Novel, out-patient based oral combination therapy for children with Langerhans cell histiocytosis that has relapsed or is refractory to therapy

Phase 2

Recruiting

Conditions: Multifocal and multisystemic (disseminated) Langerhans-cell histiocytosis,

Registration Number: CTRI/2020/07/026937

Lead Sponsor: RAMYA UPPULURI

Brief Summary: Relapsed / refractory Langerhans cell histiocytosis(LCH) has been known to have a guarded prognosis. Salvage chemotherapyprotocols can be expensive with significant toxicity and increased risk ofmorbidity and mortality. A combination of pulse dexamethasone and lenalidomideis a cost-effective outpatient-based protocol which has been derived based onthe underlying pathophysiology of LCH. The proposed protocol includes sixcycles of the oral combination as a salvage treatment option for relapsed /refractory LCH.

Detailed Description: Not available

Recruitment & Eligibility

Status: Open to Recruitment

Sex: All

Target Recruitment: 50

Inclusion Criteria

1.Patients from 1 year of age to 18 years of age with biopsy proven LCH 2.Relapsed or refractory disease after having received 6 to 12 weeks of induction chemotherapy with Vinblastine plus prednisolone as per LCH II or III protocol 3.Relapsed or refractory disease after receiving salvage chemotherapy with cladribine +/- cytarabine can also be included 4.Patients with low-risk disease at the time of relapse may be offered LenDex upfront 5.Patients with high-risk disease at the time of relapse may be offered LenDex upfront or after salvage treatment, based on the discretion of the treating physician.

Exclusion Criteria

1.Pregnancy or lactation 2.Patients with renal impairment defined as a serum creatinine > 1 3.Patients with liver impairment (excluding those due to disease) defined as SGPT and/or PT more than 5 times the upper limit of normal 4.Patients with pre-existing peripheral neuropathy.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
1.Response to six cycles of chemotherapy	EVERY 3 MONTHS FOR THE FIRST 2 YEARS \| EVERY 6 MONTHS FOR THE NEXT 2 YEARS
2.Event free survival and Overall Survival for all patients treated as per this protocol	EVERY 3 MONTHS FOR THE FIRST 2 YEARS \| EVERY 6 MONTHS FOR THE NEXT 2 YEARS
3.Adverse effects of the drugs noted during and post treatment	EVERY 3 MONTHS FOR THE FIRST 2 YEARS \| EVERY 6 MONTHS FOR THE NEXT 2 YEARS
4.Correlation of risk factors at the time of enrolment in the study with outcome	EVERY 3 MONTHS FOR THE FIRST 2 YEARS \| EVERY 6 MONTHS FOR THE NEXT 2 YEARS

Secondary Outcome Measures

Name	Time	Method
Cost-effectiveness of the combination LenDex	Every 6 months for 2 years

Trial Locations

Locations (1): APOLLO HOSPITALS
🇮🇳
Chennai, TAMIL NADU, India
APOLLO HOSPITALS
🇮🇳Chennai, TAMIL NADU, India
RAMYA UPPULURI
Principal investigator
8939710906
ramya.december@gmail.com