Cystic FibrosisPhase 2NCT00406536
Study of LYM-X-SORB™ to Improve Fatty Acid and Choline Status in Children With CF and PI
A Phase 2 study testing Lym-X-Sorb powder in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Avanti Polar Lipids, Inc. at 1 site in United States.
Trial summary
The purpose of this phase II research study is to evaluate the effectiveness of the next generation LYM-X-SORB™ in improving the essential fatty acid (EFA) and choline status for children and adolescents with Cystic Fibrosis (CF) and pancreatic insufficiency (PI). Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Cystic Fibrosis, Pancreatic Insufficiency
- What is being tested?
- Lym-X-Sorb powder
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 110 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2007to Jun 2012
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosed as having cystic fibrosis (CF) with pancreatic insufficiency (PI)
- Subjects aged 6 to 17 years of age
- In usual state of good health
- Family and subject commitment to the 18-month study protocol
- Fecal elastase < 15µg/g stool
Who cannot join
- Forced expiratory volume at one second, % predicted (FEV1) < 40% predicted
- Other chronic health conditions that may affect GI absorption, growth, dietary intake, nutritional status
- Liver disease, lung transplant, celiac disease, allergy/intolerance to wheat/gluten, pregnant
- Participation in another CF nutrition-related intervention study
- Regular intake of fatty acids (i.e., fish oils) or choline nutritional supplements
- Home parenteral lipid administration (i.e., intralipids)
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
About this study
Official title: Phase II Study: LYM-X-SORB™, an Organized Lipid Matrix: Fatty Acids and Choline in CF
The purpose of this phase II research study is to evaluate the effectiveness of the next generation LYM-X-SORB™ in improving the essential fatty acid (EFA) and choline status for children and adolescents with Cystic Fibrosis (CF) and pancreatic insufficiency (PI).
Read the detailed description
Fat malabsorption is common in individuals with cystic fibrosis (CF) and pancreatic insufficiency (PI). This places them at risk for caloric, essential fatty acid, and choline deficiency, which may in turn, lead to growth failure and a poorer clinical course. The purpose of this research study is to find whether or not taking LYM-X-SORB™ over an 18-month period, every day, will correct the problem people with CF and PI have with absorbing fat and choline. Participation will help CF doctors and other healthcare professionals learn more about the potential benefits of LYM-X-SORB™ to children and adolescents with CF and PI. These benefits may include better absorption of fat, better choline status, better growth in height, weight, muscle and bone, better lungs, and improvement of health status.
The study will enroll a total of 78 participants from Children's Hospital of Philadelphia (CHOP) and from several other Cystic Fibrosis Centers. One half of the participants will be randomly picked to receive the active powder with the LYM-X-SORB™ supplement and one half will receive a placebo (with no active supplement).
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Being tested
- Lym-X-Sorb powder, Placebo powder
- Keywords
- Cystic Fibrosis, Pancreatic Insufficiency, LYM-X-SORB™, LXS
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lym-X-Sorb powderDietary supplement
Lym-X-Sorb is an organized matrix of lyso phosphatidylcholine (LPC), free fatty acid (FFA) and monoglyceride formulated at 20wt% with flour and sugar. A dosage contains 32 grams of powder and is consumed with food twice a day (6 to 11.9 years old) or three times per day (12 to 17.9 years old) for 18 months.
Placebo powderDietary supplement
The placebo is composed of soybean oil, sunflower oil, fully hydrogenated cottonseed oil and flax seed oil dispersed at 16wt% on flour and sugar. The placebo is >99% triglycerides with no trans fatty acids. The fatty acid composition and caloric content is designed to match the active supplement (Lym-X-Sorb). A dosage contains 32 grams of powder and is consumed with food twice a day (6 to 11.9 years old) or three times per day (12 to 17.9 years old) for 18 months.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Not described Receives: Dietary Supplement: Lym-X-Sorb powder |
| 2 | Placebo comparator | Not described Receives: Dietary Supplement: Placebo powder |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvements in essential fatty acid (EFA) and choline status over a 12-month period.
Measured: 12-month period
Secondary outcomes (1)
Improvements in growth, nutritional, and pulmonary status over an 18-month period.
Measured: 18-month period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2006 (estimated)
- Study startJan 2007
- Primary completionJun 2012
- Study completionJun 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (164)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 110 participants (actual)
Sponsor and investigators
- Lead sponsor
- Avanti Polar Lipids, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Principal investigator, Walter A. Shaw, Ph.D, Avanti Polar Lipids, Inc.
