papers that talks about what that trial is. And that includes information on what the disease is and what the treatment is, what the standard treatment is, and standard checkups like x-rays or blood work, but then also what is different about the trial. And those are typically separated out within that paperwork to try to make it very clear what are the extra things we're asking of you to be a participant on the trial. 

Carina (29:05) 
And it can be a lot depending on the trial. 

Seth (29:11) 
Absolutely, yeah. So, as much as we try to make it minimal, sometimes it does become a lot. 

Clay (29:19) 
Another thought that's coming up for me is Nolan himself and preparing him for this. And I'm curious what that was like. 

Carina (29:32) 
It was heartbreaking. To be honest, we were very honest with our kids from the beginning. We all sat down as a family and we explained what that initial MRI had found and what we were looking at. It was uncertain at that time, but in as basic, most straightforward way, we did explain that. And as the treatment progressed, we kept that line of communication open with our kids.  

Nolan was scared. He was overwhelmed. His life suddenly changed. He went from playing soccer and being a goofy, funny eight-year-old to being really sick. He came home from surgery in a wheelchair because he had trouble walking. His vision had been affected from the surgery. So it took a while for those things to work themselves out and find our new normal.  

So through this process, it was important to me to support Nolan and help him know what was happening to him along the way, have his siblings be a part of that as well, so they knew why we needed to be away. And help Nolan begin to advocate for himself. 

Clay (31:13) 
Because the doctors actually sat down with Nolan and basically reviewed the clinical trial in a child-friendly way. I mean, is that right? 

Carina (31:21) 
That is right. He was eight and he was sitting on my lap because he felt so sick. And we went through what this would mean. He knew he would have to move away from home for a time to receive medicine that would hopefully cure him.  

So, that was interesting. He knew. And I don't know if Dr. Karol, you can... talk a little bit more about that.

Seth (31:51) 
Yeah, I can talk about what I've seen other families, and maybe you can tell me whether that was Nolan's experience too. But I mean, I think a huge part of getting a child comfortable with medical care at all, let alone a clinical trial, is really to be a good ally to the parents and to help them make the child comfortable.  

And so the example of Nolan sitting in your lap during this conversation is a perfect one, right? That's a place of comfort for kids and where they're going to feel safe and protected. And so it's a great example that we see with a lot of kids. We typically have these conversations with the whole family. So the kid in mom's or dad's lap or in the bed while the parents sitting right next to them as we have these conversations. And that helps the child see that the parents are calm and they're hearing you and that everyone's together on that and that I'm not some stranger that comes in with a stethoscope on my shoulders, but rather that someone that is there to take care of them with their parents. And parents know their kids best.  

And so I always ask, what's going to help them be comfortable? What are the things we're doing that are bothering them right now? And for almost every child that I meet, they were like Nolan. They were really healthy before this happened to them. And so it's usually a huge adjustment to go from someone who maybe never had their blood drawn before to being in a hospital bed and feeling sick because of the illness they have, but then also having people come in at all hours to check on them or to draw blood work or to do other things.  

And so, you know, parents are our first and best resource in terms of making the child comfortable. But then I'm really lucky to work in a place where I've got a lot of other help available to me. 

And so people like child life specialists are great not only at finding play in ways to distract children and make that health care environment much more normal, but in having that language to talk to an eight-year-old about a serious illness or to talk to even a younger child and just help them understand what's happening. 

And I've tried to steal some of their tricks along the way, but they're so much better at it than I am. Social workers are there to help families and really support them because whether you're away from home or not, having a child with a serious illness is a huge change from when everyone is healthy.  

And so really finding all of those resources that your hospital may have to support you and to support your child, to get them comfortable with health care broadly, not just a trial.