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Top 10 Achondroplasia Trials [2022 Studies] | Power

Achondroplasia Clinical Trials

Achondroplasia studies recruiting patients for novel treatments. Filter by phase, distance, and inclusion criteria to find your perfect clinical trial.

All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll in this open-label extension (OLE) study for up to an additional 24 months of treatment.

Approximately 63 participants will be offered to continue at the previously received dose of Recifercept either

Low Dose Medium Dose High Dose

or at the therapeutic dose once it is identified.

Participants will attend the clinic monthly for 24 months. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements & patient/caregiver quality of life questionnaires.

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Study of Infigratinib in Children With Achondroplasia

This study is evaluating whether a drug called infigratinib can help children with Achondroplasia.

My husband was hurt badly,  they have and still are taking really good care of him.  He is now in ICU waiting for his own room.  The staff and doctors are very understanding and they really seem to care not just for their patients but you too.

My wife went in because of blurred vision. Each member of the staff took their time and showed patients and empathy. Also, we did not receive multiple diagnosis without doing due diligence. You can tell that staff wanted to get it right and not treat you as another number. I’ve been to numerous different hospitals—I’m originally from Louisiana & served in military—Vanderbilt is at the top of the list for me!!

I was treated nicely, but one of the nurses stole my shoes and clothes that I came into the hospital with. No one in the hospital wanted to help me find my stuff, after claiming to look for weeks my stuff never came up. Watch your belongings while their because if something comes up missing they will not help you out in any way.

My dad is 87. Yesterday, he had a series of tests. Radiology and Urology. The staff treated him so well and everything was on time, which is so appreciated. The outdoor dining area is nice. The food was good and the restaurant is very clean. Again, very friendly staff. The free parking is also appreciated.

Dr. Michael Longo and Dr. Morone have been absolutely amazing during my father's stay. His chances were not the best but they both worked around the clock to make sure my father made it. I am forever thankful for the kindness and determination that came from them. We were informed and supported every single step up the way. I owe everything to them.

Best children's hospital around!! They took the most incredible care of our 15 month old girl and us too in a very scary time in our lives! Never have we experienced better bed side manor from everyone there!! We will travel from our Kentucky home every time to take our little baby here because the care they give is in matched by any other hospital! Thank you all for the amazing care you have our family!!

I had relatives travel from Kentucky, for a well-prepared and safely arranged surgery. They used their limited resources to get quality health care for their precious daughter. Unfortunately, they experienced disrespectful and selfish changes. The assigned Medical professionals, made foolish excuses for their ugly manners and rotten behavior. A good Hospital should have stricter rules about honesty, kindness, compassion, and respect. If a Medical care facility wants continuous business, Patients must be dignified and given genuine professional Services. To maintain a positive reputation, entire Medical staff should constantly research, and make adjustments to improve Health care procedures. Regardless of differences and opinions, Hospitals should sincerely endeavor on giving respectful Medical Care. This Hospital was disappointing and refused to satisfy Health care preferences. They completely failed at honoring Patient's wishes. All hard-working Families deserve the best Health Treatments possible. Until necessary changes happen, I wouldn't recommend This facility to anyone. Instead I'll recommend People go elsewhere.

We are so fortunate to have the excellent care of Cincinnati Children's Hospital in our backyard! We love the care we receive from numerous departments and are forever grateful for the continued progress we see in our child's health.

First time I ever came to this hospital was back in 01/21. First of all, I want to start of with the way they rushed to check my son was great because he was under 60days. I loved how they were working hard to treat him but I had the worst experience when I was checking out. The Nurse that was working with  him to check him out was so cruel, the reason why I am saying this is because so my baby had IV and instead of slowing taking the IV out of my babies skin. She pulled it off him with all the tapes on his skin and there was so much blood all over his clothes and mine. When I asked her she was shocked she acted like nothing happened and quickly wiped off the blood and said to me it’s okey ma’am. I wish I said something to that night. I personally think  I could have sued the hospital and she could have lost her license because there is no way a nurse should do that to a innocent baby that’s sick. But I was exhausted from not sleeping for night and night due to my baby being sick.
Today is 10/19/22
And I am in the loopy waiting for them to call me for 3hrs. I honestly thought this hospital is good but it has gotten worst. I didn’t know they take couple hours to call someone especially when you have a child that’s itching all over his body due to allergic to medication. I mean that could get worst while in the loopy. I don’t know 🤷‍♀️ this is insane.