- Investigators
- Walter A. Shaw, PhD, Principal investigator, Avanti Polar Lipids, Inc.Virginia A. Stallings, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00406536
- Sponsor's protocol ID
- DK60302
- Other IDs
- R44DK060302, 2R44DK060302-02A1
- Status last verified
- Jan 2013
- First submitted
- 29 Nov 2006
- Last updated
- 14 Jan 2013
- Results posted
- No
Publications
Freedman SD, Blanco PG, Zaman MM, Shea JC, Ollero M, Hopper IK, Weed DA, Gelrud A, Regan MM, Laposata M, Alvarez JG, O'Sullivan BP. Association of cystic fibrosis with abnormalities in fatty acid metabolism. N Engl J Med. 2004 Feb 5;350(6):560-9. doi: 10.1056/NEJMoa021218.
PubMed 14762183Innis SM, Davidson AG, Chen A, Dyer R, Melnyk S, James SJ. Increased plasma homocysteine and S-adenosylhomocysteine and decreased methionine is associated with altered phosphatidylcholine and phosphatidylethanolamine in cystic fibrosis. J Pediatr. 2003 Sep;143(3):351-6. doi: 10.1067/S0022-3476(03)00326-3.
PubMed 14517519Chen A, Innis S. Assessment of phospholipid malabsorption by quantification of fecal phospholipid. J Pediatr Gastroenterol Nutr. 2004 Jul;39(1):85-91. doi: 10.1097/00005176-200407000-00017.
PubMed 15187787Chen AH, Innis SM, Davidson AG, James SJ. Phosphatidylcholine and lysophosphatidylcholine excretion is increased in children with cystic fibrosis and is associated with plasma homocysteine, S-adenosylhomocysteine, and S-adenosylmethionine. Am J Clin Nutr. 2005 Mar;81(3):686-91. doi: 10.1093/ajcn/81.3.686.
PubMed 15755840Lepage G, Yesair DW, Ronco N, Champagne J, Bureau N, Chemtob S, Berube D, Roy CC. Effect of an organized lipid matrix on lipid absorption and clinical outcomes in patients with cystic fibrosis. J Pediatr. 2002 Aug;141(2):178-85. doi: 10.1067/mpd.2002.124305.
PubMed 12183711Roulet M, Frascarolo P, Rappaz I, Pilet M. Essential fatty acid deficiency in well nourished young cystic fibrosis patients. Eur J Pediatr. 1997 Dec;156(12):952-6. doi: 10.1007/s004310050750.
PubMed 9453380Kalivianakis M, Verkade HJ. The mechanisms of fat malabsorption in cystic fibrosis patients. Nutrition. 1999 Feb;15(2):167-9. doi: 10.1016/s0899-9007(98)00155-5. No abstract available.
PubMed 9990588KUO PT, HUANG NN, BASSETT DR. The fatty acid composition of the serum chylomicrons and adipose tissue of children with cystic fibrosis of the pancreas. J Pediatr. 1962 Mar;60:394-403. doi: 10.1016/s0022-3476(62)80065-1. No abstract available.
PubMed 14460644Farrell PM, Mischler EH, Engle MJ, Brown DJ, Lau SM. Fatty acid abnormalities in cystic fibrosis. Pediatr Res. 1985 Jan;19(1):104-9. doi: 10.1203/00006450-198501000-00028.
PubMed 3918287Lloyd-Still JD, Johnson SB, Holman RT. Essential fatty acid status and fluidity of plasma phospholipids in cystic fibrosis infants. Am J Clin Nutr. 1991 Dec;54(6):1029-35. doi: 10.1093/ajcn/54.6.1029.
PubMed 1957818van Egmond AW, Kosorok MR, Koscik R, Laxova A, Farrell PM. Effect of linoleic acid intake on growth of infants with cystic fibrosis. Am J Clin Nutr. 1996 May;63(5):746-52. doi: 10.1093/ajcn/63.5.746.
PubMed 8615359Lepage G, Paradis K, Lacaille F, Senechal L, Ronco N, Champagne J, Lenaerts C, Roy CC, Rasquin-Weber A. Ursodeoxycholic acid improves the hepatic metabolism of essential fatty acids and retinol in children with cystic fibrosis. J Pediatr. 1997 Jan;130(1):52-8. doi: 10.1016/s0022-3476(97)70310-x.
