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Alkylating agents

Cyclophosphamide for ROHHAD

Q: What treatments has Cyclophosphamide been demonstrated to be effective for?

<p>For <a href="https://www.withpower.com/clinical-trials/multiple-sclerosis">multiple sclerosis</a>, cyclophosphamide is a common form of treatment. Off-label uses for this drug include mixed-cell type <a href="https://www.withpower.com/clinical-trials/lymphoma">lymphoma</a>, acute <a href="https://www.withpower.com/clinical-trials/leukemia">leukemia</a>, myelocytic <a href="https://www.withpower.com/clinical-trials/cancer">cancer</a> and retinoblastoma.</p>

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 18 months

Awards & highlights

Study Summary

This trial is to study the effects of a cancer drug on a rare disorder that causes obesity, problems with the nervous system, and other issues. The goal is to see if the drug improves symptoms and to find objective markers of the disease.

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).Check my eligibility

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.See study design

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 below

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I use a BiPAP machine for breathing support.

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I have been diagnosed with ROHHAD syndrome by two doctors.

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I am not pregnant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 18 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~18 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 18 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Safety and toxicity of high-dose cyclophosphamide used to treat children with ROHHAD of as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.0.

Symptomatic improvement on the ROHHAD Symptoms Scale of children with ROHHAD syndrome after treatment with high-dose cyclophosphamide .

Secondary outcome measures

Neuropsychological function of patients with ROHHAD before and after high-dose cyclophosphamide measured using complementary age appropriate evaluation tools including Wechsler Preschool and Primary Scale of Intelligence RAN objects, and AIM.

To calculate Area Under Curve (AUC) of high dose cyclophosphamide in obese children.

Side effects data

From 2023 Phase 2 trial • 27 Patients • NCT04002401

88%

Pyrexia

65%

Neutrophil count decreased

62%

Nausea

58%

Hypotension

50%

Anaemia

46%

Headache

38%

Decreased appetite

38%

Fatigue

35%

Confusional state

31%

Hypokalaemia

31%

Diarrhoea

31%

Tachycardia

27%

Constipation

27%

Back pain

27%

Hypophosphataemia

23%

Dizziness

23%

Platelet count decreased

23%

Tremor

23%

B-cell lymphoma

23%

White blood cell count decreased

19%

Oedema peripheral

19%

Neutropenia

19%

Cough

19%

Hypogammaglobulinaemia

19%

Hyponatraemia

19%

Tachypnoea

19%

Agitation

15%

Alanine aminotransferase increased

15%

Thrombocytopenia

15%

Chills

15%

Dyspnoea

15%

Hypomagnesaemia

15%

Sinus tachycardia

15%

Dysphagia

12%

Hypertension

12%

Vomiting

12%

Abdominal pain

12%

Aspartate aminotransferase increased

12%

Pain

12%

Malaise

12%

Myalgia

12%

Hypoxia

12%

Arthralgia

12%

Hyperglycaemia

12%

Covid-19

12%

Peripheral sensory neuropathy

Hyperhidrosis

Aphasia

Pancytopenia

Muscular weakness

Pneumonia

Encephalopathy

Eye pain

Gait disturbance

Oral candidiasis

Urinary tract infection

Sepsis

Blood creatinine increased

Acute myeloid leukaemia

Insomnia

Somnolence

Dysuria

Asthenia

Lymphocyte count decreased

Pleural effusion

Covid-19 pneumonia

Respiratory failure

Febrile neutropenia

Embolism

Depression

Syncope

100%

80%

60%

40%

20%

Study treatment Arm

Axicabtagene Ciloleucel and Rituximab Combination

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

1995

Completed Phase 3

~3780

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Johns Hopkins UniversityLead Sponsor

2,256 Previous Clinical Trials

14,820,071 Total Patients Enrolled

Allen Chen, MD, PhDStudy ChairSidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Media Library

Cyclophosphamide (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT02441491 — N/A

ROHHAD Research Study Groups: Cyclophosphamide

ROHHAD Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT02441491 — N/A

Cyclophosphamide (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02441491 — N/A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is it currently possible to join the clinical trial?

"Information from clinicaltrials.gov shows that this medical trial is no longer accepting patients, having begun in March 2015 and last updated April 2021. This notwithstanding, there are presently 2234 other trials actively recruiting participants."

Answered by AI

What additional research has been done using Cyclophosphamide?

"Currently, 816 clinical trials exploring the effects of Cyclophosphamide are running. Of those active studies, 154 have reached Phase 3 and 27828 locations are participating in them - Philadelphia, Pennsylvania being one of the most concentrated hubs for these investigations."

Answered by AI

What is the upper limit for this clinical trial's enrolment?

"The recruitment period for this trial has ended. It was initially advertised on March 1st 2015, and the listing most recently updated on April 21st 2022. Currently, there are 1418 trials searching for participants with hypoventilation and 816 studies recruiting patients undergoing treatment with Cyclophosphamide."

Answered by AI

What treatments has Cyclophosphamide been demonstrated to be effective for?

"For multiple sclerosis, cyclophosphamide is a common form of treatment. Off-label uses for this drug include mixed-cell type lymphoma, acute leukemia, myelocytic cancer and retinoblastoma."

Answered by AI

Is this experiment in search of elderly participants?

"This trial is targeting minors aged 12 months to 22 years old. Clinicialtrials.gov lists 666 studies for participants below 18 and 1619 that involve patients 65 or older."

Answered by AI

What is the criteria for selecting participants in this experiment?

"This research project is searching for 12 patients aged between 1 year and 22 years with hypoventilation. To be eligible, the following conditions must also be fulfilled: participants must have familial care-givers, necessitate bilevel positive airway pressure (BiPAP) support and female candidates post puberty should present a negative pregnancy test outcome."

Answered by AI

Recent research and studies

PediatricsJournal

Improved Behavior and Neuropsychological Function in Children with ROHHAD After High-dose Cyclophosphamide

Jacobson, Lisa A., Shruti Rane, Lisa J. McReynolds, Diana A. Steppan, Allen R. Chen, and Ido Paz-Priel. 2016. “Improved Behavior and Neuropsychological Function in Children with ROHHAD After High-dose Cyclophosphamide”. Pediatrics. American Academy of Pediatrics (AAP). doi:10.1542/peds.2015-1080.

clinicaltrials.govStudy

Treatment of Rapid Onset Obesity, Hypoventilation, Hypothalamic Dysfunction, and Autonomic Dysregulation (ROHHAD )

2015. "Treatment of Rapid Onset Obesity, Hypoventilation, Hypothalamic Dysfunction, and Autonomic Dysregulation (ROHHAD )". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02441491.