Using Adrenocorticotropic Hormone to Treat Children with Nephrotic Syndrome

Study of Adrenocorticotropic Hormone on Children With Frequent Relapse or Steroid-dependent Nephrotic Syndrome: a Prospective, Multicenter, Randomized，Open-label Clinical Trial.

This study is testing if adding a hormone treatment to steroids can help children with nephrotic syndrome feel better and have fewer side effects.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 140 (estimated)
• Ages: 2 Years to 14 Years
• Sex: All
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine (Academic / other)
• Drugs / interventions: rituximab, beliumab, cyclophosphamide, Prednisone
• Locations: 8 sites (Wuhan, Hubei and 7 other locations)
• Trial ID: NCT06079788 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the effectiveness and safety of combining adrenocorticotropic hormone (ACTH) with steroids in treating children aged 2-14 who suffer from frequently relapsing or steroid-dependent nephrotic syndrome. The study is multicenter and randomized, aiming to provide individualized treatment options for these patients. By exploring the efficacy of ACTH, the trial seeks to offer a safer alternative to traditional steroid treatments, which can lead to significant side effects. The goal is to improve outcomes for children who do not respond well to standard therapies.

Who should consider this trial

Good fit: Ideal candidates are children aged 2-14 with steroid-sensitive but frequently relapsing or steroid-dependent nephrotic syndrome.

Not a fit: Patients with a family history of nephrotic syndrome or those with congenital or acquired immunodeficiency may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective and safer option for managing nephrotic syndrome in children.

How similar studies have performed: Previous studies have shown promising results using ACTH for nephrotic syndrome, indicating potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Age 2-14 years old;
2. Sensitive but frequent relapses or steroids dependence nephrotic syndrome
3. No severe hormonal side effects and/or low-dose steroids dependent idiopathic nephrotic syndrome in children (defined as two relapses with an average dose \< 0.5mg/kg/day or equivalent alternate-day dose)
4. Normal renal function: eGFR≥90ml/min/1.73m2;
5. Morning urine protein \<1+ or urine protein-creatinine ratio \<0.2g/g (\<20 mg/mmol) for 3 consecutive days and above when in enroll;
6. Prednisone dose was 1.5-2 mg/kg per day before admission;
7. No use of other immunosuppressants (such as tacrolimus, mortecophenolate, cyclosporin A, cyclophosphamide, levamisole, imidazole ribin, or tripterygium, etc.) within 3 months, and no use of rituximab or beliumab within 6 months.

Exclusion Criteria:

1. Family history of nephrotic syndrome, chronic glomerulonephritis, uremia and other kidney diseases;
2. Patients with congenital or acquired immunodeficiency, or with active tuberculosis, active CMV, EBV, hepatitis B, hepatitis C, HIV infection, deep fungal infection, or other active infections;
3. Recurrent or persistent hypertension;
4. Secondary nephrotic syndrome, such as nephrotic syndrome secondary to systemic lupus erythematosus, diabetes, drug poisoning and infection;
5. Combined with other kidney diseases, such as polycystic kidney, ANCA vasculitis, urinary system malformations, etc.;
6. Patients with hypertension, diabetes, tuberculosis, suppurative or fungal infection, gastric and duodenal ulcer disease and heart failure; Patients with other serious heart, liver and other important organs, blood system, endocrine system and other system lesions;
7. Co-occurrence of other monogenic genetic diseases known to affect the condition of nephrotic syndrome;
8. Patients with serious autoimmune diseases or tumors;
9. Use of other immunosuppressants (such as tacrolimus, mortecophenolate, cyclosporin A, cyclophosphamide, levamisole, imidazole ribin, or tripterygium, etc.) within 3 months, and no use of rituximab or beliumab within 6 months;
10. Patients who are known to be allergic to ACTH, glucocorticoids, or any of the components of these drugs, and patients with severe hormone-related side effects
11. History of organ transplantation (excluding corneal and hair transplantation);
12. Patients who had participated in other clinical trials within three months prior to enrollment;
13. Any patient whom the investigator determines is not suitable for inclusion in the trial.

Where this trial is running

Wuhan, Hubei and 7 other locations

• Tongji Hospital — Wuhan, Hubei, China (Recruiting)
• Nanjing Children's Hospital — Nanjing, Jiangsu, China (Recruiting)
• Kunming Children's Hospital — Kunming, Yunnan, China (Recruiting)
• Children's Hospital, Zhejiang University School of Medicine — Hangzhou, Zhejiang, China (Recruiting)
• Ningbo Women & Children's Hospital — Ningbo, Zhejiang, China (Recruiting)
• Yuying Childrens Hospital of Wenzhou Medical University — Wenzhou, Zhejiang, China (Recruiting)
• Children's Hospital affiliated to Capital Institute of Pediatrics — Beijing, China (Recruiting)
• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine — Shanghai, China (Recruiting)

Study contacts

• Study coordinator: jianhua Mao, MD

Conditions Nephrotic Syndrome in Children; Nephrotic Syndrome; Frequently Relapsing Nephrotic Syndrome; Steroid Dependent Nephrotic Syndrome; Adrenocorticotropic Hormone