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Alkylating agents
Cyclophosphamide for ROHHAD
N/A
Waitlist Available
Research Sponsored by Johns Hopkins University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patients requiring bilevel positive airway pressure (BiPAP) support are eligible
Diagnosis of ROHHAD syndrome confirmed by two physicians
Must not have
Inadequate pulmonary function, i.e. forced vital capacity or forced expiratory volume at one second < 50% of predicted for children greater than 8 years of age, or oxygen saturation <93% on pulse oximetry for younger children
Ventilator dependent
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Summary
This trial uses high-dose cyclophosphamide to treat children with ROHHAD syndrome. The medication aims to weaken the immune system to stop it from damaging the brain, which should help reduce severe symptoms. Cyclophosphamide has been used to treat various conditions in children, including nephrotic syndrome and other severe diseases.
Who is the study for?
This trial is for children with ROHHAD syndrome, confirmed by two doctors. They should have shown some improvement after rituximab treatment and be cared for at home. It's not for kids with severe heart or lung issues, those on a ventilator, pregnant girls, those with chromosomal abnormalities, or active cancer (except neuroblastoma in follow-up).
What is being tested?
The study tests high-dose cyclophosphamide to suppress the immune system in patients with ROHHAD syndrome. The goal is to see if it can improve symptoms and find objective ways to measure disease activity.
What are the potential side effects?
Cyclophosphamide may cause side effects like increased risk of infections due to weakened immunity, potential bladder irritation or bleeding, nausea and vomiting, hair loss during treatment period.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I use a BiPAP machine for breathing support.
Select...
I have been diagnosed with ROHHAD syndrome by two doctors.
Select...
I am not pregnant.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My lung function is below 50% of what's expected for my age, or my oxygen levels are below 93%.
Select...
I rely on a machine to help me breathe.
Select...
I have a known genetic abnormality.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Side effects data
From 2023 Phase 2 trial • 27 Patients • NCT0400240188%
Pyrexia
65%
Neutrophil count decreased
62%
Nausea
58%
Hypotension
50%
Anaemia
46%
Headache
38%
Fatigue
38%
Decreased appetite
35%
Confusional state
31%
Hypokalaemia
31%
Tachycardia
31%
Diarrhoea
27%
Constipation
27%
Hypophosphataemia
27%
Back pain
23%
Dizziness
23%
B-cell lymphoma
23%
Tremor
23%
Platelet count decreased
23%
White blood cell count decreased
19%
Cough
19%
Agitation
19%
Hyponatraemia
19%
Neutropenia
19%
Tachypnoea
19%
Hypogammaglobulinaemia
19%
Oedema peripheral
15%
Thrombocytopenia
15%
Dysphagia
15%
Alanine aminotransferase increased
15%
Hypomagnesaemia
15%
Sinus tachycardia
15%
Chills
15%
Dyspnoea
12%
Aspartate aminotransferase increased
12%
Pain
12%
Arthralgia
12%
Myalgia
12%
Vomiting
12%
Hypertension
12%
Abdominal pain
12%
Hyperglycaemia
12%
Hypoxia
12%
Peripheral sensory neuropathy
12%
Covid-19
12%
Malaise
8%
Dysuria
8%
Blood creatinine increased
8%
Hyperhidrosis
8%
Insomnia
8%
Encephalopathy
8%
Lymphocyte count decreased
8%
Muscular weakness
8%
Sepsis
8%
Pancytopenia
8%
Acute myeloid leukaemia
8%
Asthenia
8%
Eye pain
8%
Urinary tract infection
8%
Somnolence
8%
Oral candidiasis
8%
Pneumonia
8%
Gait disturbance
8%
Aphasia
4%
Depression
4%
Syncope
4%
Embolism
4%
Pleural effusion
4%
Respiratory failure
4%
Febrile neutropenia
4%
Covid-19 pneumonia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Axicabtagene Ciloleucel and Rituximab Combination
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: CyclophosphamideExperimental Treatment1 Intervention
Cyclophosphamide will be given by vein once a day for four straight days.
Ten days after starting cyclophosphamide, filgrastim, a drug that helps normal blood cells to grow, will be given by vein once every day to try to help your blood cells grow faster.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
2010
Completed Phase 4
~2310
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
High-dose cyclophosphamide is used in the treatment of ROHHAD due to its potent immunosuppressive properties. Cyclophosphamide works by inhibiting DNA replication, which prevents the proliferation of rapidly dividing cells, including those of the immune system.
This is particularly important for ROHHAD patients, as the syndrome is believed to be driven by an autoimmune attack on specific areas of the brain. By suppressing the immune system, cyclophosphamide can help reduce this harmful immune response, potentially alleviating the severe symptoms associated with the disorder.
[Treatment of patients with advanced ovarian cancer at the Gynecological Clinic of Pécs Medical University].Chemotherapy for paranasal sinus carcinoma. A 10-year experience at Wayne State University.Hyperthermia generated with ferucarbotran (Resovist®) in an alternating magnetic field enhances cisplatin-induced apoptosis of cultured human oral cancer cells.
[Treatment of patients with advanced ovarian cancer at the Gynecological Clinic of Pécs Medical University].Chemotherapy for paranasal sinus carcinoma. A 10-year experience at Wayne State University.Hyperthermia generated with ferucarbotran (Resovist®) in an alternating magnetic field enhances cisplatin-induced apoptosis of cultured human oral cancer cells.
Find a Location
Who is running the clinical trial?
Johns Hopkins UniversityLead Sponsor
2,316 Previous Clinical Trials
14,872,781 Total Patients Enrolled
Allen Chen, MD, PhDStudy ChairSidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have an active cancer diagnosis, but my neuroblastoma only needs follow-up.My lung function is below 50% of what's expected for my age, or my oxygen levels are below 93%.I rely on a machine to help me breathe.I use a BiPAP machine for breathing support.I have been diagnosed with ROHHAD syndrome by two doctors.I felt better after receiving a course of rituximab.I am not pregnant.Your heart's pumping function is not strong enough.I have a known genetic abnormality.Your brain MRI shows no abnormalities.You are receiving care at home from your family.
Research Study Groups:
This trial has the following groups:- Group 1: Cyclophosphamide
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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