What side effects are associated with this type of intervention?

High-dose cyclophosphamide, used as an immunosuppressive treatment for ROHHAD, can cause several side effects. These include immunosuppression, increasing the risk of infections, bone marrow suppression leading to anemia, leukopenia, and thrombocytopenia, gastrointestinal disturbances such as nausea and vomiting, and potential long-term risks like secondary malignancies and infertility.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for ROHHAD mentioned in the provided context. However, a clinical trial is studying the use of high-dose cyclophosphamide for its immunosuppressive effects, which inhibit DNA replication, to treat this condition. This approach is based on the hypothesis that ROHHAD may be driven by an immune system attack on specific brain areas.

What other types of research exist?

Promising novel alternatives to high-dose cyclophosphamide for ROHHAD patients in 2024 could include immune checkpoint inhibitors such as anti-PD-1 or anti-PD-L1 therapies. These therapies have shown efficacy in other conditions involving immune system dysregulation and may offer a targeted approach with potentially fewer side effects.

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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

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%

Vomiting

12%

Hypertension

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

Aphasia

Hyperhidrosis

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

~3770

0 / 3Eligibility criteria met

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.

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