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

Q: What is the mechanism of action of this treatment?

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.

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 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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My lung function is below 50% of what's expected for my age, or my oxygen levels are below 93%.

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I rely on a machine to help me breathe.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

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%

Fatigue

38%

Decreased appetite

35%

Confusional state

31%

Tachycardia

31%

Diarrhoea

31%

Hypokalaemia

27%

Constipation

27%

Hypophosphataemia

27%

Back pain

23%

Dizziness

23%

Tremor

23%

B-cell lymphoma

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%

Hypomagnesaemia

15%

Thrombocytopenia

15%

Dysphagia

15%

Alanine aminotransferase increased

15%

Sinus tachycardia

15%

Chills

15%

Dyspnoea

12%

Aspartate aminotransferase increased

12%

Pain

12%

Arthralgia

12%

Myalgia

12%

Hypertension

12%

Abdominal pain

12%

Hyperglycaemia

12%

Hypoxia

12%

Vomiting

12%

Peripheral sensory neuropathy

12%

Covid-19

12%

Malaise

Dysuria

Muscular weakness

Blood creatinine increased

Hyperhidrosis

Insomnia

Acute myeloid leukaemia

Encephalopathy

Sepsis

Pancytopenia

Asthenia

Eye pain

Urinary tract infection

Lymphocyte count decreased

Somnolence

Oral candidiasis

Pneumonia

Gait disturbance

Aphasia

Pleural effusion

Depression

Embolism

Syncope

Respiratory failure

Febrile neutropenia

Covid-19 pneumonia

100%

80%

60%

40%

20%

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

0 / 6Eligibility 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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