PubMed 9003851Lloyd-Still JD, Bibus DM, Powers CA, Johnson SB, Holman RT. Essential fatty acid deficiency and predisposition to lung disease in cystic fibrosis. Acta Paediatr. 1996 Dec;85(12):1426-32. doi: 10.1111/j.1651-2227.1996.tb13947.x.
PubMed 9001653Strandvik B, Gronowitz E, Enlund F, Martinsson T, Wahlstrom J. Essential fatty acid deficiency in relation to genotype in patients with cystic fibrosis. J Pediatr. 2001 Nov;139(5):650-5. doi: 10.1067/mpd.2001.118890.
PubMed 11713441Gibson RA, Teubner JK, Haines K, Cooper DM, Davidson GP. Relationships between pulmonary function and plasma fatty acid levels in cystic fibrosis patients. J Pediatr Gastroenterol Nutr. 1986 May-Jun;5(3):408-15. doi: 10.1097/00005176-198605000-00013.
PubMed 3723260
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study: LYM-X-SORB™, an Organized Lipid Matrix: Fatty Acids and Choline in CF
- ClinicalTrials.gov ID
- NCT00406536
- Acronym / sponsor ID
- DK60302
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Keywords
- Cystic Fibrosis, Pancreatic Insufficiency, LYM-X-SORB™, LXS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 110 participants (actual)
- Lead sponsor
- Avanti Polar Lipids, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Jan 2007
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 4 Dec 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this phase II research study is to evaluate the effectiveness of the next generation LYM-X-SORB™ in improving the essential fatty acid (EFA) and choline status for children and adolescents with Cystic Fibrosis (CF) and pancreatic insufficiency (PI).
Detailed description
Fat malabsorption is common in individuals with cystic fibrosis (CF) and pancreatic insufficiency (PI). This places them at risk for caloric, essential fatty acid, and choline deficiency, which may in turn, lead to growth failure and a poorer clinical course. The purpose of this research study is to find whether or not taking LYM-X-SORB™ over an 18-month period, every day, will correct the problem people with CF and PI have with absorbing fat and choline. Participation will help CF doctors and other healthcare professionals learn more about the potential benefits of LYM-X-SORB™ to children and adolescents with CF and PI. These benefits may include better absorption of fat, better choline status, better growth in height, weight, muscle and bone, better lungs, and improvement of health status.
The study will enroll a total of 78 participants from Children's Hospital of Philadelphia (CHOP) and from several other Cystic Fibrosis Centers. One half of the participants will be randomly picked to receive the active powder with the LYM-X-SORB™ supplement and one half will receive a placebo (with no active supplement).
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Dietary Supplement: Lym-X-Sorb powder |
| 2 | Placebo comparator | Dietary Supplement: Placebo powder |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Diagnosed as having cystic fibrosis (CF) with pancreatic insufficiency (PI)
- Subjects aged 6 to 17 years of age
- In usual state of good health
- Family and subject commitment to the 18-month study protocol
- Fecal elastase < 15µg/g stool
Key exclusion criteria (6 listed)
- Forced expiratory volume at one second, % predicted (FEV1) < 40% predicted
- Other chronic health conditions that may affect GI absorption, growth, dietary intake, nutritional status
- Liver disease, lung transplant, celiac disease, allergy/intolerance to wheat/gluten, pregnant
- Participation in another CF nutrition-related intervention study
- Regular intake of fatty acids (i.e., fish oils) or choline nutritional supplements
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lym-X-Sorb powderLym-X-Sorb is an organized matrix of lyso phosphatidylcholine (LPC), free fatty acid (FFA) and monoglyceride formulated at 20wt% with flour and sugar. A dosage contains 32 grams of powder and is consumed with food twice a day (6 to 11.9 years old) or three times per day (12 to 17.9 years old) for 18 months. | Dietary supplement | 1 |
| Placebo powderThe placebo is composed of soybean oil, sunflower oil, fully hydrogenated cottonseed oil and flax seed oil dispersed at 16wt% on flour and sugar. The placebo is >99% triglycerides with no trans fatty acids. The fatty acid composition and caloric content is designed to match the active supplement (Lym-X-Sorb). A dosage contains 32 grams of powder and is consumed with food twice a day (6 to 11.9 years old) or three times per day (12 to 17.9 years old) for 18 months. | Dietary supplement | 2 |
Primary outcome
Improvements in essential fatty acid (EFA) and choline status over a 12-month period.
Time frame: 12-month period
1 secondary outcome
Improvements in growth, nutritional, and pulmonary status over an 18-month period.