Very clean. Staff is very caring! Cute color codes and animal Themes. Organized appointments. Highly recommend.

This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

This study is evaluating whether a drug called BMN111 can help children with Achondroplasia grow taller.

a really nice campus, definitely a breath of fresh air in atlanta

This is now one of my dream schools after just one tour. I had an amazing experience and cant wait to apply! I loved the landscaping and all the buildings. It was even more amazing when I noticed all the maintenance involved and construction going on to make it an even more amazing campus than it already is.

Great school located in a wonderful neighborhood. Would highly recommend students check it out to pursue either their undergraduate or graduate level degree. Very supportive faculty and staff and beautiful facilities. Definitely a gem in Atlanta.

Only downside is the recent change/elimination of some academic majors. Would recommend keeping more liberal arts programs.

There is a great community at Emory. Teachers and classes are very interesting. Food is decent. Dorms are very nice and rooms are pretty spacious. There is lots of support and people to guide and help you as you transition in your first year. Lots of programs and events all the time to keep you busy and to enjoy with friends! Great university!!!

My mom was scheduled for Cardiologist visit in BCH  by here PCP,  but by the time we  had an appointment (3 month later) We where dined access to our doctor because recommendation from our doctor was not processed yet. This only opened up when my wheelchair mother visited for confirmed appointment. They simple sent us home. They also asked us to switch PCP to one form there Baylor's account if we want to avoid problems in the future.
1. Absolutely unprofessional treatment.
2. Bureaucracy that defeats purpose of hospital.
3. One deportment knows nothing about other departments action .
4. No one in administration cares about anybody. Personal success  doesn't depend on quality of service for this people.
6. Fictional facilities for complains  and quality issue reporting . Phone number listed just dropping calls. Site generates error when submitting complain.

The supposedly Dr. Lisreina toro is super incompetent and rude; totally unprofessional; doesn’t know about manners; completely impolite, and arrogant that thinks that because has tittle can treat people like trash

The website vaccines.gov literally refers to this location as to where you can get vaccinated so I call them twice and there is no answer either time. Then about an hour or so later some extremely rude individual calls from the number, just says "hello" and "you're the one calling me" before I have to ask if this was the "Baylor college of medicine." She says yes, so I attempt to explain my situation, then she just cuts me off and says "stop playing on my phone" and go to Walgreens. This is easily one of the most atrocious examples of customer service I have ever encountered. I would give a -5 if it was possible. Thorough retraining or replacement is evident here. It's almost laughable how terrible that individual was.

This facility has the rudest staff I have ever dealt with.   They did not run my insurance before the appt and sent me a bill for $550.  I called them and they said it had not been approved.   I called my insurance and they said it had never been submitted and it would only take 3 days to approve it.  I called them back and they pushed a button and stated now it is submitted.    Also I was transferred about 4 times for each call,  and they asked verification each time.    Which is unnecessary, was not trying to get any info,  just trying to get an appt.    I called a different urologist after the incident,  got an appt and insurance verification.    Without being stressed.   I will not recommend the staff to anyone

I tried to see a doctor for the first time and was treated incredibly unprofessionally. I scheduled my appointment via the online portal about two months in advance and received a call with less than 48 hours notice that the doctor I had scheduled with will not be in the office that day and to reschedule, despite receiving several texts and email to confirm my appointment within two weeks of the appointment. This is the most unprofessional service I have ever encountered at a doctor's office, and I will be looking elsewhere to see a physician.

Achondroplasia Clinical Trials

Reciferceptfor Achondroplasia

Infigratinib 0.128 Mg/kgfor Achondroplasia

Active BMN 111: Daily Subcutaneous Injection Of 15 Ug/kg Of BMN 111for Achondroplasia