Clofarabine for Langerhans in Pedi

NCT01796405 | Withdrawn Phase 2 DMC

• 1 other identifier: 12-050
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if the drug is effective for treating different diseases. It also means that the FDA has not yet approved clofarabine for your disease. Clofarabine is a chemotherapy drug that has been used in the treatment of leukemia in children and adults. Information from other research studies suggests that this drug may also be effective in patients with LCH. The purpose of this study is to estimate the response rates of participants with recurrent LCH to clofarabine within each of two strata: a) low-risk participants with disease reactivation, and b) high-risk participants with risk-organ involvement. Other purposes are to estimate the progression-free survival after clofarabine treatment, estimate survival of participants with refractory multi-system LCH with risk organ involvement treated with clofarabine and to describe toxicities of clofarabine in participants with LCH.

Conditions

Langerhans Cell Histiocytosis

Outcome Measures

Primary Outcomes (1)

• Estimated Response Rate

  To estimate the response rate of participants with recurrent LCH to clofarabine within each of two strata: a) low-risk participants with disease reactivation; and b) high-risk participants with risk-organ involvement that is refractory to standard treatment.

  2 years

Secondary Outcomes (3)

• Estimated of Progression Free Survival

  2 years

• Estimated survival with risk organ involvement

  2 years

• Chemotherapy feasibility

  2 years

Study Arms (2)

Low-Risk

EXPERIMENTAL

Multifocal or Low-Risk Multi System Clofarabine, Low Dose, Two Cycles

Drug: Clofarabine

High-Risk

EXPERIMENTAL

High-risk Multi System Clofarabine, Standard Dose, Two Cycles

Drug: Clofarabine

Interventions

Clofarabine DRUG

High-Risk; Low-Risk

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Have failed first line treatment with prednisone and vinblastine
• Clinical evidence of involvement of hematopoietic system, liver or spleen
• Have failed salvage treatment with cladribine/cytarabine or are not considered to be eligible for such treatment
• Willing to use effective contraception during study and for six months after study treatment
• Adequate marrow and organ function

You may not qualify if:

• Pregnant or breastfeeding
• Have received chemotherapy or radiotherapy within 2 weeks of study entry
• Have not recovered from adverse events due to previously administered agents
• Receiving other study agents
• Taking drugs with known renal toxicity
• Use of alternative medicine during study treatment
• Uncontrolled intercurrent illness
• History of a different malignancy except if disease-free for at least five years or within five years for cervical cancer in situ and basal and squamous cell carcinoma of the skin
• Known to be HIV positive on antiretroviral therapy

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead

MeSH Terms

Conditions

Histiocytosis, Langerhans-Cell

Interventions

Clofarabine

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Adenine Nucleotides; Purine Nucleotides; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Nucleotides; Ribonucleotides

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 19, 2013
• First Posted: February 21, 2013
• Primary Completion: October 1, 2017
• Last Updated: August 25, 2015

Record last verified: 2015-08