Time frame: 18-month period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2013.
- Background: PubMed 14762183Freedman SD, Blanco PG, Zaman MM, Shea JC, Ollero M, Hopper IK, Weed DA, Gelrud A, Regan MM, Laposata M, Alvarez JG, O'Sullivan BP. Association of cystic fibrosis with abnormalities in fatty acid metabolism. N Engl J Med. 2004 Feb 5;350(6):560-9. doi: 10.1056/NEJMoa021218.
- Background: PubMed 14517519Innis SM, Davidson AG, Chen A, Dyer R, Melnyk S, James SJ. Increased plasma homocysteine and S-adenosylhomocysteine and decreased methionine is associated with altered phosphatidylcholine and phosphatidylethanolamine in cystic fibrosis. J Pediatr. 2003 Sep;143(3):351-6. doi: 10.1067/S0022-3476(03)00326-3.
- Background: PubMed 15187787Chen A, Innis S. Assessment of phospholipid malabsorption by quantification of fecal phospholipid. J Pediatr Gastroenterol Nutr. 2004 Jul;39(1):85-91. doi: 10.1097/00005176-200407000-00017.
- Background: PubMed 15755840Chen AH, Innis SM, Davidson AG, James SJ. Phosphatidylcholine and lysophosphatidylcholine excretion is increased in children with cystic fibrosis and is associated with plasma homocysteine, S-adenosylhomocysteine, and S-adenosylmethionine. Am J Clin Nutr. 2005 Mar;81(3):686-91. doi: 10.1093/ajcn/81.3.686.
- Background: PubMed 12183711Lepage G, Yesair DW, Ronco N, Champagne J, Bureau N, Chemtob S, Berube D, Roy CC. Effect of an organized lipid matrix on lipid absorption and clinical outcomes in patients with cystic fibrosis. J Pediatr. 2002 Aug;141(2):178-85. doi: 10.1067/mpd.2002.124305.
- Background: PubMed 9453380Roulet M, Frascarolo P, Rappaz I, Pilet M. Essential fatty acid deficiency in well nourished young cystic fibrosis patients. Eur J Pediatr. 1997 Dec;156(12):952-6. doi: 10.1007/s004310050750.
- Background: PubMed 9990588Kalivianakis M, Verkade HJ. The mechanisms of fat malabsorption in cystic fibrosis patients. Nutrition. 1999 Feb;15(2):167-9. doi: 10.1016/s0899-9007(98)00155-5. No abstract available.
- Background: PubMed 14460644KUO PT, HUANG NN, BASSETT DR. The fatty acid composition of the serum chylomicrons and adipose tissue of children with cystic fibrosis of the pancreas. J Pediatr. 1962 Mar;60:394-403. doi: 10.1016/s0022-3476(62)80065-1. No abstract available.
- Background: PubMed 3918287Farrell PM, Mischler EH, Engle MJ, Brown DJ, Lau SM. Fatty acid abnormalities in cystic fibrosis. Pediatr Res. 1985 Jan;19(1):104-9. doi: 10.1203/00006450-198501000-00028.
- Background: PubMed 1957818Lloyd-Still JD, Johnson SB, Holman RT. Essential fatty acid status and fluidity of plasma phospholipids in cystic fibrosis infants. Am J Clin Nutr. 1991 Dec;54(6):1029-35. doi: 10.1093/ajcn/54.6.1029.
- Background: PubMed 8615359van Egmond AW, Kosorok MR, Koscik R, Laxova A, Farrell PM. Effect of linoleic acid intake on growth of infants with cystic fibrosis. Am J Clin Nutr. 1996 May;63(5):746-52. doi: 10.1093/ajcn/63.5.746.
- Background: PubMed 9003851Lepage G, Paradis K, Lacaille F, Senechal L, Ronco N, Champagne J, Lenaerts C, Roy CC, Rasquin-Weber A. Ursodeoxycholic acid improves the hepatic metabolism of essential fatty acids and retinol in children with cystic fibrosis. J Pediatr. 1997 Jan;130(1):52-8. doi: 10.1016/s0022-3476(97)70310-x.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Walter A. Shaw, PhD principal investigator
Avanti Polar Lipids, Inc.
Virginia A. Stallings, MD principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lym-X-Sorb powder in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Avanti Polar Lipids, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvements in essential fatty acid (EFA) and choline status over a 12-month period. (time frame: 12-month period). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00406536, which also lists 164 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2013.